InsideHealthPolicy.com’s
FDA Week
an exclusive weekly report on Food and Drug Administration policy, regulation and enforcement
Vol. 21, No. 8 — February 27, 2015
Stakeholders: Keep Communication About NGS Tests Outside FDA Purview
Stakeholders told FDA during a public workshop Friday (Feb. 20) on the agency’s potential new approach to nextgeneration sequencing tests that communication regarding certain information from such tests should continue to be
considered practice of medicine and fall outside of FDA regulation. The meeting also touched on the challenges of
getting payer coverage for such tests.
FDA held the meeting to gather feedback on its plan to regulate NGS tests differently from other laboratorydeveloped tests, which the agency is moving to regulate under its medical device risk classification. The effort is part of
continued on page 6
In what industry sees as a welcome trend...
FDA Clears Path For Some Genetic Tests To Skip 510(k) Process
FDA plans to reclassify DNA carrier screening tests as Class II devices exempt from premarket review in an effort
to deregulate direct-to-consumer (DTC) genetic tests, the agency said Friday (Feb. 20) as it approved the first-ever
consumer marketing of a genetic carrier test, specifically the Bloom syndrome test 23andMe. An industry lawyer said
the move is a welcome trend by FDA to exempt certain diagnostic and digital health products from the 510(k) process.
In the first six weeks of 2015, FDA released three separate guidance documents on devices it intends to deregulate.
continued on page 7
FDA Says Whole Genome Sequencing A Breakthrough In Food Safety
FDA says its use of whole genome sequencing (WGS) is a huge breakthrough in identifying and tracing the source
of foodborne illness outbreaks from bacteria like Salmonella and Listeria. WGS has allowed FDA to quickly trace
outbreaks back to their geographical sources earlier in the process than ever before, an agency microbiologist said
Monday (Feb. 23) at a Food and Drug Law Institute Food Week panel. A GenomeTrakr database, developed by FDA
through partnerships with other agencies and states, will limit the scope of foodborne outbreaks within five years, he
said.
continued on page 9
Advisors Give FDA Nod To Put 26 Drugs On Non-Compounding List
An advisory committee this week concurred with FDA’s suggestion that 26 drugs be listed as unsafe or not effective
for compounding, firming up a highly anticipated updated list that is key to the agency’s implementation of the new
compounding law. The committee reached the decision nearly unanimously, though there was some splitting over the
oral use of a drug that treats serious bacteria-based infections when other antibiotics are ineffective. An FDA official
told advisors the Investigational New Drug (IND) route could be used to provide patient access when a physician
believes an individual patient needs a drug on the list.
continued on page 10
Public Citizen Hits FDA For ‘Bowing’ To Industry Over Labeling Rule
Public Citizen took a swipe at FDA Thursday (Feb. 26) for delaying its contentious generic drug labeling rule in
order to give the brand and generic drug industry an opportunity to publicly pitch an alternative plan to get new safety
information on drug labels. The generic and brand lobby groups sent FDA their alternative in late November and plan to
present it at an FDA public meeting next month, but Public Citizen denounced the industry plan as worse than the status
quo that requires generic labels match their brand counterparts. Public Citizen backs FDA’s proposed rule that would
require generic drug makers unilaterally update their labels if new safety information becomes available.
continued on next page
The plan by the Generic Pharmaceutical Association and Pharmaceutical Research and Manufacturers of America
would let FDA determine if a labeling change is necessary, and then require the brand-name and generic companies to
change their labels via electronic labeling within 30 days. Laws governing labeling currently require only brand-name
companies to change their labels if new safety information is discovered—and generic companies must have the same
information on their labels.
Public Citizen criticized the GPhA/PhRMA alternative in a statement Thursday (Feb. 26), saying it is “bad”
because it would lead to:
Fewer labeling updates, as no company would be required to, or have incentive to, request updates.
Slower updates, as the FDA would have to preapprove every warning update.
No company being held accountable to injured patients for failure to warn about newly discovered risk.
FDA first proposed its generic labeling rule in 2013 and had planned to finalize it in December of 2014, but instead
pushed the date to September 2015, amid intense pressure from industry to change the rule. GPhA threatened to sue FDA
if it approved the rule as proposed. Earlier this month, FDA announced it would reopen the docket for more comments
and plans to host a public meeting next month to allow stakeholders to present their ideas.
