here - Faculty of Pharmaceutical Medicine

FPM e-newsletter
No. 46 – Autumn 2015
International perspectives on
Pharmaceutical Medicine
Contents
Page 2
Editorial
Asad Khan
Introduction
Ibrahim Farr
Page 5
Global Clinical Trials: A Bridge Too Far
Keith Bragman, Ben Cottam
Page 9
Pharmaceutical Medicine and Market Access in a Free Market Economy: One of Many
Opportunities for the Faculty of Pharmaceutical Medicine in North America?
Peter Gardiner, Declan O’Callaghan
Page 12
A tribute to Dr Jack Watters
Keith Bragman
Page 13
Pharmaceutical Medicine in Africa
Bernd Rosenkranz, Michael Reid, Elizabeth Allen
Page 17
Pharmaceutical Medicine in the Asia-Pacific region
Victoria Elegant, Pippa Biswas, Ritu Sahni
Page 20
Advancing Education and Training in Pharmaceutical Medicine in Europe
Dominique Dubois, Jens Wurthner, Piotr Krzeski, Fergal Donnelly
Editorial
Dr Asad Khan MFPM
Editor
Hello and welcome to the autumn edition of your newsletter! This edition focuses on the practice of
pharmaceutical medicine outside the UK, our international members spread all over the world and the
wonderful work that is being done by our international committee and all the members.
I am sure after reading all the brilliant articles you will realise that from our humble beginnings 25 years
ago, our specialty has come a long way! The diversity of the markets, countries, regulations and
therapeutic areas in which we operate is staggering. A single multi-centric international clinical trial,
pharmacovigilance or medical affairs function may face very different challenges in different parts of the
world and it is this diversity and variety that makes many of us tick and makes our specialty so exciting
and stimulating. Pharmaceutical industries in more and more countries are now beginning to realise
the value, importance and significance of pharmaceutical physicians and I am sure that in the next 25
years our specialty will be duly recognised in all the major markets of the world!
Finally, I would sincerely like to thank our international committee members who have contributed
some very insightful and informative articles. Remember all your suggestions for improvement are
warmly welcome. Please contact me or Ben Cottam in the Faculty office ([email protected]) with
your ideas. Happy reading!
Introduction
Dr Ibrahim Farr FFPM (Spain)
Chairman, International Committee
The International Committee (IC) is one of the operational committees of the Faculty of
Pharmaceutical Medicine (FPM). The committee has been in existence almost since the inception of
the FPM and has become increasingly active in the FPM strategic and tactical planning, in order to
better serve the sizeable (24%) international FPM membership, promote Pharmaceutical Medicine (PM)
globally and make the FPM a reference educational and standard-setting body beyond the UK.
Figure 1: FPM membership – proportion by geographical location – September 2015
2
The main objective of the IC is to support the FPM in its mission "… to advance the science and
practice of pharmaceutical medicine for the benefit of the public". Hence, the IC encourages input to
FPM activities from non-UK based members; helps in promoting the FPM and PM to relevant
international bodies; supports the development of the specialty internationally, including the Diploma
in Pharmaceutical Medicine exam (DPM); harmonises professional and ethical standards globally and
represents as wide a membership as possible. Actually, over the last six years, the IC has evolved from
a Europe-centric Committee to a truly global one with a larger number of members who have
represented or are representing several countries and regions globally: Australia, Belgium, China,
France, Germany, India, Ireland, Italy, Japan, Poland, Singapore, South Africa, Spain, Switzerland, USA
and... counting! Despite the wider spread of the membership of the IC,
we have been able to keep attendance to our four to five meetings held
each year pretty crowded and well above a 75% attendance rate, call on
call. Moreover, we have stimulated the members of the IC to connect
with those FPM members who reside in their respective countries and
bring them into the loop. To start with, we have networked successfully
in North America and Switzerland to engage the local FPM membership
(and pharmaceutical physicians who are not yet members) and cement a
solid partnership for the future, keep a vital link for them with the FPM
and prepare the ground to establish an environment supportive to
upgrade PM as a medical specialty in those regions.
The IC has also led efforts over the last few years to establish ex-UK
venues for the DPM, with the objective of bringing the exam closer to
the place of residence of many potential future members. The
experience has been mixed but we hope to continue to explore the
possibilities in the future. We will also support the development of
events and activities held outside the UK. Finally, we’ve drafted the IC
slides in the FPM slides deck prioritising the activities of the IC and
providing the international members with a tool to promote the FPM
widely in their countries and beyond.
When we were invited to collaborate in this special FPM
e-Newsletter to present and reflect on PM and its international footing, we thought that this is very
timely since the second 3-year tenure of most of the current members of the IC will come to an end
by November 2015. The following sections, which have had contributions by almost all of the IC
members, will reflect on PM development in North-America, Africa, Asia-Pacific and Europe. These
individual sections are introduced by an article comparing and contrasting the different regulatory
environments for the publication of clinical trial data across the major regions of the world. We hope
that this overview of the development of our specialty in these regions will lay the ground for the new
generation of pharmaceutical physicians to continue the progress of our specialty beyond what is now
in many of these countries and regions. We hope you will enjoy this journey throughout our global
footing..!
3
4
Global Clinical Trials: A Bridge Too Far
Dr Keith Bragman MD(Lond) FRCP(Lond) FRCP(Edin) FRCPath PFPM
President, Faculty of Pharmaceutical Medicine
Dr Ben Cottam PhD
Policy and Communications Officer, Faculty of Pharmaceutical Medicine
Introduction
This edition of the newsletter examines the practice of pharmaceutical medicine around the world.
Despite the progress in promoting the needs of the patient and society in medicines development, we
still do not have a uniform global playing field. This is despite the degree of joined-up thinking
throughout the world. Many clinicians, regulators, legislators and the pharmaceutical industry now see
the value of clinical trial registration and results disclosure. Data dissemination and participant
anonymity in clinical trials is currently being debated in the USA and Europe. The European Medicines
Agency (EMA) has recently published proposed requirements. However the uniform translation of
principles into national legislation and then into a regulatory framework remains elusive. In this article
we compare and contrast the ethical guidelines and regulatory requirements in some of the major
regions of the world.
Ethical Guidelines
The current international ethical guidelines are alike in tone and emphasis, though they are not
particularly precise or prescriptive. Let’s begin with the Declaration of Helsinki (October 2013):
35.
Every research study involving human subjects must be registered in a publicly
accessible database before recruitment of the first subject.
36.
Researchers, authors, sponsors, editors and publishers all have ethical
obligations with regard to the publication and dissemination of the results of research.
Researchers have a duty to make publicly available the results of their research on
human subjects and are accountable for the completeness and accuracy of their
reports. All parties should adhere to accepted guidelines for ethical reporting. Negative
and inconclusive as well as positive results must be published or otherwise made
publicly available. Sources of funding, institutional affiliations and conflicts of interest must be declared in the
publication. Reports of research not in accordance with the principles of this Declaration should not be accepted
for publication.
