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InsideHealthPolicy.com’s
FDA Week
an exclusive weekly report on Food and Drug Administration policy, regulation and enforcement
Vol. 20, No. 51 — December 19, 2014
Industry Looks At 21st Century Cures To Set Stage For PDUFA VI
Representatives from the pharmaceutical and biotechnology industries said last Friday (Dec. 12) they expect work
on the bipartisan 21st Century Cures initiative this spring to be a staging ground for negotiations over the sixth iteration
of the Prescription Drug User Fee Act (PDUFA) in 2016 and 2017. Industry leaders also said they expected the biotechnology industry to enter the negotiations with FDA as an equal partner with the pharmaceutical industry, speaking on a
PDUFA VI panel at an IIR-sponsored FDA/CMS Summit in Washington.
Brian Harvey, vice president of U.S. Regulatory Strategy for Pfizer, echoed the sentiments of other panelists saying
continued on page 4
Senate Bill Sets Up New ‘Dormant Drug’ Class Eligible For 15 Years Exclusivity
Sens. Orrin Hatch (R-UT) and Michael Bennet (D-CO) have introduced legislation that picks up on a controversial
House proposal to offer 15 years of data protection as an incentive for drug makers to develop therapies that treat one or
more unmet medical needs. Brand drug makers back the idea and have urged its inclusion in the House Energy and
Commerce Committee’s upcoming 21st Century Cures bill, but some House Democrats, generic drug makers, AARP and
pharmacy benefit managers charge the patent idea would merely extend market monopolies of high-priced drugs like
Sovaldi.
continued on page 6
Drug Quality, Generic Drug Super Offices Are CDER ‘Front Burner’ Priorities
FDA drug officials said moving forward with an internal reorganization of the Center for Drug Evaluation and
Research is a priority for the drug center heading into 2015. This includes the formal standup of the Office of Generic
Drugs and a new Office of Pharmaceutical Quality as “super offices” that are expected to help the center move through
generic drug applications and open up communication lines between FDA and generic drug companies. However, the
center is concurrently grappling with 600 staff vacancies.
FDA drug chief Janet Woodcock told stakeholders her priorities include the center’s reorganization, implementing
continued on page 8
Pew: UDI Integration Into Healthcare System Should Start With EHRs
A renewed push to implement Unique Device Identifiers (UDI) into patients’ electronic health records came last
week from Pew Charitable Trusts and several other stakeholders at a conference centered around UDIs. The rekindled
fervor comes on the heels of a comprehensive report sponsored by FDA and conducted by The Brookings Institution on
UDIs that recommends the Office of the National Coordinator (ONC) for Health Information Technology propose a
field for UDIs as part of certification criteria when CMS issues its proposed Stage III Meaningful Use objectives for
EHRs.
continued on page 9
FDA Accepts Third Biosimilar Application As Stakeholders Wait For Guidance
Canadian pharmaceutical company Apotex announced Wednesday (Dec. 17) that FDA has accepted the company’s
application for pegfiligrastim — a biosimilar version of Amgen’s Neulasta. This is the third biosimilar company to
declare that FDA has accepted its application since Sandoz announced the agency’s acceptance of its biosimilar
filigrastim application in July. With the third filing of a biosimilar for market in the United States, stakeholders are still
waiting for FDA guidance on naming, interchangeability and labeling that the agency has indicated may be coming soon
as the clock ticks on the 10-month application review deadline established by the Biosimilar User Fee Act and the 2012
continued on next page
FDA Safety and Innovation Act.
Pegfiligrastim is the long-acting form of filigrastim — marketed by Amgen as Neupogen — which boosts white
blood cell counts for cancer patients to help them fight off infections and fever, according to a press release issued by
Apotex.
“We are very pleased to be at the forefront of companies who will introduce high quality biosimilar products into the
US marketplace,” Apotex President and CEO Jeremy Desai said. “The benefits for patients, payers and providers from
biosimilars will be significant. We are dedicated to playing a leading role in the effort to increase the American public’s
access to more affordable versions of these life-saving therapies and generate substantial savings for the US health care
system.”
An FDA official said earlier this month the agency is working to get out interchangeability and labeling guidance in
the near term and Inside Health Policy has reported that the naming guidance has made its way to HHS Secretary Sylvia
Burwell for final approval.
With the application process underway Apotex will enter into what is known as the “patent dance” with Amgen.
Under this process Apotex must provide Amgen with a copy of the application and a description of the manufacturing
process within 20 days of the application’s acceptance. Then, within 60 days, Amgen must give Apotex a list of patents
the company thinks would be infringed and which of those the company would be willing to license.
In the first test case of the “patent dance” process, Amgen filed suit against Sandoz in October charging the
biosimilar applicant violated the law’s patent exchange process by not sharing its manufacturing information, and asking
a federal court to prevent Sandoz from seeking FDA approval, among other legal requests.
Amgen also filed a citizen petition with FDA in early November asking the agency to create a certification process to
ensure that companies filing a biosimilar application commit to following statutorily outlined procedures to share
application and manufacturing information. Amgen said a new administrative mechanism would prevent companies from
circumventing disclosure requirements that the company asserts are mandatory.