Michael Carome, director of the Health Research Group at Public Citizen, maintains that those actions by
FDA to delay the rule were “bowing” to industry, keeping vital safety information away from consumers. “We think
the rule that FDA proposed is a very common-sense, reasonable rule for approving drug safety with respect to patient
care. It’s a very simple rule and there’s no reason for the delay implementing it in a final form,” Carome said. “And we
know industry has brought great pressure to bear both directly on agency and through members of Congress. There have
been no good arguments, we believe that are valid and should result in the rule not going forward.
But GPhA President Ralph Neas strongly disagrees. “From the beginning, the trial lawyers and Public Citizen
have been wrong on the facts, wrong on the policy and wrong on the law. The current language of the proposed rule on
generic labeling — which incorporates 95 percent of what the trial lawyers and Public Citizen lawyers publicly submitted
back in 2011— would endanger public safety, endanger public savings and would endanger the continued viability and
enormously effective Hatch-Waxman Act,” said Neas, in an interview with FDA Week.
The industry groups say their alternative proposal strikes a balance between public health concerns and industry, by
giving FDA the authority to determine if a labeling change is required. Neas said that is the most effective way of
ascertaining if a change is necessary because the agency is the sole entity that has all the information on the drugs.
But Public Citizen again disagrees, saying instead that the alternative would further delay patient safety
information from reaching consumers. Carome told FDA Week in an interview that the agency has indicated it is
already too stretched thin with resources, and the additional burden of making labeling decisions imposed by the industry
proposal would push their limited resources even further. For those reasons, Carome said, the industry proposal would
make matters worse.
“Right now, at least, for those drugs that are available in both in brand-name version and generics, you have
brand-name companies who have incentives to do robust safety monitoring surveillance for drugs on the market and
take prompt action to update their labels,” said Carome. “This alternative proposal says they no longer have that
incentive because we’re just going to let FDA do all substantive changes on drug labeling regarding safety information.”
But PhRMA responded that Public Citizen’s criticisms are inaccurate and unfounded, saying the rule should be
revamped to avoid having generic drug labels differ from innovator labels. “FDA should amend the rule to ensure that
patient safety would not be compromised by FDA allowing innovator and generic versions of the same drug to bear
different safety information,” said Jeffrey Francer, vice president and senior counsel at PhRMA, in a statement to FDA
Week. — David Hood
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FDA Week - www.InsideHealthPolicy.com - February 27, 2015
FDA Plans To Bring In Food Experts To Beef Up Field Inspection Efforts
In a move an FDA official called “game changing,” the agency is bringing in food subject experts to work with its
field inspectors in a bid for consistent, real-time responses to food safety issues, and hopes better-trained field inspectors
will also be able to advise stakeholders on ways to prevent future problems as they conduct their inspections. FDA is
beefing up its food center staff, bringing in subject matter experts and stepping up its training of inspectors as it begins
implementing and enforcing the 2011 Food Safety Modernization Act (FSMA), an agency official said Monday (Feb. 23).
FSMA is a one of FDA’s top priorities for 2015 as it faces court-ordered deadlines to release seven final rules over
the next year, and President Barack Obama’s 2016 budget seeks a $109.5 million increase for the agency’s food safety efforts.
“FDA needs to speak with one voice,” said Ted Elkin, acting deputy director for Regulatory Affairs at FDA’s Center
for Food Safety and Applied Nutrition (CFSAN). “We can’t have field people in Michigan speaking differently than our
field inspectors are doing in California. We really have to be speaking with one voice, and we have to be in concert
between different organizations. To do that we’re going to have to find that common ground and that ability to not just
talk about it but implement it.”
Under a court-ordered consent decree FDA entered into last February, the agency will release final FSMA rules on
preventive controls for human food and preventive controls for animal food by Aug. 30; produce safety, the foreign
supplier verification program and third party verification by Oct. 31; sanitary transport by March 31, 2016; and Intentional Adulteration by May 31, 2016.
“We really have to get this right,” Elkin said. “And we really have to get this right by working in partnerships with
others... In terms of the goals to get us to the right type of rule, and the opportunity to get it right is a challenge that is
meetable. We are determined to do that.”