Good Pharmaceutical Medical Practice (FPM, 2014)
Clause 71: The Faculty advocates the following principles [...]:

Timely registration of all clinical trials

Prompt communication of overall clinical trial results to all participants
 Authors of documents that are released into the public domain must be fully
conversant with the study results and supporting data

Summary results must be made public as soon as possible after completion of a clinical trial

The date of release of information should not be dependent upon market or pricing approval
or the continuation or discontinuation of the clinical trial or the whole project

Release of anonymised patient-level data to third parties must be in line with national
regulations
5

Promotional materials and/or information destined for patients, healthcare professionals, and
pricing and reimbursement bodies must be complete, balanced and easily understood by the
intended audience

You are also required to comply with national and international legislation relating to clinical
trial disclosure
The World Health Organisation (WHO) statement on reporting timeframes for clinical trials follows
similar lines (http://www.who.int/ictrp/results/reporting/en/). However, when we examine the actual
regulatory requirements for the registration of trials and reporting of results in the European Union
(EU), United Kingdom (UK), United States of America (USA), Japan, India and Africa, there are marked
differences between regions. For the sake of brevity, only two countries (Japan and India) have been
given in Asia:
Global registration standards
Host
country
or region
EU
Jurisdiction
Registration portal
Public portal
Mandatory?
*
Information **
Timescale
EU
EudraCT
https://eudract.ema.europa.eu
EU Clinical Trials
Register (EU-CTR)
here
Yes
Not required:
Date of first
enrolment
UK
Global
Same
No
All
USA
Global
International Standard
Randomised Controlled Trial
Number (ISRCTN) registry
http://www.isrctn.com/
National Institutes of Health
(NIH) Clinicaltrials.gov
Same
Yes (for trials
under FDAAA
801)
‘Protocol Data
Element
Definitions’
outlined here
Japan
Japan
Japan Primary
Registries Network
(JPRN)
http://rctportal.niph.
go.jp/
No
All +
Items according
to local needs
to form a set of
85 registration
items
India
India
University Hospital Medical
Information Network (UMIN
CTR)
http://www.umin.ac.jp/ctr/
Japan Pharmaceutical
Information Center - Clinical
Trials Information (JapicCTI)
http://www.japic.or.jp/
Japan Medical Association Center for Clinical Trials
(JMACCT CTR)
http://www.jmacct.med.or.jp/
Clinical Trials Registry – India
http://ctri.nic.in/
Trial must be
registered before
the first patient is
recruited.
Not specified
(encouraged to be
before first patient
enrolment)
No later than 21
days after
enrolment of the
first participant
(with some
exceptions here)
Not specified
(recommended on
or before first
patient enrolment)
Same
Yes
South
Africa
(SA)
Africa
Pan African Clinical Trial
Registry (PACTR) pactr.org
(includes South African
National Clinical Trial
Register (SANCTR))
Same
No
All + additions
http://www.who
.int/bulletin/volu
mes/86/8/08010808/en/
All
(mandatory in
SA)
Not specified
(recommended
before enrolment of
first participant)
Not specified
(“ideally” before the
enrolment of the
first participant)
* In all cases, this refers to studies that meet the WHO/ICMJE 2008 definition of a clinical trial
** Deviation from WHO Trial registration dataset. The minimum amount of trial information that must appear in a register
in order for a given trial to be considered fully registered. There are currently 20 items in the WHO Trial Registration
Data Set. http://www.who.int/ictrp/network/trds/en/
6
Reporting of trial results
Publication of summary results
Portal
EudraCT
clinicaltrials.gov
General requirements
Ref Annex IIIa of regulation EU No
536/2014. The results for phase I-IV
clinical trials are to be posted:
o summary results and layperson
summary – 12 months after the
end of the trial
o the clinical study report of trials
– 30 days after the decision on
marketing authorization or
withdrawal
Summary results must be published,
whether the drug has been approved
for use or not, no later than 12
months after the Completion Date.
Exemptions
 Phase I trials, conducted solely in adults and which are
not part of an agreed Paediatric Investigation Plan (PIP)
o Protocols, subject information sheets, Investigational
Medicinal Product Dossiers (IMPDs) and investigator
brochures, may be deferred differentially
o Exceptions to publication are personal data of trial
subjects, IMPD quality/manufacturing section, and the
financial contracts
Submission of results may be delayed if either:
o The trial reached its Completion Date before the drug
(or biologic, or device) is initially approved, licensed, or
cleared by Food and Drug Administration (FDA) for any
use (no later than 30 days after the drug or device is
approved, licensed or cleared)
o The trial studies a new use of an FDA-approved drug
and manufacturer filed or will file within 1 year an
application to FDA for approval or clearance of that
use.
Results deadline:
1) The earlier of the date that is 30 days after the
date that:
 The new use of the drug or device is
approved, licensed, or cleared by FDA
 FDA issues a letter for the new use of the
drug or device, such as a complete response
letter
 The application or premarket notification for
the new use is withdrawn without
resubmission for no less than 210 days
or
Japan
India
No legal requirement
However, member companies of the
Japanese Pharmaceutical
Manufacturers Association (JPMA) are
‘required’ to abide by the international
‘Joint Position on the Disclosure of
Clinical Trial Information via Clinical
Trial Registries and Databases’
Since April 2009, the Japanese Ministry
of Health, Labour and Welfare
(MHLW) has issued and applied the
latest version of the ‘Ethical Guidelines
for Clinical Research’. Although only
administrative guidelines without legal
force, they are considered to be 'soft’
law and perceived as having
effectiveness.
Not currently a requirement
2)
2 years after the date a certification is
submitted, if none of the events listed above has
occurred
n/a
n/a
7
Publication of full data set
Portal
Requirements
Exemptions
EudraCT/EMA
The European
Medicines Agency
(EMA) is the only
jurisdiction to date
that has developed a
framework for the
public release of
study reports (upon
request).
The following (including appendices)
will be available upon request:
module 2.5 (clinical overview)
module 2.7 (clinical summary)
module 5 (clinical study reports
(CSRs)
• Clinical reports available on-screen
for any user, with a simple
registration process;
• Downloadable clinical reports
available to identified users
Commercially confidential information
(proposed by the applicant or market
authorisation holder and agreed by the
EMA)
Currently – individual patient data
(IPD) (see ‘future implementation’)
Future
implementation
The EMA will also
work to find the
most appropriate
way to make IPD
available, in
compliance with
privacy and data
protection laws.
The world is still very heterogeneous
Most large pharmaceutical companies now have their own clinical trial
data portals. In some cases these go beyond the local regulatory
requirements. A number of companies – Astellas Pharma Inc., Bayer
AG, Boehringer Ingelheim GmbH, Eisai Co. Ltd, GlaxoSmithKline, Eli
Lilly and Company, Novartis AG, Roche Pharmaceuticals, Sanofi S.A.,
Takeda Pharmaceutical Company Ltd, UCB and ViiV Healthcare – have
formed an alliance called Clinical Study Data Request –
https://www.clinicalstudydatarequest.com/ – where researchers can request anonymised patient-level
data from these companies. The requests are reviewed by an independent panel.
Links to the major company policies and portals are provided below:
GlaxoSmithKline – http://www.gsk.com/en-gb/our-stories/how-we-do-randd/data-transparency/
Roche Pharmaceuticals – http://www.roche-trials.com/
Pfizer – http://www.pfizer.com/research/clinical_trials/trial_data_and_results
Novartis AG – https://www.novartis.com/our-work/research-development/clinical-trials/clinical-trial-information-disclosure
Sanofi SA – http://en.sanofi.com/rd/clinical_trials/our_data_sharing_commitments/our_data_sharing_commitments.aspx
Merck – http://www.merck.com/clinical-trials/policies-perspectives.html
AstraZeneca – http://astrazenecagrouptrials.pharmacm.com/
Conclusion
There is no doubt that we are becoming a more open society. However, differences in clinical trial
registration and the reporting of results between countries and regions are still slowing the progress
of transparency. The absence of a level playing field between countries or regions is continuing to
hinder the flow of information and leads ultimately to market differences. The International Conference
on Harmonisation (ICH) is now 25 years old. ICH has become an extraordinarily effective tool.