IHP attempted to ask Apotex how it planned to go forward with the “patent dance” process for its biosimilar application for pegfiligrastim in light of Amgen’s lawsuit against Sandoz, but an Apotex representative said at this point the
company cannot publicly share any information other than what was included in its press release.
FDA’s Oncologic Drug Advisory Committee is scheduled to hold a public hearing on Sandoz’s filigrastim
application Jan. 7, and the agency is expected to finish the application review in March.
In August Celltrion was the second company to announce that FDA had accepted its biosimilar application. Celltrion
biosimilar infliximab is a copy of Jansen Biotech’s Remicade. Celltrion already markets its infliximab biosimilar under
the brand name Remsima in more than 50 countries. — Todd Allen Wilson
Patty Murray Tapped As Ranking Member Of Senate HELP Committee
Sen. Patty Murray (D-WA) will become ranking member of the Senate health committee as the upper chamber
transitions to Republican control in the next Congress, confirming expectations that she would take the spot. As
chair of the powerful Budget Committee, Murray has aggressively advocated for FDA’s funding and food safety
laws.
“Senator Murray is committed to looking for innovative ways to expand access to life-saving treatments and cures,
while maintaining high scientific standards to ensure safety and consumer protection,” an aide said, responding to an FDA
Week inquiry about Murray’s FDA priorities. “She will also continue to push for investments in biomedical research to
improve health and well-being for families in Washington state and across the country.”
“I plan to work with incoming-Chairman Alexander and my colleagues on both sides of the aisle to make sure that
our country is doing everything possible to... improve health care access and affordability... for all workers and all
families,” said Murray in a statement.
Industry groups in the past have applauded Murray’s support on issues relating to biotechnology and the reauthorization of the Prescription Drug User Fee Act in 2012 (PDUFA). The Pharmaceutical Research and Manufacturers of
America also has praised Murray for her support in permanently reauthorizing the Best Pharmaceuticals for Children Act
and the Pediatric Research Equity Act. Generally, the association said it was looking forward “to working with Sen.
Murray in her new capacity.” The Advanced Medical Technology Association and the Biotechnology Industry Organization expressed the same sentiment in similar statements — though they declined to specify on what issues they are
looking forward to working with her on.
In 2006, Murray and then-Sen. Hillary Clinton stalled both of President George W. Bush’s nominees for FDA
commissioner because of rules regarding over-the-counter contraception. Clinton and Murray double-teamed Bush’s
nominees until they were satisfied that contraception could be available to consumers 17 and older without a
prescription. — David Hood
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Drug Distributors Seek Extra Time To Meet Drug Tracing Requirements
A drug distributors group is asking FDA for at least an extra three months to meet the drug tracing law’s new data
requirements, warning in a Dec. 15 letter to Commissioner Margaret Hamburg that some distributors won’t be ready by
the Jan. 1 deadline and supply chains could be disrupted without enforcement relief. The group asks for a written statement saying the agency will temporarily offer enforcement flexibility, and asks FDA to urge states to also exercise
restraint until the supply chain can fully comply.
In the event that FDA doesn’t issue a written statement, the group asks the agency to consider alternatives such as
offering a temporary exemption from the data transmission and receipt requirements or providing direction to supply
chain partners who receive product without the appropriate transaction data.
A Healthcare Distribution Management Association official made a similar plea at a Food and Drug Law Institute
conference earlier this month. HDMA Executive Vice President Perry Fri said at the time, “There are some smaller
companies on the supplier side, and definitely a lot on the dispensing side, that don’t have a good sense of what the
impact on their business will be. We have questions about what happens if these companies are not prepared.” (see FDA
Week, Dec. 5).
The letter notes that the Jan. 1 compliance deadline is significant because the Drug Supply Chain Security Act does
not allow a supply chain partner to take ownership of or subsequently sell the associated product unless there is successful transmission and receipt of completion transaction data.
“Consequently, based on current information about supply chain readiness, there is very real potential for some
product transactions to lack appropriate data, contain errors in data or resort to paper-based systems — all of which could
result in disruptions to the provision of prescription drug products to patients who need them,” HDMA writes in its Dec.
15 letter to Hamburg.
HDMA adds that compliance with the drug tracing requirements involves development of complex information
technology processes, as well as operations and logistics changes. Factors that may cause disruptions to product flow, the
group writes, include: substantial variability in technical expertise from trading partner to trading partner; complexities
related to the IT systems that are attempting to connect with one another; and throughout the supply chain, an uneven and
incomplete understanding of the DSCSA and its requirements.
The group says that although most of its members may be ready by Jan. 1, some may not.
“As of today, we believe that the majority of primary distributors and their supply chain partners will be ready to
exchange the required data, for the majority of transactions, by the January 1 implementation deadline,” HDMA writes.