Elkin said the agency is updating its IT infrastructure to allow for more robust data collection and analysis.
FDA is also working to set new performance measures and metrics in order to be preventative rather than reactive, Elkin said.
He also said the Office of Food Safety has grown by 60 people, with the agency hoping to bring on more in order to
implement FSMA.
In order for the agency to “speak with one voice,” Elkin said, the agency is bringing on subject matter experts
to work with field inspectors. He said inspectors will be able to call subject matter experts when they encounter
problems in the field and work through the issue. He said FDA is also investing in regulator training to provide consistency in decision making.
“Hopefully that will be something we can do much better in a real time situation,” Elkin said. “I think that will be for
us actually game changing.”
Elkin said field inspectors will also advise stakeholders on ways to prevent future problems more efficiently as they
conduct their inspections.
The president’s 2016 budget asks for a $109.5 million increase in dedicated funds for FDA’s efforts to implement
FSMA. The budget also seeks $191.7 million in new food user fees. Obama has proposed the user fees in the past, but
they have met with a chilly reception in Congress. — Todd Allen Wilson
Eshoo, Lance Reintroduce Bill Exempting FDA User Fees From Sequester
A bill to shield FDA’s user fees from sequestration that took out an estimated $85 million in 2013 was reintroduced
by Reps. Anna Eshoo (D-CA) and Leonard Lance (R-NJ) on Wednesday (Feb. 25). Industry groups praised the bill,
saying broadly that protecting user fees from sequestration would help FDA approve drug, biologic, biosimilar, medical
device and diagnostic products more quickly.
The bill, called the FDA Safety Over Sequestration Act, would simply insert a clause in the Balanced Budget and
Emergency Deficit Control Act of 1985 exempting user fees from automatic budget cuts. In 2013, the bill garnered 87
bipartisan co-sponsors but died. The Omnibus Appropriations legislation, passed a year ago, retroactively restored $2.85
million of sequestered user fees to FDA from 2013, and the Murray-Ryan budget deal eliminated cuts for 2014 and 2015.
But FDA faces another round of sequestration in 2016.
Jim Greenwood, president and CEO of the Biotechnology Industry Organization, maintained that sequestering user
fees paid by private companies to FDA does not lower the nation’s deficit. “Rather, the only real effect is that the FDA
would not be able to access all fees, thus negatively impacting the ability of the FDA to fulfill its mission of ensuring
patient access to safe and effective new drugs and biologics,” said Greenwood, in a statement.
Stephen Ubl, president and CEO of the Advanced Medical Technology Association, agreed, saying the bill would
restore the agreement negotiated by all three parties in the Medical Device User Fee Amendments. “That is a win-win-win
for FDA, the industry and the millions of American patients who will benefit from more timely access to innovative
medical technologies,” said Ubl in a statement. “However, going forward into FY16 and beyond, additional medical
device user fees are at risk since sequestration cuts could come into effect once more. This situation requires a permanent
fix,” he said. — David Hood
FDA Week - www.InsideHealthPolicy.com - February 27, 2015
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Brookings Lays Out Plan For Medical Device Postmarket Surveillance System
A new Brookings Institution report sets out a seven-year plan for stakeholders to fully adopt and implement an
effective postmarket surveillance system for medical devices (MDS). FDA first proposed the MDS in 2012 and established a planning board to come up with the report for how to create and implement it.
The system is intended to provide more and better information on the benefits and risks of medical devices in order
to detect any problems or adverse events associated with devices. The MDS is also supposed to be funded by a multistakeholder public-private partnership, with initial seed money coming from FDA, approved by congressional appropriators.
While the 76-page Brookings report, released Monday (Feb. 23), estimates MDS will cost $200-$250 million, based
on precedents set by the National Patient-Centered Clinical Research Network (PCORnet) and the Sentinel system, which
tracks drugs in postmarket space. The report points out that while Congress enacted legislation in 2012 mandating FDA
broaden the Sentinel system to include medical devices as well, it did not specify how to pay for it through appropriations
or user fees.