Openness and results publication with dissemination of data is in its infancy. Attitudes may be changing,
but we still have to travel a long way from attitude to legislation in search of a global standard.
What are your thoughts on the international standards for clinical trial publication?
Do you agree that better harmonisation is required globally?
Are the current systems adapting quickly enough to reflect public opinion?
Get in touch [email protected]
8
Pharmaceutical Medicine and Market Access in a Free Market
Economy: One of Many Opportunities for the Faculty of
Pharmaceutical Medicine in North America?
Authors
Dr Peter Gardiner MB ChB, MRCP, FFPM (U.S.A.)
Senior Pharmaceutical Physician and President, Gardiner Consulting, LLC
Dr Declan O’Callaghan MB BCh BAO (Republic of Ireland)
Medical Director, Wyeth Pharmaceuticals Ltd
Market access
As pharmaceutical physicians, we have always been involved in the cost of drug development – typically
being asked how we can reduce clinical trial expenditures and yet complete study enrolment and
analysis faster. However, we are becoming increasingly involved in advising on the acquisition and
interpretation of clinical data that supports economic modeling and, ultimately, the pricing of the drugs
that companies are developing and commercialising. Evaluating cost-effectiveness and supporting the
efforts of our pricing and reimbursement colleagues are becoming equally as essential as demonstrating
clinical efficacy and safety. “Market Access” is an area of drug development and commercialisation with
which we all need to be increasingly familiar, as the focus shifts to “who pays the bill?” It is curious that
our industry has focused extensively on educating prescribers about the benefits of medicines, yet
these individuals are not directly accountable for the costs of their therapeutic decisions - it is always
easier to spend other people’s money than your own! Whilst it is hard to convince governments to
approve and reimburse medicines, it is harder still to persuade insurers to list them on formularies,
and hardest of all to persuade patients to pay for the entire cost of the medicine themselves.
One size does not fit all. Market access models range from centralised,
nationally controlled systems, such as in France, through mixed systems
with national price setting but regional formulary processes governing
ultimate access (e.g. Canada and United Kingdom), to the decentralised
system in the United States of America (USA). Although this is changing
in the USA, with rapid consolidation of health care insurers and the
increase in the insured population through the Affordable Care Act.1
Health Technology Assessment (HTA) is a critical component, if not a
requirement, for market access in almost every major pharmaceutical
market. The United Kingdom’s National Institute for Health and Care
Excellence (NICE) has been a pioneer in guideline and standard setting in this regard.2 In the USA, the
Boston-based, non-profit Institute of Clinical and Economic Review (ICER) has been credited with
helping some drug plan managers secure big discounts on new hepatitis C drugs, including sofosbuvir.3
Whilst there is no doubt sofosbuvir has revolutionised the treatment of patients with hepatitis C, can
the USA health care system tolerate a drug initially priced at $1,000 per tablet (or $134,000 for the
minimum recommended 12 weeks of treatment)?4 The 120,000 patients reportedly treated to date
are less the issue, compared to the 1.4 million infected, diagnosed, but not treated and the additional
1.5 million infected but not yet diagnosed.5 It has recently been reported that yet-to-be-published
recommendations from the American Association for the Study of Liver Diseases and the Infectious
Diseases Society of America will include, for the first time, cost-effectiveness language and conclude
that new treatments for hepatitis C are cost-effective.6 Interestingly, the costs referenced in this
assessment are apparently after discounts.
9
This debate over the cost of drugs for rare diseases extends to other orphan diseases. The US Food
and Drug Administration granted orphan drug status to 43 medicines in 2014 – 10 years ago only 10
therapies had orphan status.7 Worldwide sales of orphan drugs were less than $20 billion in 2000, but
are forecast to be of the order of $176 billion by 2020.7 Synageva
has recently submitted its first product for approval – a drug to treat
a disease affecting just 3,000 people in the developed world.7 It is
perhaps not surprising that Synageva is led by an ex-Genzyme
executive; Genzyme pioneered the approach to developing drugs
for rare diseases and marketing them at high prices, with the
introduction of its therapy for Gaucher’s disease in the 1990s.
Of note, ICER has recently received a $5.2 million grant from a Houston philanthropic foundation to
expand their study of whether new drugs are worth the price tag.8 One of the next projects will be to
assess the new, potent cholesterol-lowering drugs, known as PCSK9 inhibitors. The first of these drugs,
alirocumab, was just approved by the FDA on July 24, 2015.9 Given the huge potential market for this
class of drug, the implications for health care budgets could be overwhelming – an initial price of $40
per day ($14,600 per year) has been reported.10 Following a recent, positive Committee for Human
Medicinal Products opinion, alirocumab has been submitted to the European Medicines Agency for
approval.
The costs of new drug research and development and, in particular, the cost of failure, are often used
to justify the high price of pharmaceutical products. This issue received attention recently with the
disclosure of an updated assessment from the Tufts Center for the Study of Drug Development
(CSDD).11 Tufts CSDD calculated the cost of developing and gaining market approval for a new drug
as $2.6 billion. This is a substantial increase from their 2003 estimate of $802 million and the
methodology has certainly been criticised; for example, they included $1.2 billion as the cost of capital,
with only $1.4 billion attributed to actual funds spent on research and development.12
Clearly, cost-effectiveness and market access are topics of relevance to all pharmaceutical physicians,
regardless of where in the world we work. The Faculty of Pharmaceutical Medicine is well placed to
serve as an educational and mentoring resource for this and many other issues of relevance to our
day-to-day work and to our continuing professional growth and development.
Pharmaceutical Medicine in the USA – structure, organisations and resources
Given the size and scale of the N American pharmaceutical industry and marketplace, recent
discussions by the FPM International Committee, as well as at the FPM Board level, led to an initial
outreach in 2014 to those members and fellows of the FPM who live and work in N America. Of
approximately 100 physicians contacted formally by the FPM, about 20 responded and a number of
informal teleconferences were held. The principal topics discussed included opportunities for
networking, the role of the FPM in education and training, and revalidation for UK-licensed physicians
working in N America. A face-to-face meeting of interested and available colleagues was held in June,
during the recent Drug Information Association (DIA) 2015 meeting in Washington DC. We were
very fortunate to be joined, in-person, by Dr. Keith Bragman, President of FPM and Dr. Robert Hardi,
President of the Academy of Physicians in Clinical Research (APCR).
The vision of APCR is that clinical research is performed ethically, responsibly, and professionally
everywhere in the world. APCR’s mission is to promote excellence in clinical research and is the
leading organisation championing the interests and perspectives of clinical research professionals.
APCR plays an active role in public discourse of clinical research and in raising awareness of the issues
important to clinical research professionals.13 APCR’s focus is on clinical research and a substantial
proportion of its members are academic clinical investigators. There is currently no professional
organisation in the USA directly representing the interests of physicians in the pharmaceutical (or
10
medical device) industry.
The FPM N American Network meeting held in Washington DC was an interactive, brain-storming
session that covered a variety of topics. These included:
•
•
•
•
•
How can the FPM effectively represent the needs and views of its North American members?