“However, despite extensive and productive efforts to establish methods for data exchange, it is anticipated that some
trading partners will encounter challenges beyond their control, which may result in disruptions in the supply
chain.” —Erin Durkin
AHIP: ACA’s Cost-Cutting Goals Unachievable If PhRMA Has ‘Blank Check’
America’s Health Insurance Plans CEO Karen Ignagni last Friday (Dec. 12) made another plea for drug makers to
work with payers and providers to make pricing of specialty drugs more competitive and transparent, saying that absent
such a move changes in the health care landscape designed to increase quality and access while lowering costs may not be
sustainable in the long term. The Affordable Care Act has created a retail health care market in which consumers are
focused on affordable, high-quality care, and government-sanctioned monopolies for innovative drugs create a stumbling
block, she said at an IIR-sponsored FDA/CMS Summit in Washington.
“The recognition of the problem is the idea that we can no longer continue a system where one sector expects
a blank check,” Ignagni said. “That’s the way the system has been organized, and that doesn’t work if the goal is
affordability.”
Ignagni said that while specialty drugs make up only 1 percent of plans’ medication purchases they account for 25
percent of spending. She said plans have been working with providers, consumers and state and federal agencies in order
to spur competition, but competition is lacking in the specialty drug market.
While there are a number of models that are helping plans control costs such as medical homes, Accountable Care
Organizations and bundling, the high price of specialty drugs is a factor that can raise premium prices for consumers.
While she didn’t specifically mention any high-priced specialty drugs, her comments come as the debate rages over
the high cost of new hepatitis c cures Sovaldi and Harvoni, which combines Sovaldi (sofosbuvir) and ledipsavi. Sovaldi,
has been priced by maker Gilead Sciences at $84,000 for a 12-week course, and the price tag of Harvoni, comes in at
$94,500.
Ignagni noted that state Medicaid directors have been vocal in their belief that the prices for these much-needed,
innovative drugs are unsustainable. The National Association of Medicaid Directors wrote Congress in October asking
for the federal government to either control the price of specialty drugs or help states pay for them.
She said she doesn’t want to stifle much-needed innovation, and understands that in asking drug makers to come on
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board with controlling costs means a hit to their bottom lines.
“Because my cost containment is someone else’s revenue reduction,” Ignagni said.
The federal government and states can help she said by implementing policies that remove barriers to competition,
such as the FDA encouraging the development of biosimilars and helping get those product get to the marketplace
quickly, she said.
One way drug manufacturers can help, Ignagni said, is by increasing transparency with regard to how much
it costs to develop a specialty drug.
“Now we have to turn to having a conversation about innovation, having a conversation about value,” Ignagni said.
“How do we know what is being spent on R&D (research and development) — how do we know that we are subsidizing
R&D versus marketing, advertising and other kinds of costs? I think we’ve gotten to the point that to sustain a private
market, just as now in the health plan community and hospital community, we need transparency here.”
Ignagni said she understands that bringing demand and supply sides of the equation together to find a way to spur
competition to control costs while also fostering innovation is difficult, but she said early efforts to have these conversations with drug makers have been promising.
“With out a doubt, moving to a retail market is a change, but I think that with change comes opportunity and certainly
challenges,” Ignagni said. “I’m excited about the opportunities for partnership, and the conversations to talk about
transparency; to talk about value; to talk about preserving our private sector system; and to talk about doing it
together.” — Todd Allen Wilson
CURES May Influence PDUFA VI . . . begins on page one
that 21st Century Cures will be a “dry run” for PDUFA.
“What better way to really sort of formulate your ideas and vet them to make sure they really hold water than
to have to do it that much earlier,” Harvey said. “I’m very optimistic that something may come out of 21st Century
Cures, but even for those things that don’t make it through the process we’re that much further along for PDUFA VI.”
The 21st Century Cures initiative is a bipartisan effort spurred by House Energy and Commerce Chair Fred Upton (RMI) and Ranking Minority Member Diana DeGette (D-CO) that is expected to include a broad spectrum of provisions
aimed at spurring development of new drugs and devices, and may bump up research funding at the National Institutes of
Health. Upton said last month he expects to have a draft of the 21st Century Cures bill ready in January, with hopes of
getting a full bill to the House floor by Memorial Day. Janet Woodcock, director of FDA’s Center for Drug Evaluation
and Research (CDER), said at an earlier panel during the FDA/CMS Summit that the 21st Century Cures initiative is a
“front burner” priority for the agency.
PDUFA was first passed in 1992 and updated in 1997, 2002, 2007 and 2012. The next version — PDUFA VI —
needs to be passed by Sept. 30, 2017, when the latest version is set to expire.
Harvey said PDUFA VI needs to include a focus on modernizing the way clinical trials are done, adding that
clinical trials are becoming unsustainable as the cost for a single trial is rising above $1 billion.
“We really need to use new technology,” Harvey said. “We need to use 21st century regulations and not 18th or 19th
century perspectives to run clinical trials and do it in a more efficient way.”
Kay Holcombe, senior vice president for Science Policy at the Biotechnology Industry Organization (BIO), said the
21st Century Cures legislation will also help determine what resources FDA needs going forward that can be built into
PDUFA. She said FDA gets “saddled” with a multitude of requirements in PDUFA updates, but finds that user fees don’t
cover the costs of meeting those requirements. The 21st Century Cures bill is expected to impose new requirements on the
agency, so FDA will know what it needs to fulfill those requirements as PDUFA VI is negotiated, Holcombe said.