Greg Daniel, managing director for Evidence Development and Innovation at Brookings’ health research center, told
FDA Week that Sentinel and PCORnet received seed money from the government and that there is little reason to believe
Congress would not invest into MDS. The challenge, said Daniel, is to build support for the system before it exists. The
goal would be to get the ball rolling with the seed money and immediately bring in others to sustain it.
“(I)t will take some government seed money to build. Quite honestly, the private sector is part of the long-term
sustainability plan for the system,” said Dainel. “We think that once the system is up and running, medical device manufacturers, payers and other members of the private sector will actually start deriving value by participating and that will
incentivize that.”
The cornerstone to MDS, the report says, are unique device identifiers (UDI), or labels which contain key information about the device, required to be affixed to every device approved by FDA. The actual labels are able to be read by
humans and scanned by manufacturers and clinicians. The idea is to be able to track the device as it moves through the
healthcare supply chain until it reaches a patient. FDA has done its part, requiring all devices to have a UDI and setting
up a massive UDI database launched last month filled with information for Class III (high-risk) devices. The report
assumes UDIs will also be adopted into insurance claims forms and patient electronic health records, despite the difficulties stakeholders have run into at CMS and the Office of the National Coordinator, respectively.
In the first two years, the MDS plan recommends the initiation an incubator project tasked to “develop a five-year
implementation plan for MDS through fact-finding activities and pilot program,” the report says. “The incubator project
should be initiated by FDA, adequately staffed and resourced, and guided by a multi-stakeholder group with relevant
medical device experience.” That group, the report specifies, would be made up of patients, clinicians, hospitals, payers
and medical device manufacturers.
“If we want to know what the real benefit-risk profile of a technology is, then we have to see its use in the wild, in
clinical practice,” said Jeff Shuren, director of FDA’s Center for Devices and Radiological Health. But you have to gather
that data systematically and you have to able to make sense of it. And if you can do it, you can fundamentally change how
we introduce technologies, and how we assess technologies in the U.S. And that is what is behind our proposal in 2012 to
establish a national medical device postmarket surveillance system.” —David Hood
Experts To Industry: Work To Meet FSMA Requirements Ahead Of Rules
Consumer and industry food safety experts say companies should begin putting together risk management and food
safety plans as well as training employees ahead of FDA’s implementation of the 2011 Food Safety Modernization Act
(FSMA), because if they wait to begin the process after the agency releases seven final rules over the next 15 months it
will be too late.
The experts, speaking at a Food and Drug Law Institute Food Week panel Monday (Feb. 23), urged companies to
being implementing hazard analysis and critical control points (HACCP) style plans; registering their individual facilities
every two years; providing food safety plans and associated materials to FDA inspectors; identifying suppliers —
especially foreign suppliers — they will need to verify; and meeting performance standards set by FDA for controlling
hazards. One expert said that companies should keep in mind that food allergens are now classified as a hazard under
FSMA.
“Don’t think you can start getting ready after the final rules are released,” said Caroline Smith DeWaal, director of
Food Safety at the Center for Science in the Public Interest. “It’s really time now to be getting your house in order for
FSMA. It’s coming. The regulatory process may make it look like it’s leisurely, but it will be here sooner than you think.”
FDA is mandated by a court-ordered consent decree to issue seven final rules over the next 15 months, including
final FSMA rules on preventive controls for human food and preventive controls for animal food by Aug. 30; produce
safety, the foreign supplier verification program and third party verification by Oct. 31; sanitary transport by March 31,
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FDA Week - www.InsideHealthPolicy.com - February 27, 2015
2016; and intentional adulteration by May 31, 2016.
Martin Hahn, a partner at Hogan Lovells law firm, pointed out that compliance with the various rules will be expected by the fall of 2016 and spring of 2017. Even though the FSMA rules are not in effect, he said, companies that
begin preparing now could choose to let FDA review their plans in order to get feedback on whether they are on the right track.
L. Robert Lake, a senior consultant with EAS Consulting Group who worked at FDA for 30 years, said companies
should begin by identifying what challenges they face based on what they already know about FSMA requirements;
determining what tasks musts be performed; assigning responsibilities for implementation; and implementing requirements that are already clear. Lake noted that FDA has issued FSMA guidance on registration, record keeping, prior notice
and administrative detention.