How can the views and needs of the N American FPM community, and of other physicians
working in the pharmaceutical, medical device and allied businesses, be effectively
communicated to the FPM and, as appropriate, incorporated into the FPM’s policies, practices
and priorities?
Are there opportunities beyond networking, education/training & revalidation?
Are there opportunities for collaboration with other organisations, for example: APCR,
International Federation of Associations of Pharmaceutical Physicians (IFAPP), DIA?
What is the level of interest in continuing the FPM outreach in N America and how to do so?
In addition to Drs. Bragman and Hardi, we were joined by a number of members and fellows of the
FPM, as well by representatives of IFAPP and DIA. Feedback was, in general, positive and it was felt
that the dialogue amongst interested members and fellows in N America should continue. All
suggestions and input regarding this effort are welcome. Some of the ideas under consideration include
exploring what motivates the current FPM membership in N America to remain members or fellows
and what are the demographics of the group, in terms of location, current and former employment,
experience, etc. Also, pharmaceutical medicine has limited recognition in the USA and is not
considered an established medical specialty, as it is in the UK. So, for example: how might FPM
contribute to creating interest in and awareness of the specialty? In addition to the groups represented
at our introductory meeting, it may be appropriate to consider appropriate engagement with the FDA
or other interested parties, such as the Institute of Medicine.
With the aging population and increased longevity, the demands and stresses on health systems in the
USA, and elsewhere in the world, will continue to increase. As an established, global organisation
representing the varied medical roles and functions within pharmaceutical medicine, the FPM has the
potential to play a critical role in N America. Please send your comments and suggestions regarding
the most appropriate roles for the FPM in N America to the Faculty at [email protected].
References
1.
2.
3.
4.
5.
6.
7.
8.
9.
10.
11.
12.
13.
Affordable Care Act; March 2010; www.hhs.gov/healthcare/rights [Accessed 10 August 2015]
National Institute for Health and Care Excellence; www.nice.org.uk/about [Accessed 10 August 2015]
Institute of Clinical and Economic Review; www.icer-review.org/ [Accessed 10 August 2015]
Sovaldi® (sofosbuvir) Prescribing Information; March 2015
T Brennan, MD, CVS Caremark; Boston BioTech CMO Meeting; presentation - Boston CMO Network; May 2015
Hepatitis C drugs called cost-effective; Boston Globe; July 14, 2015
High prices for drugs to treat rare diseases take a toll; Boston Globe; May 12, 2015
Rising US drug prices are focus of research grant; Wall Street Journal; July 21, 2015
FDA approves Praluent to treat certain patients with high cholesterol; FDA News Release July 24, 2015;
www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm455883.htm [Accessed 10 August 2015]
Regeneron and Sanofi Announce FDA Approval of Praluent® (alirocumab) Injection, the First PCSK9 Inhibitor
in the U.S., for the Treatment of High LDL Cholesterol in Adult Patients; Press Release July 24, 2015;
www.prnewswire.com/news-releases/regeneron-and-sanofi-announce-fda-approval-of-praluent-alirocumabinjection-the-first-pcsk9-inhibitor-in-the-us-for-the-treatment-of-high-ldl-cholesterol-in-adult-patients300118572.html [Accessed 10 August 2015]
How the Tufts Center for the Study of Drug Development pegged the cost of a new drug at $2.6 billion –
Backgrounder; November 18, 2014; http://csdd.tufts.edu/files/uploads/cost_study_backgrounder.pdf
Avorn J. The $2.6 billion pill – methodologic and policy considerations. N Engl J Med 2015;372:1877-9
Academy of Physicians in Clinical Research; www.apcrnet.org/FunctionalMenuCategory/AboutAPCR.aspx
[Accessed 10 August 2015]
11
A tribute to Dr Jack Watters
Dr Keith Bragman
President
Dr Jack Watters, Vice President Pfizer, New York and Trustee of the Faculty of Pharmaceutical Medicine
(b 1952; q University of Edinburgh 1977), died after a short illness on the 30th June 2015.
Dr Jack Watters died earlier this year after a short illness from advanced cancer. I, for one, will
miss his humour, self-deprecating style and wise contribution to the Faculty Board of Trustees.
After first qualifying in medicine, working in Edinburgh and Basel, Jack moved to New York in
1989 and subsequently became a US citizen. He rose to become Group Medical Director for
Sterling Winthrop, where he had global responsibility for the portfolio.
He subsequently moved to Pfizer in 1994. He passionately believed in the power of the industry
to make a positive contribution to society. He became a Vice President with responsibility for the
company’s external medical affairs. This included the Diflucan (fluconazole) franchise, which
contributed to people living with HIV and Aids. He also led Pfizer’s approach to healthy ageing,
established a centre of excellence in paediatrics, and maintained the company’s contribution to
clinical care across the world. So many individuals are living longer and healthier lives because of
Jack’s commitment to medical care.
He was an activist for equal rights, he served on the board of the American Federation for Age
and Research (AFAR), and he sat on the health technology advisory panel for the University of
Strathclyde. Three years ago, he joined the Faculty Board of Trustees. Jack always believed that
the Faculty was responsible for the principles by which pharmaceutical medicine should be
practised, and that, first and foremost, we had an obligation to the patient and society.
I used to see Jack regularly at Faculty Board meetings. We used to debate the issues of the day.
Jack was always practical and understood the implications of the bigger picture. He was widely
read, a thoroughly decent human being, and a true humanist. He will be greatly missed.
12
Pharmaceutical Medicine in Africa
Authors
Prof Bernd Rosenkranz MD PhD FFPM (South Africa)
Head, Division of Clinical Pharmacology, Faculty of Medicine and Health Sciences, Stellenbosch University, and
President, Fundisa African Academy of Medicines Development
Dr Michael Reid MBChB MSc MedSci PG Dip Pharm Med (South Africa)
Senior Medical Manager South Africa and Sub-Saharan Africa, Pfizer Laboratories (Pty) Ltd
Dr Elizabeth Allen BSc (Hons) Pharmacy MPH (Epi) (South Africa)
Senior Clinical Research Manager, Malaria Clinical Research Group and Project Manager, Global Health Trials South
Africa, Division of Clinical Pharmacology, Department of Medicine, University of Cape Town
Pharmaceutical economic environment in Africa
According to a McKinsey Report, Africa is one of the world’s fastest-growing economic regions, with
a rise in its pharmaceutical industry value from $4.7 billion in 2003 to $20.8 billion in 2013.5 Africa’s
estimated growth rates between 2013 and 2020 are in the range of 6 and 11% for prescription drugs,
generics, over-the-counter (OTC) drugs and medical devices. The growth in medical care as a whole
is reflected in the acquisition of 70,000 new hospital beds, 16,000 doctors, and 60,000 nurses in Africa
between 2005 and 2012.
Besides the well-known burden of infectious diseases (HIV/AIDS,
tuberculosis, malaria and tropical diseases), non-communicable diseases
(NCDs) are increasingly important in Africa. NCDs accounted for 28% of
morbidities and 35% of mortalities in Africa in 1990, with a projected rise
to 60% and 65% respectively by 2020.10
Africa is, however, a very diverse continent of 54 countries, each with its
own economic landscape and political complexities. The top 5 pharma
markets (billion US-$, IMS Health 2014 estimates) – South Africa (4.9), Egypt (3.9), Algeria (3.5),
Morocco (1.8) and Nigeria (1.2) – account for about 70% of the total African market value. Within
each country, there are major differences between the rich and the poor, with major cities responsible
for a large part of the economic growth. The oil price collapse and crises, such as the recent Ebola
outbreak, contribute to economic instability and regional differences.