“It gives us a good base of information as we’re going into PDUFA VI in terms of the resources the agency is going
to have to come up with in order to do things out of 21st Century Cures,” Holcombe said.
When asked whether industry should be paying more or all of the costs for the drug approval process — user fees
account for roughly 70 percent of the costs currently — Holcombe said it is highly dependent on what the benefits for
drug makers are.
“I don’t want to sound crass, but when you put money on the table you’re kind of wondering what it is that you’re
getting back,” Holcombe said.
She said industry may be supportive of paying a higher share if that leads to reducing the time and cost
associated with developing and producing new drugs.
Holcombe also said that the “industry will speak with one voice” during PDUFA negotiations with BIO on equal
footing with the pharmaceutical industry — noting that the two groups have already been talking and working with each
other in this regard.
“BIO very wants to be, obviously, not taking a back seat to PhRMA, but working with PhRMA as an equal partner,”
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FDA Week - www.InsideHealthPolicy.com - December 19, 2014
she said.
Robert Metcalf, the vice president of Global Regulatory Affairs for Eli Lilly who will be heading the Pharmaceutical
Research and Manufacturers of America’s (PhRMA) regulatory affairs arm starting next year, said PhRMA views its
relationship with BIO going forward as “very much of a joint partnership across BIO and PhRMA as equals.”
Metcalf and patients’ advocates on the panel also said PDUFA VI will build on PDUFA V’s efforts to open up the
drug development and regulatory process to patient’s concerns.
Jeff Allen, executive director of patient advocacy group Friends of Cancer Research, said PDUFA V built the
framework for including patients in the conversation and has created an opportunity for drug makers to build those
concerns into their trials.
“It has to start earlier than it historically has in terms of understanding what patient outcomes are desired and
incorporate those into the experimental process,” Allen said.
Metcalf said PDUFA VI will be an opportunity to began incorporating patient input into the development
process in a scientific and systematic way.
“Patient input can and should be not only a motive, but also scientific as well,” Metcalf said. “That’s the opportunity
I see going forward. We’ve got to bring in skills and capabilities that we haven’t necessarily done, like social science
capabilities.” — Todd Allen Wilson
Small Farmers: FDA’s Reproposed Food Rules Still Violate Congress’ Intent
More than 12,000 farmers, businesses and individuals recently wrote to FDA that its reproposed preventive controls
and produce safety rules go against congressional intent and still don’t provide due process in the event a stakeholder’s
regulatory exemption is revoked.
“Despite the many groups and individuals who urged FDA to implement the Tester-Hagan provision fairly and in a
way that fulfills the intent of the law, the agency’s new proposed rules still do not do so,” states the letter, spearheaded by
the Farm and Ranch Freedom Alliance (FARFA).
At issue are FDA’s policies surrounding the Tester-Hagan provisions of the rules that lay out a process for FDA to
exempt small-scale, direct-marketing farmers and facilities from some of the requirements under the Food Safety Modernization Act’s produce safety and preventive controls rules. The groups say FDA has laid out overly broad criteria for
when the agency will revoke such exemptions, including a wide range of non-urgent situations, and doesn’t offer due
process to stakeholders involved.
“The FDA can revoke a producer’s exemption even in the absence of any immediate threat to public health,” the
groups write. “Indeed, the FDA may revoke the exemption simply if it determines it is necessary to ‘protect the
public health and prevent or mitigate a foodborne illness outbreak based on conduct or conditions associated’ with
the farm.”
The groups complain that FDA imposes hasty deadlines for compliance that will shut down any producer whose
exemption is revoked; and still bases the size requirements for qualifying small-scale produces on all the food sold. “Yet
the FDA is directed to regulate only certain foods under FSMA, and that the same scope should be applied to the exemption,” the groups say.
They also say that FDA has added a new provision in the proposed Hazard Analysis and Risk-Based Preventative
Controls (HARPC) rule that “could undermine the Tester-Hagan exemption by requiring that facilities conduct a ‘supplier
program.’” They say the supplier program will not only impose significant costs on the businesses, but could also
“indirectly impose requirements on, and create barriers for, those farmers and producers who are supposed to be exempt
from the HARPC rule under the Tester-Hagan exemption.”
The stakeholders urge the agency to do the following to ensure appropriate due process and reliance on
standard procedures:
Include a specific statement of the reasons in the notice of revocation so the produce can respond.
Set standards for what FDA must find in order to revoke the exemption.
Provide at least 90 days for producers to submit the facts and documentation showing that their exemption
should not be withdrawn.
Guarantee a hearing so that producers can present their case in person before having their exemption revoked.
Provide the standard post-decision procedural protections, such as motion for reconsideration and a motion for
stay.
FDA revised the produce safety rule, the preventive controls for human and animal food rules, and the foreign
supplier verification program rule in September to address concerns and pushback from industry and other stakeholders.