Lake, DeWaal and Hahn all emphasized that it is important for companies to begin training their employees
on FSMA and its requirements now. Hahn said it is important that employees understand the importance of meeting
FSMA requirements and what that will entail, especially for employees that have worked in the industry for years and are
set in their ways.
“It requires a cultural change,” Hahn said. “We as an industry have to impress on our employees — particularly those
who have been there 20 or 30 years who’ve been doing it the same way — that we need to do things differently, because
if that record is not recorded at the time that it occurred as far as FDA is concerned it didn’t take place. When these rules
become mandatory you’ve got to make sure those employees understand the importance of good record keeping.”
DeWaal said it is “critically important at this stage” for companies to begin putting together their food safety plans,
identify known hazards — which include chemical, physical, natural toxins, pesticides, drug residues, decomposition,
parasites, allergens, unapproved food or color additives and other hazards that may be unintentionally introduced — and
identify how they will control for those hazards. She said companies can do this by developing a standard HACCP-style
plan, that can be updated as rules and regulations are finalized.
Lake said it is important that companies in developing their food safety plans take into account the eight
major known food allergens — milk, eggs, fish, crustacean shellfish, tree nuts, wheat, peanuts and soybeans. He said
that in the past allergens have only been a labeling issue, but under FSMA they are now classified as hazards. While this
doesn’t mean that these cannot be used in products, the FDA will be strict about allergen cross-contact with products that
do not normally contain them and will require preventive controls in this area.
“I know that FDA, and I think it’s still true, gives priority to food safety issues over food labeling issues,” Lake said.
“Allergens are now a food safety issue, so I think that ups FDA’s focus on food allergens.”
He said it is important that companies began making sure they are labeling products with known allergens correctly
and set in place procedures for keeping undisclosed allergens out of their products. Lake said this is an area in which it is
especially important to train employees.
“I think many employees don’t fully understand that a mix up in a food label or a cross-contact through storage bins
that haven’t been adequately cleaned…can present a life-threatening situation to some people who have strong allergies
to those things,” Lake said. — Todd Allen Wilson
FDA Looks Outside Rulemaking To ‘Reinvigorate’ Nutrition Efforts
FDA plans to “reinvigorate” its focus on nutrition this year, but will do so largely through educational efforts rather
than the regulatory arena in 2015, an FDA official said at a Food and Drug Law Institute Food Week conference in
Washington Monday (Feb. 23).
“Believe it or not in CFSAN there is an ‘N,’ and it’s called nutrition — it’s the Center for Food Safety and Applied
Nutrition,” said Ted Elkin, acting deputy director for regulatory affairs at CFSAN. “That has not always been on the
forefront of how we’ve looked at our objective, and it really should be.”
Elkin said the agency’s food center will continue efforts related to nutrition after having released proposed rules on
nutrition labels and serving sizes and final rules for vending machine and menu labels last year.
“If you think about food safety and you think about the metrics the incidence of deaths related to food safety versus
the incidence of death related to stroke, heart attack, obesity there’s really no comparison,” he said. “So the effort to
reinvigorate nutrition has been a part of how we need to do our business.”
FDA also plans to continue efforts to reduce the use of partially hydrogenated oils — the primary source of industrially produced trans fat in the food supply, and promote a broad, gradual reduction of added sodium in the food supply.
Aside from publishing a proposed rule on gluten-free labeling of fermented and hydrolyzed food, Elkin said the
agency is not planning on proposing rules or guidance on nutrition issues because getting out the seven final FSMA rules
will dominate CFSAN’s rule making efforts.
“I don’t know that nutrition has to be necessarily solved by rule making,” Elkin said. “In the meantime there’s
plenty we can be educationally and suggesting in terms of addressing nutrition concerns that we maybe haven’t done as
well as we should have.” — Todd Allen Wilson
FDA Week - www.InsideHealthPolicy.com - February 27, 2015
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FDA Approach To NGS Tests Discussed . . . begins on page one
the White House’s recently unveiled Precision Medicine Initiative, in which the administration proposes to give FDA
extra funding to beef up its expertise and databases to support a new regulatory structure that both spurs innovation in
precision medicine and protects public health. President Barack Obama said last month that the agency needs to differentiate between these tests and other medical devices.