As everywhere else, pharmaceutical companies in Africa are exposed
to mergers and acquisitions, joint ventures, strategic alliances,
partnerships, and private-equity deals. South African generics company
Aspen Pharmacare has grown to be one of the top ten generic
companies in the world. Such expansion of the local pharmaceutical
industry requires trained pharmaceutical specialists to support it. The
bigger multinational pharmaceutical companies are increasingly active
in drug production and clinical trials across Africa. According to
McKinsey, more than 300 companies have drug manufacturing sites on
the continent.5
Clinical trials across all phases have a long tradition in South Africa in particular, with an estimated
annual value of about 1.3 - 2 billion Euro.3 About 270 new trials start every year, and 550 trials are
active in the country at any given time.8
13
Impact on pharmaceutical industry
In the industry, these developments have led to the establishment of medical departments within
pharmaceutical companies and subsequently of contract research organisations (CROs) during the
1980s. In South Africa, physicians working in the pharmaceutical industry were organized in the South
African Association of Pharmaceutical Physicians (SAAPP), which had 83 members in 2005. This
organization is currently being re-activated after a period of dormancy. Clinical research specialists
meanwhile are represented by the South African Clinical Research Association (SACRA), an active
nonprofit organization that has regular consultation with the regulatory body about pertinent issues,
is involved with the move towards the registration of Good Clinical Practice training providers and
hosts an annual conference.
Clinical research in sub-Saharan Africa is mainly performed by international and local companies, but
increasingly also by clinicians, in the form of investigator-initiated trials. There is a clear need for more
and better clinical research in Africa addressing the major contributors to the burden of disease in this
part of the world.2,7
Another challenge is the ever changing regulation of the pharmaceutical marketplace. In South Africa,
certification with the Marketing Code Authority (MCA) established in 2014 is a requirement, and
pharmaceutical professionals must comply with its code of conduct. Employees must be adequately
trained. Unfortunately, other African countries do not yet have such an oversight body and hence
minimal guidance on marketing of medicines.
Medicines regulation
Regulatory agencies in Africa are also struggling. The South African Medicines Control Council (MCC)
is the most established regulatory body in sub-Saharan Africa. Because of the increasing complexity of
product submissions and the present working model which relies on external reviewers mainly from
universities and research institutions, review timelines are long and unpredictable. Delays in clinical
trial submissions can also be off-putting to sponsors considering South Africa within their clinical
development plans, although typically good recruitment rates may make up for lost time. Legislative
amendments to change this situation are leading to the formation of a new agency, the South African
Health Products Regulatory Agency (SAHPRA). A critical mass of trained internal and external
reviewers will be required to carry out the regulator’s mandate. For low and middle income countries,
WHO promotes the establishment of regional multinational joint regulatory organizations to reduce
the delayed uptake of new medicines, vaccines and medical innovations.6
Teaching and training of competencies in medicines development and regulation
The increasing need for experts and accredited specialists in pharmaceutical
companies, clinical trial groups and regulatory agencies across Africa needs to be
addressed with urgency.
In the medical affairs departments for example, pharmaceutical companies across
Africa have to hire personnel without necessary professional exposure and
competencies for their roles. Thus, there is a reliance on “on the job training”. As
the pharmaceutical landscape evolves, the role of the pharmaceutical specialist in
translational medicine has become ever more important. Principles of evidencebased medicine are being applied more regularly in patient management, requiring a pharmaceutical
specialist who understands the science of medicines development and is able to communicate this into
clinical practice. Thus, with limited training in pharmaceutical medicine in Africa, “on the job training”
will not be adequate to provide all necessary competencies.
Whereas Pharmaceutical Medicine is not a specialty in any of the African countries, Clinical
14
Pharmacology has been recognised in South Africa as a full medical specialty since 2009.9 In 2011, the
Faculty of Pharmaceutical Medicine exported its Diploma in Pharmaceutical Medicine examination to
South Africa as a pilot project. Unfortunately, this initiative has subsequently been abandoned due to
the lack of medically qualified candidates prepared to take this examination. However, this may be
revisited in the future.
Regulatory science and medicines development are not taught at undergraduate level in Africa.
However, several South African universities have developed bachelor and/or master courses in
regulatory science, drug development, pharmacoeconomics, pharmacovigilance and pharmaceutical
affairs. In 2010 a fully accredited 2-year diploma programme in medicines development was started at
Stellenbosch University following the PharmaTrain syllabus and standards. As the first non-European
training course, this programme is accredited as a Centre of Excellence by the PharmaTrain Federation.
Non-academic training programmes and workshops in medicines development and drug regulation are
also offered by the Fundisa African Academy of Medicines Development (www.fundisa-academy.com)
which presented the first Clinical Investigator Certificate (CLIC) course according to the PharmaTrain
syllabus this year.
The MCC will require a new model of staff training, combining capacity building through a blended elearning platform, including mentoring, workplace assignments and specific courses in cooperation with
the universities and other training providers. Recommendations for the training of regulatory experts
in South Africa were provided by the EU-funded Ecorys Health Consortium. This year, the Institute
for Regulatory Science (IRS) was established in the National Department of Health to coordinate
training relevant for agency staff, clinical trial specialists and industry employees in the region.
Ongoing initiatives for capacity building in medicines development and regulatory sciences in emerging
African countries were discussed at the 4th African Regulatory Conference held in Dakar, Senegal in
April 2015. Progress has been made in the development of pharmacovigilance in Africa, and some
African countries have become members of the WHO Programme for International Drug Monitoring.
In 2010, WHO designated the University of Ghana Medical School as a WHO Collaborating Centre
for Advocacy and Training in Pharmacovigilance.
An initiative with no borders is The Global Health Network, which provides a website forum for
clinical investigators and their teams to address technical competence, and knowledge-sharing.1 This
offers open access, peer-reviewed e-learning products, tools and templates, articles, discussion forums
and a professional membership scheme that supports career development in clinical trials. Since its
launch in 2010 at Oxford University, this website has become widely popular, with more than 230,000
visits recorded to date.
Following a workshop at the 17th World Congress of Basic and
Clinical Pharmacology (WCP2014) in Cape Town4, PharmaTrain
Federation and the International Federation of Associations of
Pharmaceutical Physicians & Pharmaceutical Medicine (IFAPP)
jointly established a Working Group on Education of Medicines
Development in Low and Middle Income Countries. Zimbabwe,
Ghana and Ethiopia were selected as initial African pilot countries
in which to consider expanding the IFAPP-PharmaTrain remit to these regions.
References
1.
2.
3.
The Global Health Network. Global Health Trials. https://globalhealthtrials.tghn.org/ (accessed 21 July 2015)
Isaakidis, P, Swingler, GH, Pienaar, E, Volmink, J, Ioannidis, JP. Relation between burden of disease and
randomized evidence in sub-Saharan Africa: survey of research. BMJ 2002 324(7339);702-706
Kahn, M, Gastrow, M. Pharmacologically active: clinical trials and the pharmaceutical industry. S Afr Med
J 2008 98(2);114-116
15
4.