“FDA has the chance to show that it really does pay attention to what the public says, by making changes before the
final rule,” said Judith McGeary, a founder and executive director of the Farm and Ranch Freedom Alliance. “If not, then
we will look to our elected officials to remind the agency that it has to respect the will of Congress.”— Erin Durkin
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Bill Adds 15 Years Exclusivity . . . begins on page one
The newly introduced Dormant Therapies Act would offer 15 years data exclusivity for a new class of drugs called
“dormant therapies.” To be eligible, a medication would have to be intended for one or more unmet medical needs, have a
suitable clinical trial plan developed by the sponsor, and at the time of request contain an active moiety that is not the
same as, or highly similar to, an active moiety for another application.
A dormant drug’s patent would be extended for 15 years once FDA accepts the application, mirroring the approach in
a bipartisan House bill, Modernizing Our Drug and Diagnostics Evaluation and Regulatory Network (MODDERN) Act,
spearheaded by Rep. Leonard Lance (R-NJ). At a hearing this summer, Energy and Commerce health subcommittee chair
Joe Pitts (R-PA) suggested it was time to rethink long-standing drug patent law, and the National Health Council, whose
members include the innovator pharmaceutical and biologics lobby groups, touted the bipartisan bill as a way to encourage repurposing of drugs that stalled in early clinical trials. But some Democratic lawmakers suggested lawmakers
instead turn to other incentives, such as tax credits and public funding for basic research (see FDA Week, June 19).
The 15 years data exclusivity included in the House and Senate bills aims to make up for patent time lost
during drug development.
“It takes on average 14 years for a compound to make its way through the therapeutic pipeline from discovery,
through clinical trials, to formal approval, and eventually to the patient,” says a press release from Hatch’s office.
“Because patents last for only 20 years, much of this time is consumed during the lengthy research and development
process. The result is companies are investing in research on compounds that can be brought to market quickly, rather
than new treatments that could serve people with the most complex medical needs.”
In order to receive the designation, sponsors must list all patents and application for patents that may provide
protection for the medicine, a waiver of patent rights to enforce any patent described in that list which may expire after
the end of the protection period, and any additional information required in order to determine eligibility for designation.
Hatch envisions the bill as spurring treatments for such conditions as Alpha-1, ALS, Alzheimer’s, epilepsy, lupus,
mesothelioma and multiple sclerosis, the release says.
“I’m pleased Sen. Bennet has joined me in this important effort to encourage the development of desperately needed
treatments for some of the most troubling diseases and disabilities out there,” said Hatch. “We hope to create a timecertain protection to encourage innovators to capture lost opportunities and bring new and essential products to market
for the patients who need them.”
Brand drug makers and some patient groups floated similar ideas in comments sent to the House Energy and Commerce Committee for its 21st Century Cures initiative. But generic drug makers, AARP and Rep. Henry Waxman (D-CA)
urged Congress to proceed with caution.
“Virtually any drug with a novel active ingredient could qualify for this reward,” Waxman said. “Well, that’s giving
away too much. That’s going to upset the ability of making drugs affordable and is far more of an incentive that I think is
justified.”
Pitts recently said company incentives could be included in the upcoming 21st Century Cures bill, a draft of which the
panel hopes to unveil in January. Drug makers have sought added exclusivity for a range of drugs, including for small
molecule drugs and first-in-class treatments (see FDA Week, Oct. 27). — Erin Durkin
IACP: Compounding Advisory Committee Short On Practicing Pharmacists
FDA on Tuesday announced the 14 members of a new Pharmacy Compounding Advisory Committee formed to
advise the agency as it beefs up its compounding oversight, prompting quick concerns from the international compounding lobby that the panel lacks adequate representation of actual practicing pharmacists. Two groups that have pushed for
stronger FDA compounding oversight — Public Citizen and Pew Charitable Trusts — have seats on the committee, which
was called for by the Drug Quality and Security Act. The National Institutes of Health also has representation.
The DQSA requires FDA to consult with the advisory committee prior to releasing a list that identifies drugs or
categories of drugs that present demonstrable difficulties for compounding and that are reasonably likely to lead to an
adverse effect on safety and effectiveness.
Compounders are anxiously awaiting to see what drugs will be targeted, and the list was the subject of a
Government Accountability Office report earlier this year.
“[Defense Health Agency] officials told us that they are considering denying payment for certain compounded drug
prescriptions that include bulk drug substances, and are waiting to review the list of bulk drug substances that may be
used to make compounded drugs — and the lists of drugs that may not be compounded because they present demonstrable difficulties for compounding — that FDA is required to develop under the DQSA,” according to the GAO report
(see FDA Week, Oct. 10).
The formation of the new advisory panel comes as FDA gears up to make DQSA implementation a key issue next
year. The agency’s drug chief recently pegged compounding oversight a “front burner” priority for the agency. The new
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panel’s charter calls for it to advise FDA on scientific, technical and medical issues concerning pharmaceutical compounding.
“Advisory committees are a very important source of knowledge and advice for drug regulation,” said Janet Woodcock, director of FDA’s Center for Drug Evaluation and Research. “This is a key step toward implementing the compounding provisions of the Drug Quality and Security Act, and I expect we will benefit greatly from the advice and
recommendations the members of the committee provide.”
The law states the committee shall include representatives from the National Association of Boards of Pharmacy, the
United States Pharmacopeia, pharmacists with current experience and expertise in compounding, physicians, and patient
organizations.