In a discussion paper released in December, the agency says there are challenges to regulating next-generation
sequencing tests because these tests can identify an unlimited number of variants based on over 3 billion base pairs of the
human genome. Clinical relevance of many variants detected by NGS tests may have limitations because of the rarity of
the mutations, and challenges associated with communicating information regarding the significance of the presence of
genetic variants in a way physicians and consumers understand.
One of the areas addressed by the paper is whether and how to communicate information about less wellunderstood variants. “Some stakeholders have asserted that information regarding genetic variants for which there is not
sufficient evidence to demonstrate clear clinical significance, but for which there is evidence to establish a likely association, may have some value to physicians and their patients in clinical decision-making in certain circumstances,” the
paper states.
The agency asks for public comment on the value of conveying information about genetic variants for which there is
limited evidence of clinical significance; whether some caveat as to the limitations of the available data may be needed;
and, if this can be of value, how and under what circumstances the information should be conveyed, to assure the information is effectively communicated.
Stakeholders weighed in with various opinions at Friday’s public meeting.
Len Lichtenfeld, deputy chief medical officer for the American Cancer Society, took issue with “FDA approving
which information can be shared with a patient,” adding that the agency doesn’t have the resources to do so.
In public comments, David Flannery, medical director of the American College of Medical Genetics and Genomics,
also said that communication of information from these tests should remain in the practice of medicine, outside of FDA’s
jurisdiction.
In addition the Association for Molecular Pathology said in comments submitted to FDA that the assay design,
validation and interpretation of NGS procedures are essential medical services performed by highly qualified professionals. “As these activities are central to the practice of medicine, they must remain outside the purview of FDA,” said the
group.
Other stakeholders addressed the issues surrounding coverage of such tests.
Carl Gordon, partner of OrbiMed: Healthcare Fund Management, told FDA that it needs to have a certain goal in
mind when it comes to the new approach and focus on where problems are, but adds that he is not aware of any big
errors. He also says payers are currently reluctant to cover diagnostics.
Donna Messner, vice president and senior research director for the Center for Medical Technology Policy, said
payers are looking for effective technology but there are challenges “when they don’t feel warm and fuzzy about analytical validity and clinical validity of tests that you are asking them to cover.” They want confidence that a test represents an
answer that’s reliable and comprehensible.
“They don’t feel confident that’s what they are looking at right now,” said Messner.
The issue comes under FDA’s approach for assessing the clinical performance of NGS tests, where the agency
references its clearance of Illumina’s NGS-based cystic fibrosis assays. “FDA and the developer addressed the challenge
of providing evidence for the clinical relevance of rare variants by allowing the developer to make use of a well-curated
third-party database consisting of evidence from multiple sources to establish clinical significance, as an alternative to
conducting a new study or using existing literature,” FDA says.
The agency sought public input on how this approach could be applied more broadly to enable access to timely,
accurate and reliable genomic information in a manner that is meaningful for clinical decision-making.
FDA Commissioner Margaret Hamburg said next generation sequencing tests may be better handled through a new
regulatory approach rather than under FDA’s current approach to devices.
“Most diagnostic tests follow a one test — one disease paradigm that readily fits FDA’s current device review
approaches for evaluating a test’s analytical and clinical performance,” said Hamburg in a blog last month. “Next generation sequencing produces a massive amount of data that may be better handled using a new approach.” — Erin Durkin
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FDA Week - www.InsideHealthPolicy.com - February 27, 2015
FDA Rejects Last Two Sunscreen Ingredients, Calls For More Safety Data
FDA officially passed on two remaining sunscreen ingredients Tuesday that have been pending approval or rejection
by the agency since they were submitted as far back as 2002, on the grounds that the ingredients were not considered
generally recognized as safe and effective (GRASE). Industry advocates, who they say were expecting FDA to approve
the ingredients upon passage of a law last year, vowed to return to Congress to discuss another potential legislative
solution.
The agency rejected six other pending applications in January and met with stakeholders earlier this month to explain
the next steps under the recently passed Sunscreen Innovation Act.
FDA on Tuesday (Feb. 24) said it was also not approving the two remaining ingredients, enzacamene and ecamsule,
because “the data are insufficient to classify it as GRASE and not misbranded, and additional information is necessary to
allow us to determine otherwise.” The additional information, says the letter to L’Oreal, the ecamsule applicant, is safetybased. The letter lays out a study that would provide sufficient information.