Kerpel-Fronius, S, Rosenkranz, B, Allen, E., Education and training for medicines development, regulation and
clinical research in emerging countries. Front Pharmacol 2015 6:80. doi:10.3389/fphar.2015.00080
5. McKinsey & Company Report. Insights into Pharmaceuticals and Medical Products in Africa: A Continent of
Opportunity for Pharma and Patients, 2015.
www.mckinsey.com/insights/health_systems_and_services/africa_a_continent_of_opportunity_for_pharma_an
d_patients, 2015 (accessed 03 July 2015)
6. Rago, L. Regulatory appraisal of biological medicines and harmonization initiatives for availability and access.
17th World Congress of Basic and Clinical Pharmacology (WCP 2014), Cape Town, South Africa; Biological
medicines development (PharfA Symposium 3)
7. Siegfried, N, Clarke, M, Volmink, J. Randomised controlled trials in Africa of HIV and AIDS: descriptive study
and spatial distribution. BMJ 2005 331(7519);742-747
8. Strugo V, Katsoulis L, Chikoto H, Southwood T, Coetzee M. Conducting clinical trials in South Africa. In: Chin
R & Bairu M (eds): Global Clinical Trials (Chapter 12), Elsevier Press, 2011
9. Walubo, A, Barnes, K, Kwizera E., et al Clinical Pharmacology becomes a specialty in South Africa. S Afr Med J
2013 103;150-151
10. WHO, African Health Observatory.
www.aho.afro.who.int/profiles_information/index.php/AFRO:Disease_burden_-_Noncommunicable_diseases_and_conditions, 2015 (accessed 03 July 2015)
16
Pharmaceutical Medicine in the Asia-Pacific region
Authors
Dr Victoria Elegant FFPM LRCP MRCS DRCOG (China)
Vice-President, Medical and Regulatory Affairs, Asia-Pacific, Baxter Asia-Pacific
Dr Pippa Biswas MFPM (U.K.)
Director, Symogen Ltd
Dr Ritu Sahni FFPM MRCP (Australia)
Independent Pharmaceutical Physician
The Asia-Pacific region is extremely vibrant, fast moving, and characterised by its extreme
heterogeneity from a medical, regulatory and economic perspective. It is also unique, in that several
countries in the region require local clinical data on their own populations in order to register
compounds, and also have mandatory requirements for post-marketing surveillance studies.
It includes both developed countries – South Korea, Australia, New Zealand, Taiwan, Hong Kong,
Singapore, Japan, and developing countries – China, India, Vietnam, Philippines, Indonesia, Malaysia,
Thailand, and others. There are 35 countries in the region, each with their own culture and language
– in many countries such as India and China, there are multiple regional languages.
All countries in Asia-Pacific (APAC) require a New Drug Application (NDA) to contain sufficient data
to support the efficacy, safety and quality of a pharmaceutical product before their regulatory agencies
can approve its marketing authorisation.
Clinical data submitted to support efficacy and safety of the
pharmaceutical product should be generated from well-designed
trials conducted in accordance with Good Clinical Practice (GCP).
They should be robust enough to stand up to reviews by wellestablished regulatory agencies such as the US Food and Drug
Administration (FDA) or European Medicines Agency (EMA).
There are however certain specific considerations that need to be
taken note of when preparing for NDA filing in countries in this
region. Many APAC countries require the prior marketing
authorization approval in a reference country/countries and/or the
country of origin of a pharmaceutical product has been obtained,
before they in turn issue their approval based on a summary
review complemented by such a reference approval. As such, the
Certificate of Pharmaceutical Product (CPP) is required as part of
the NDA dossier in these countries. Some countries will require
the CPP at the time of submission, while some countries can
accept an initial submission dossier without a CPP and start their
review first but require that the CPP be furnished at a later period so that they can issue their approval
after their summary review.
There is little harmonisation between the regulatory systems in each country. In the ASEAN region –
Singapore, Malaysia, Thailand, Vietnam, Brunei, Myanmar, Philippines, Cambodia, Laos and Indonesia,
much work is ongoing to harmonise requirements and regulations. Most countries have adopted the
ASEAN CTD for submissions. The ASEAN region also has specific Zone IV stability requirements.
17
Local Data
Requirements
Local Phase I/
Pharmacokinetic
(PK)
requirements
Local subject
number in Phase
III
Post marketing
surveillance
China
Korea
Taiwan
Vietnam
India
Yes – local PK
and it may be
conducted in
parallel with
Phase III study
I – 20 subjects
Per regulations:
Phase III – 100
(small
molecules) and
300 (large
molecules) in
active arm +
statistical
significance
Maybe
Not
required
Not required
Not required
Not
required
30 to 50 in
active arm
(in
practice)
Per regulations:
If total n>200, 5%
or 30 whichever
is lower,
If total n < 200,
10% or 10
whichever is
lower
~30 in active
arm (in
practice)
100 in total
Yes
No
No
Yes
Table 1: Requirements for local clinical data required for NDA submission
India has updated its regulations recently, following accusations that there were many clinical trials
being conducted in India without adequate protection for subjects. The new regulations mandate fixed
compensation according to a specific formula, review of trials by the Drug Controller General of India
(DCGI), and video taping of subject consent. Initially almost all trials in India stopped while the
regulations were being updated, but in the last year the number of companies conducting clinical studies
in India has increased. There are excellent centres with investigators experienced in conducting trials.
The ministry is also taking steps to accredit and register clinical trial centres.
China is currently possibly the toughest regulatory environment, with imported
new chemical entities (NCEs) taking up to six years for registration. Local
clinical trials to collect local data are required, and the backlog of application
for Clinical Trial Permissions due to the high volume and relative under-staffing
at the China Food and Drug Administration (CFDA) is proving frustrating for
many companies. The CFDA recently released guidelines for the conduct of
multi region clinical trials (MRCT), with at least three countries including China,
for unmet medical needs and life threatening disease, which may help to speed
up registrations. This month, a new guidance was issued. This describes (i)
launching a pilot programme of a market authorisation holder (MAH) system
for drugs, (ii) allowing synchronous in-country clinical trials for new drugs that have not yet been
marketed overseas, and (iii) adopting qualified clinical data obtained directly from multi-centre clinical
trials.
Overall, the reform emphasises improving the efficiency of the review system, resolving the drug
application backlog by the end of 2016, improving the quality of the generic drugs, encouraging the
development of innovative drugs and creating a more transparent review and approval process.
Japan has required local data for many years, and is now open to multi-region or global trials including
Japanese patients, provided the number of Japanese patients is sufficient to meet the Japanese
regulatory requirements. Last year, the Pharmaceutical Affairs Law was revised in order to strengthen
safety of drugs and device registration. It is now the third largest market in the world, after the US and
China, and so remains very attractive.
In Australia, current data suggest that around 1000 new clinical trials are commenced in Australia each
year by pharmaceutical, biotechnology and medical device companies, representing a $1 billion
investment. The top 10 global pharmaceutical companies are responsible for 20% of this investment.1
18
There has been a shift in the type of clinical trial activity conducted in Australia. Historically, Phase III
studies have dominated. However, since 2008, there has also been a significant increase in early-phase
activity, reflecting global trends, along with local capabilities in early-phase clinical research.
Australia boasts a quality medical research infrastructure and a skilled workforce, a world-class
healthcare system, a stable socio-economic environment, an ethnically diverse population and a strong
intellectual property regime. An efficient regulatory system, including a rapid clinical trials approval
system, its proximity to Asia, a strong mechanism of support services, streamlined processes and
globally competitive tax incentives for research and development (R&D) investment, all contribute to
making it attractive for conducting trials.