The International Academy of Compounding Pharmacists complained that only one of its nominees was
chosen.
“While we are pleased that the advisory committee finally has been announced and that the members are wellqualified scientifically, the IACP has serious concerns about the lack of representation on the panel of actual
practicing compounding pharmacists. The IACP submitted 24 nominees, many with extensive real-life compounding
practice experience, and only one who is a practicing compounding pharmacist was selected by the FDA,” the group
said.
The announcement comes after IACP sent a letter to FDA in September complaining that the agency had developed
criteria for the upcoming compounding list without compounders’ input.
“[T]he agency has developed its criteria list without consultation or input from Pharmacy Compounding Advisory
Committee,” the group states. “Congress created this Advisory Committee in the original and reaffirmed language of
section 503A to assure that experts in the pharmacy and medical community would have practitioner input into the
implementation of the agency’s activities surrounding compounding.”
In the introduction of the guidance, which was released in July, FDA states, “Several parts of section 503A require
rulemaking and consultation with a Pharmacy Compounding Advisory Committee to implement.”
Members of the committee include:
- Jurgen Venitz, associate professor at the Virginia Commonwealth University School of Pharmacy, as chairperson.
- Jayne Peterson from the FDA drug center’s Office of Executive Programs as acting federal officer.
- Michael Carome, Public Citizen’s Health Research Group director.
- Ned Braunstein, vice president and head of regulatory affairs at Regeneron Pharmaceuticals.
- Gigi Davidson, director of Clinical Pharmacy Services at North Carolina State University’s College of
Veterinary Medicine.
- Robert DeChristoforo, chief of the Clinical Center Pharmacy Department at the National Institutes of Health.
- John DiGiovanna, staff clinician at the Center for Cancer Research at the National Cancer Institute, NIH.
- Padma Gulur, professor at University of California, Irvine.
- Stephen Hoag, professor at University of Maryland School of Pharmacy.
- William Humphrey, director of pharmacy operations at St. Jude Children’s Research Hospital.
- Elizabeth Jungman, director of Pew Charitable Trusts’ work on public health, including initiatives related to
antibiotics and innovation, drug safety, and prescription drug abuse.
- William Mixon, owner and manager of The Compounding Pharmacy.
- Katherine Pham, Neonatal Intensive Care Unit Pharmacy specialist at the Children’s National Medical Center.
- Allen Vaida, executive vice president at the Institute for Safe Medication Practices.
- Donna Wall, clinical pharmacist at Indiana University Hospital.
The committee consists of a core of 12 voting members including the chair and two non-voting members.
Public Citizen’s Carome vowed to use his position on the committee to push for aggressive FDA oversight. “As
the consumer representative on the FDA’s Pharmacy Compounding Advisory Committee, I intend to aggressively advocate for rigorous FDA safety standards for all compounded medications to protect patients from serious harm,” said
Carome. “Implementation of such standards is essential for preventing another tragedy like the fungal meningitis outbreak
linked to injectable medications manufactured by the New England Compounding Center that sickened more than 750
patients around the country, killing 64.”
Carome, since joining Public Citizen in 2011, has worked extensively to boost the safety of pharmacy compounding
by pushing Congress, FDA and other government agencies to step up their regulatory efforts.
Pew was also a key player as lawmakers drafted the new compounding law. On Nov. 20, Pew — joined by the
American Public Health Association, Biotechnology Industry Organization, Generic Pharmaceutical Association,
Pharmaceutical Research and Manufacturers of America, and Trust for America’s Health — wrote to FDA in
support of its enforcement efforts. The letter urged the agency not to be swayed by compounders’ calls for enforcement flexibility. — Erin Durkin
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CDER Focuses On Super Offices . . . begins on page one
the compounding law, generic drug user fee goals and working with the House lawmakers on their 21st Century Cures
initiative.
CDER also is in the process of improving staffing levels as it faces more than 600 staff vacancies, she said at a recent
FDA/CMS Summit sponsored by IIR. “We are struggling to run the HR process,” said Woodcock, adding it takes a lot of
time and expense to recruit people.
Woodcock listed the center’s reorganization as one of her “front burner” priorities going into 2015.
“We have to further stand up the Office of Generic Drug ‘super office,’” said Woodcock. “We really planned the
reorganization carefully to support critical functions of generic drug review.” She added that through a Drug Lifecycle
Council, the office will help generic drug sponsors work through administrative problems that have been stalling their
applications.
Woodcock also noted that the new Office of Pharmaceutical Quality, slated to stand up Jan. 11, will centralize quality
review for new and generic drugs. The OPQ will combine drug quality work into one “super-office,” with a mission to
improve oversight of quality through the life cycle of a drug product. The office creates a uniform drug quality program
across all sites of manufacturing for new drugs, generic drugs and over-the-counter drugs.
Woodcock said that the office will perform “real time” interactions with generic drug sponsors to answer
easily resolved questions. Recently, the office rolled out a plan that would open phone lines and other methods of
communication to generic drug companies waiting on abbreviated new drug application (ANDA) approvals while their
applications are at the Chemistry, Manufacturing and Controls portion of the approval process.
“Real-time communication does not replace OGD’s formal communication methods, but rather enhances the review
process in an effort to increase transparency and decrease the number of review cycles,” said Susan Rosencrance, acting
director of the Office of Lifecycle Drug Products in OPQ (see FDA Week, Dec. 11).
Woodcock also detailed how the office seeks to identify the quality status of all facilities manufacturing drugs for the
U.S. market, and adds that the “Pharmaceutical Platform” IT system will support this initiative. The system already has
links to the Office of Regulatory Affairs and other CDER databases, said Woodcock.
The center’s plan to elevate the Office of Generic Drug and create the Office of Pharmaceutical Quality was approved last year. Under the restructuring, OPQ will house the product quality-related groups, including CMC and
Microbiology review functions, which had previously been in OGD. — Erin Durkin
ONC Lays Out 10-Year Vision For Health IT Improving Health Quality
Within a decade, the HHS Office of the National Coordinator for Health Information Technology (ONC) says it
will develop a health IT infrastructure for driving quality improvements in health care that will combine clinical
decision support (CDS) and clinical quality measurement (CQM) in a “continuous feedback loop” that allows
patients, physicians, public health officials and researchers to access information in real time. ONC envisions that
patients and their doctors will have secure access to the data, which will inform decisions about patient care. Deidentified, privacy-protected patient data also would be available in metadata sets for public health officials’ and
researchers’ use.
“ONC envisions an electronically enabled (quality improvement) ecosystem that promotes better health and care,
improved communication and transparency, rapid translation of knowledge for all stakeholders and reduction in the
burden of data collection and reporting for providers,” ONC says in a recently released paper, “Health IT Enabled
Quality Improvement: A Vision to Achieve Batter Health and Health Care.”
“Data created during the normal course of care can, when collected in standard formats (e.g., CDEs), be transformed
real-time into knowledge to inform clinical decisions, report on notifiable conditions or events, measure quality of care
and provide evidence for patient-centered outcomes research,” the paper adds.
ONC says its vision depends on achieving true interoperability — the office plans to release its map for
interoperability early next year — that includes common data elements (CDE). The vision is also contingent on stakeholders investing in the needed technology.
“In order to be successful in these new payment environments, providers must invest in delivery system re-design
which includes more robust leveraging of health information technology and interoperability. Public and private payers
alike must commit to this transition in order for the incentives to be large enough for providers to make these substantive
system-wide changes,” ONC says.
In the paper, ONC notes that at this point all states have some form of health information exchange services and more
than half of the nation’s hospitals can electronically access patient information outside of their own systems. On top of
this, ONC says more than half of office-based professionals and more than 80 percent of hospitals are participating in the
electronic health records Meaningful Use (MU) program — with nearly 70 percent of providers in MU submitting CQM
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FDA Week - www.InsideHealthPolicy.com - December 19, 2014
information to CMS.
The MU program, however, has been fraught with complications, with some providers having trouble getting the
latest certified technology, and stakeholders asking for more flexibility and time to attest to MU. CMS and ONC extended
the time for first time MU users to apply for hardship exemptions earlier this month, and extended the time frame for
attesting to MU stage two earlier this year. CMS and ONC have yet to release guidelines for MU stage three, but those
regulations are expected in the near future. —Todd Allen Wilson
ONC Should Create UDI Field . . . begins on page one
Through ONC, a set of guidelines called Certification Criteria directs what is in an EHR. Currently, EHRs only must
show that a patient has a medical device without any specifics as to what the device is. Without a change from ONC,
EHRs likely will not include UDI information.
“Including a new field in the EHR will have many benefits including improved care coordination amongst physicians,
being able to identify patients that are affected when there’s a recall and many other benefits,” said Ben Moscovitch, an
officer of medical devices at The Pew Charitable Trusts.
“Just having the field isn’t enough, we need additional capabilities, for example the ability to use barcode scanning to
include the UDI in the EHR, search the EHR when there’s a recall and to incorporate other information that might be
useful for clinicians and patients.”
ONC originally included UDI-EHR incorporation in a proposed rule in February but ultimately tabled the provision,
citing public comments expressing concerns that the technical changes were too steep. The final rule was released in
September and the Brookings report pushed back, recommending that ONC revive the tabled provision.
“Recognizing that UDI adoption within the certification criteria should be a requirement, ONC support for UDI
integration within EHRs is critical,” the report says. “As such, it should recommend a field for UDIs as part of the
associated certification criteria when CMS issues its proposed Stage III Meaningful Use objectives. These objectives are
currently under development and are expected in 2017.”
Similarly, UDI implementation should also involve insurers and insurance claims, Moscovitch said. He pointed to
FDA’s existing Sentinel Initiative which tracks drugs and vaccinations primarily through claims. The Sentinel Initiative
uses administrative claims data and electronic clinical data from major health plans to provide meaningful information
about drugs and vaccines in a national database for everyone in the healthcare system to use. Moscovitch said that UDIs
could also be assimilated into claims in the same way so that insurance companies could communicate better with
provider systems and patients.
“Right now, the claims that providers send to health plans often state that a certain procedure took place but not what
specific device was used in that procedure. If you included a field for UDI in the claim, that would provide additional
specificity.”