There is a national focus on continuous improvement of the industry through
government reform and policy innovation. Two significant examples are the
Therapeutic Goods Administration (TGA) reforms and the Australian Government
Clinical Trials Initiatives.
The TGA is responsible for the regulation of medicines and medical devices in
Australia. In December 2011 a comprehensive package of TGA reforms, drawn
together by the Australian Government, were announced in TGA reforms: A blueprint
for the TGA’s future.2 The blueprint reforms aim to improve community understanding
of the TGA’s regulatory processes and decisions, enhance public trust and confidence
in the safety and quality of therapeutic goods. A four-year implementation plan was put into place,
focusing on the areas of communication and stakeholder engagement, advertising of therapeutic
products, complementary medicines, medical devices and promotion of therapeutic products.
The Australian Government, in partnership with industry and other stakeholders, is currently
undertaking initiatives (Australian Government Clinical Trials Initiatives) to improve the clinical trials
environment in Australia, whilst maintaining the highest quality and ethical standards.3 These include a
focus on reducing study start-up times, working towards a nationally consistent approach to clinical
trials, boosting patient recruitment and developing a standard list of costs for clinical trials.
There are a number of ongoing activities aimed at advancing education in pharmaceutical medicine in
the region. There are courses already available in Beijing, China, Osaka, Japan and at the University of
New South Wales (UNSW), Sydney, Australia. The recently formed cross industry group in China –
China Medical Affairs Network – held a 3 day training course in Shanghai in December 2014 which had
30 attendees, and the group is intending to repeat this as it was well received. Discussions are ongoing
with the group and the UNSW to customise the offering from UNSW to meet the needs of the
Chinese environment4. Since the course and assessments are all online, we are confident it can be
adapted for China and potentially the rest of the region. Education of this kind is a critical requirement
to keep pace with the growth in the industry, and in particular the growth in clinical trials and medical
affairs activities needing experienced, well trained people.
In summary, the region is fast moving, with a constantly changing environment, and with many countries
taking steps to improve their healthcare, including strengthening registration and safety requirements.
It is critical to have people on the ground, who keep abreast of all the changes and can help shape the
environment in order to bring much needed therapies to patients in the region.
References
1.
2.
3.
4.
Clinical Trials Capability Report, Australian Trade Commission, Australian Government, May 2015
Delivering Reforms- Implementation plan for TGA Reforms: A blueprint for TGA’s future, Australian
Government, July 2012
https://www.australianclinicaltrials.gov.au/australian-government-clinical-trials-initiatives
Opinions Concerning the Reform of the Review and Approval System for Drugs and Medical Devices Issued by the State
Council (the “Opinions”, Guo Fa Notice [2015] No.44)
19
Advancing Education and Training in Pharmaceutical Medicine in
Europe
Authors
Dr Dominique Dubois MD BCPM FFPM (Belgium)
Physician Specialist in Pharmaceutical Medicine, Vice-Chairman Pharmed post-graduate programme in
Pharmaceutical Medicine and Medicines Development Sciences
Dr Jens Wurthner MD PhD FFPM (Switzerland)
Lead Clinical Program Leader, Translational Clinical Oncology, Novartis Institutes for BioMedical Research
(NIBR)
Dr Piotr Krzeski MD PhD FFPM (Poland)
Medical Director, Medpace
Dr Fergal Donnelly MD FFPM (Belgium)
Principal Scientific Officer European Commission DG Research & Innovation
The PharmaTrain education and training initiative
Competency-based education is founded on
competencies, or predefined abilities, as outcomes of
the curriculum. Competency is defined as the
observable ability of any professional, integrating
multiple components such as knowledge, skills, values
1
and attitudes. Alignment of competencies is essential for maintaining the high quality of interprofessional education and training in medicines development and clinical research.2
Training in integrated medicines development, based upon relevant competencies, is a major mission
of the Innovative Medicines Initiative (IMI) of the European Union. This is Europe’s largest public–
private initiative, and aims to speed up the development of better and safer medicines for patients.3
The Faculty is a public partner within the PharmaTrain project, which is supported by the Innovative
Medicines Initiative Joint Undertaking (IMI JU) and the European Federation of Pharmaceutical
Industries and Associations (EFPIA). This consortium has developed a syllabus for pharmaceutical
sciences that is the basis for new European programmes dealing with integrated drug development.4
PharmaTrain commenced its activities in 2009 as an education and training project funded by IMI and
has implemented reliable standards for high-quality postgraduate education and training in medicines
development. This will ensure the continued competence of medicines development scientists and
clinical investigators. Learning outcomes are embedded in the PharmaTrain assessment process of
training programmes and PharmaTrain Centre of Excellence recognition. The PharmaTrain Federation
is the successor organisation to PharmaTrain and is now in the process of developing PharmaTrain
Specialist in Medicines Development Awards, along with PharmaTrain “à la carte” training courses that
are tailored to more individual needs. It is one of a great variety of such activities funded by IMI so that
future generations of scientists will be fully acquainted with the complexity of pharmaceutical R&D and
will promote better collaboration between the different sectors within its long and complex pipeline.
20
The IFAPP commitment to education and continuing professional development (CPD) in
Pharmaceutical Medicine
The International Federation of Associations of Pharmaceutical Physicians and
Pharmaceutical Medicine (IFAPP) is a global federation, strongly devoted – since 2001
– to education in Pharmaceutical Medicine around the world.5,6 In 2013, a working
group formed within IFAPP, including members of the Faculty’s International
Committee, PharmaTrain, academic institutions, and national member associations,
defined a set of core competencies for pharmaceutical physicians and drug
development scientists.7 Seven domains and fifty seven competencies in medicines
development were identified and mapped against the PharmaTrain Learning
Outcomes. A set of core competencies is summarised in the “Statement of Competence”:
The pharmaceutical physician and drug development scientist is able to:
-
Identify unmet therapeutic needs, evaluate the evidence for a new candidate for clinical
development and design a Clinical Development Plan for a Target Product Profile.
Design, execute and evaluate exploratory and confirmatory clinical trials and prepare manuscripts
or reports for publication and regulatory submissions.
Interpret effectively the regulatory requirements for the clinical development of a new drug
through the product life-cycle to ensure its appropriate therapeutic use and proper risk
management.
Evaluate the choice, application and analysis of post-authorization surveillance methods to meet
the requirements of national/international agencies for proper information and risk minimization
to patients and clinical trial subjects.
Combine the principles of clinical research and business ethics for the conduct of clinical trials and
commercial operations within the organisation.
Appraise the pharmaceutical business activities in the healthcare environment to ensure that they
remain appropriate, ethical and legal to keep the welfare of patients and subjects at the forefront
of decision making in the promotion of medicines and design of clinical trials.
Interpret the principles and practices of people management and leadership, using effective
communication techniques and interpersonal skills to influence key stakeholders and achieve the
scientific and business objectives.