The Brookings-FDA study shared the same sentiments, saying the Sentinel Initiative is limited to just drugs, which
renders the system “less useful for broad medical device safety surveillance.” The report goes further, saying the system
should have scalable, information-rich data that would include adverse event reports and other key details. The data
would be accrued continuously for real-time analysis and be privacy-protected.
Brookings and FDA’s Center for Devices and Radiological Health (CDRH) is convening a multi-stakeholder
National Medical Device Postmarket Surveillance System Planning Board that is assigned with overseeing postmarket
surveillance of medical devices. The board’s report is expected to be released early next year. In addition, Duke Clinical
Research Institute, under the direction of FDA’s Medical Device Epidemiology Network and collaboration with the
agency’s device center, is convening a Medical Device Registry “task force” to analyze a number of things like best
practices for registries. “Together the Planning Board and Registry Task Force will support the development of
infrastructure that will be successful, in part, by leveraging the integration of UDIs across the health care sector,”
the report says. —David Hood
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FDA Gets Last-Minute Bump For FDA Food Safety, Ebola, Compounding
The year-end budget deal gives FDA food safety activities a bump above the amount Senate and House appropriators
had originally allocated, accounting for more than half of the $37 million increase in FDA’s budget that also beefs up
FDA’s compounding oversight, drug counterfeit controls and antimicrobial resistance monitoring program. The spending
agreement also sets aside a one-time $25 million funding pot for Ebola-related efforts, but withholds $20 million from
salaries and expenses until FDA’s opioid guidance is finalized.
The $1.1 trillion “cromnibus” package funds FDA at $2.59 billion through the end of the fiscal year, and provides a
$27.5 million dollar increase to FDA’s food safety efforts, accounting for close to 75 percent of the overall $37 million
increase over the agency’s 2014 budget. This increase is also above the $25 million House appropriators and the $23
million Senate appropriators originally provided the agency for food safety activities.
The rest of the funding increase is divided as follows: $15 million for pharmacy compounding; $4.82 million
for counterfeit drugs; $3 million for the National Antimicrobial Resistance Monitoring System; and $2 million for
foreign drug inspections. The agreement also incorporates proposed administrative savings in the amount of
$15.689 million.
Sandra Eskin, director of the Pew Charitable Trusts’ food safety program, praised lawmakers’ move to give FDA
more than they originally intended as the agency prepares for the release of key food safety rules the latter half of 2015.
She expressed hope that Congress will provide additional funding on the order of $300 million in the next two years.
The amount set aside for FDA’s pharmacy compounding efforts is more than the $12 million that House appropriators had originally planned to provide, but $10 million less the the $25 million requested by the administration. (see FDA
Week, May 22).
The bill provides the agency with a one-time $25 million funding pot to respond to the Ebola epidemic, and
orders the agency to provide quarterly obligation reports by program with specific accomplishments. This was part of a
White House multi-billion dollar emergency appropriations request to support Ebola efforts (see FDA Week, Nov. 6).
However, the funding agreement withholds $20 million from FDA’s salaries and expenses until the agency
finalizes its guidance on abuse-deterrent opioids’ evaluation and labeling. If the guidance is not finalized by June 30,
these funds will be transferred to FDA’s Office of Criminal Investigation to assist federal, state and local agencies to
combat the diversion and illegal sales of controlled substances.
The bill report also contains language that directs FDA to notify lawmakers of major changes to existing policies,
saying FDA has failed to do so in the past year.
“During the past year, FDA has informed non-governmental stakeholders of important decisions and announcements
before they informed the Committees,” states the legislation. “A collaborative working relationship between the Committees and agency is necessary to ensure efficient and effective implementation of Congress’s funding decisions. These
actions jeopardized this relationship. As such, FDA is directed to ensure the Committees are notified of major changes to
existing policies and any significant developments in its operations prior to providing non-governmental stakeholders
such information.”
The Alliance for a Stronger FDA said it is grateful that Congress recognized the agency’s responsibilities continue to
grow, but added that it remains concerned the agency is falling further behind each year with more responsibilities than
funding.
“We will continue to remind Congress that FDA’s mission and responsibilities are not static from one year to the
next; in fact, they are growing quite rapidly,” says the group. “There are a number of new laws the agency is implementing. Plus, scientific complexity increases every year and globalization is an incredible challenge that shows no sign of
leveling off. Simply put: Carrying out FDA’s responsibilities costs more than the monies appropriated to accomplish
them.”
The group says on its website: “Under usual negotiating protocols for appropriations, the higher of the House/
Senate bills is the most you can hope to receive. We were able to get the higher Senate number (about a 1.5%
increase) plus the Ebola supplement. We are grateful that the Congress recognized that FDA’s responsibilities
continue to grow, but we remain deeply concerned that the agency is falling further behind each year with more
responsibilities than funding.”
Moving forward, the group says it is already working on FY 16, which formally starts with the president’s State of the
Union in January. “We will not only be advocating for increased FDA funding, but will also need to advocate for some type of
agreement that prevents sequester cuts being implemented in FY 16,” the group says on its website. —Erin Durkin
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FDA Week - www.InsideHealthPolicy.com - December 19, 2014