The LifeTrain Common Framework for CPD in the biomedical sciences
LifeTrain is a new pan-European framework for continuing professional development (CPD) in the
biomedical sciences.8 The framework consists of a series of key messages and four sets of agreed
principles – one each for professional/scientific bodies, course providers, employers, and individual
professionals. The LifeTrain framework focuses on competency profiles, competence assessments and
competency portfolios.9,10 The European Medicines Research Training Network (EMTRAIN) – one of
the five education and training projects supported by the IMI – conceptualised the LifeTrain framework
and developed the course catalogue “on-course®” containing around 7000 post-graduate courses in
biomedical sciences (Master’s, short courses and PhD programmes) and incorporating quality standards
developed by the IMI education and training projects.11-13
Competency and Pharmaceutical Medicine specialty recognition in Europe
Whilst the special and differing competencies of Pharmaceutical Medicine are widely recognised, so far
only a few European countries have recognised it as a postgraduate medical specialty with board
certification in its own right, namely the UK, Ireland and Switzerland.14 In Belgium, specialists whose
names appear on the Register of the Belgian College of Pharmaceutical Medicine are entitled to bear
the title of “Physician Specialist in Pharmaceutical Medicine”, and negotiations for recognition by the
Higher Council of Specialists are ongoing.
21
Several qualified institutions offer courses that are usually part of an individual’s educational path for
those who seek licensure in this specialty. Detailed information on biomedical postgraduate education
and training programmes, including short courses (CPD), masters and PhD programmes, can be
consulted on the “on-course®” course portal www.on-course.eu
History and current state of the art: Examples of Belgium, Switzerland, and Poland
Belgium
Belgium is an example of a country with advanced recognition and
development of the discipline of pharmaceutical medicine. The
Belgian Association of Pharmaceutical Physicians (BeAPP) was
established in 1972. All members are pharmaceutical medicine
professionals working in or for the pharmaceutical industry and a
majority are fellows or candidate fellows of the Belgian College of
Pharmaceutical Medicine (BCPM).15 BeAPP became a member of
IFAPP at its foundation in 1975 and has been permanently
represented on its Executive Committee since then.
The association is opening up to non-MDs active in pharmaceutical
medicine, this mainly in response to increased requests of e.g. Medical Science Liaison (MSL) and other
non-MD professionals who feel attracted to the domains and topics BeAPP is covering.
The Pharmed post-graduate programme in Pharmaceutical Medicine and Medicines Development
Sciences is recognised by the Faculty of Pharmaceutical Medicine as one of few European courses that
provide appropriate postgraduate training in pharmaceutical medicine.16 The Pharmed programme
follows closely the Faculty’s syllabus of pharmaceutical medicine. Since 2003, the Pharmed course is
also accredited by the IFAPP Council of Education in Pharmaceutical Medicine (CEPM). Pharmed
successfully passed a detailed assessment of full compliance with the PharmaTrain quality standards,
and as a result received the PharmaTrain Centre of Excellence award in November 2011.
Efforts to have pharmaceutical medicine recognised by the Higher Council of Specialists are ongoing.
The FPM description of the certification routes in the UK serves as a reference the dialogue with our
local health authorities.17 The curriculum requirements currently being discussed are to a great extent
guided by the Pharmaceutical Medicine Specialty Training (PMST) programme recommendations of the
Faculty.
Switzerland
Switzerland is a country in which the pharmaceutical industry
plays a major and longstanding role in the country’s
manufacturing business. In 2014, more than one-third of all
Swiss exports were pharmaceuticals, and pharmaceutical
companies headquartered in Switzerland reached a worldwide
market share of $95 billion.18 In terms of employment, the
pharmaceutical sector offers 41,900 jobs directly in
pharmaceutical companies, and another 130,000 jobs in ancillary industries. Therefore, it may not come
as a surprise that this country provides adequate professional organisations in which both physicians
and non-physicians can express their interest in the profession, receive training, and contribute to its
advancement. Pharmaceutical medicine is also one of 44 accredited medical specialties, with
registration by the Swiss Medical Association (FMH, Fédération Médicale Helvétique.19 It is possible to
transfer a GMC licence in Pharmaceutical Medicine to the FMH, which is a bureaucratic, but feasible,
act; however, proficiency in German is mandatory.
22
The two professional organisations dealing with pharmaceutical medicine are the Swiss Society of
Pharmaceutical Medicine (SGPM) and the Swiss Association of Pharmaceutical Professionals (SwAPP).
The former was founded in 1997 for physicians in Switzerland with, or in training for, specialisation in
Pharmaceutical Medicine. The SGPM is member of the Umbrella organisation SSPT (Swiss Society of
Pharmacology and Toxicology). Today the SGPM counts about 150 members, of whom more than 100
are holders of the Swiss specialist title in Pharmaceutical Medicine. The remainder either are in
postgraduate training or hold an equivalent certificate.20
SwAPP was founded in 1995 as the professional association for qualified specialists working in one of
the many fields of pharmaceutical medicine and drug development. SwAPP members come from
pharmaceutical companies, contract research organisations, health care institutions, biotech
companies, health authorities and ethics committees. SwAPP welcomes members with an academic
background, for example in life sciences, biology, pharmaceutics or a similar field, and physicians, as
well as non-academic health professionals.21 Both organisations entertain informal friendly contacts
with each other and co-organize an annual symposium.
Poland
Poland is one of the most prolific countries for industry sponsored clinical research. With 400-500
clinical trial registrations annually, Poland remains one of the most important countries for the conduct
of clinical research in Europe and the largest in Central Eastern Europe.22 Despite this, few
pharmaceutical companies located their R&D operational centres in Poland. Building of pharmaceutical
medicine competencies is mainly focused on investigator training, often limited to Good Clinical
Practice.
Although medical doctors are involved in regulatory, medical, commercial affairs, project management
and pharmacovigilance roles, there is no widely recognized requirement for formalized building of
pharmaceutical medicine competency among physicians in Poland. The competency level is growing
thanks to growing individual experience to some extent supported by local subsidiaries of international
pharma corporations and contract research organisations. Notable educational initiatives in the region,
such as the Cooperative European Medicines Development Course (CEMDC), are often hindered by
funding issues originating from lack of local medical community and industry support.23 A good number
of physicians trained in Poland have taken on pharmaceutical roles in the UK and elsewhere in Western
Europe. GCPpl (www.gcppl.org.pl) is the association for clinical research professionals.
Though there is a lack of census or survey data, we estimate the number of pharmaceutical physicians
in Poland to easily exceed 100. Their role is undermined by lack of any form of formal recognition of
their competency. Despite some early efforts by individuals affiliated with the FPM, there exists a
widespread misunderstanding of the role of pharmaceutical physicians working in the industry, most
often confused with clinical pharmacology or commercial operations.24, 25
Pharmaceutical physicians are denied a licence to practise medicine in Poland by medical governing
bodies (Medical Chambers), which not only undermines their role but also pushes them outside the
corporate regulations and codes of practice such as Good Pharmaceutical Medical Practice or formal
recognition of the need for continued professional education and revalidation. The current situation
calls for active contribution of those engaged in the mission of the FPM.
References
1.
2.
3.
Frank JR, Snell LS, Cate OT et al. Competency-based medical education: theory to practice. Med Teach 2010;
32: 638–645
Silva H, Sonstein S, Stonier P et al. Alignment of Competencies to Address Inefficiencies in Medicines
Development and Clinical Research: Need for Inter-Professional Education. Pharm Med 2015; 29:131–140
Goldman M. The Innovative Medicines Initiative: A European response to the innovation challenge. Clin.
Pharmacol.Ther 2012; 91(3): 418–425
23
4.
5.
6.
7.
8.
9.
10.
11.
12.
13.
14.
15.
16.
17.
18.
19.
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