“We completed another requirement by taking initial action on the last two pending ingredients, ecamsule and
enzacamene,” said Theresa Michele, director of the drug center’s Division of Nonprescription Clinical Evaluation
in a blog. “We tentatively determined, as we had with the other six ingredients, that we need more data to decide if
these ingredients are, in fact, GRASE for use in OTC sunscreen products... At this time there is not enough generally available data to determine whether any of the ingredients under review meet FDA’s safety and effectiveness
standards.”
The announcement came as another disappointment to stakeholders, who were disappointed the first time in January
when FDA rejected the first six ingredients. “It’s disappointing it took them this long to tell us,” said Wendy Selig,
president and CEO of the Melanoma Research Alliance. “I hope that the fact FDA has gone back now to all the companies involved with pending applications and said ‘we’re not satisfied, we need more data,’ that the companies don’t look
at this market and say, ‘there’s no way in.’”
Industry stakeholders said they have 10 years worth of postmarket data verified by European countries and others
that prove the quality of their products. They expected FDA to consider the applications since the law requires the agency
determine whether the provided information is sufficient to conduct a review. Michael Werner, a partner at Holland and
Knight and staff member at the Public Access to SunScreens Coalition, told FDA Week the coalition would go back to
Congress, which he said is likely to be disappointed with the agency’s decision. But in the meantime, he said, the coalition would work with to get the data the agency requests.
“Certainly in our conversations with champions of this legislation, and folks in Congress, there’s a lot of concern
over the way FDA is implementing the statute,” said Werner. “I anticipate when FDA comes before Congress or even
Secretary Burwell comes before Congress over the next several weeks to talk about their priorities in 2015 and 2016,
there will be a lot of tough questions that will be asked.” —David Hood
CDRH Deregulates Genetic Test . . . begins on page one
Bradley Merrill Thompson, an attorney for Epstein Becker & Green who serves as general counsel for the mHealth
Regulatory Coalition, told FDA Week that the guidance documents signal that the agency is interested in spurring innovation. FDA’s approval of 23andMe’s application blazes the trail for other DTC genetic testing products to bypass the
510(k) process, he said.
“To me, the press release says that for the whole category, they plan to leave it in Class II, but exempt it from
the most burdensome aspect of Class II, which is 510(k) requirement,” said Thompson. “And that’s pretty remarkable.”
Thompson said the leadership at FDA’s Center for Devices and Radiological Health is genuinely interested in
innovative products and is committed to making sure the agency can get them to market as quickly as possible. He said
the change in attitude at CDRH had also to do in part with industry’s cooperation with FDA in providing sufficient data to
support expeditious approvals.
The interesting thing, Thompson said, was that 23andMe even received a warning letter in 2013 from FDA, demanding the company stop marketing its product because it was not granted clearance. The agency called the marketing of the
device in violation of the Federal Food, Drug and Cosmetic Act. The letter said 23andMe’s product is intended to
diagnose diseases or other conditions or in the cure, mitigation, treatment, or prevention of disease.
Although the company submitted two 510(k) applications, FDA said the applications did not include the device’s
intended uses. The company complied and submitted more data. Then, in a change, said Thompson, FDA decided to
classify all carrier screening tests as Class II.
FDA was originally wary of DTC genetic tests because of their track record of inaccuracy. While FDA believed
consumers had the right to know their genetic makeup, the agency maintained DTC products were generating too many
faulty results, Thompson said.
“FDA believes that in many circumstances it is not necessary for consumers to go through a licensed practitioner to
FDA Week - www.InsideHealthPolicy.com - February 27, 2015
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have direct access to their personal genetic information. Today’s authorization and accompanying classification, along
with FDA’s intent to exempt these devices from FDA premarket review, supports innovation and will ultimately benefit
consumers,” said Alberto Gutierrez, director of the Office of In Vitro Diagnostics and Radiological Health at CDRH.
“These tests have the potential to provide people with information about possible mutations in their genes that could be
passed on to their children.”
“In just six weeks of this year, FDA has just done a remarkable job of deregulating low-risk technologies,”
Thompson said. “And you got to believe they’re not done.”
FDA issued a guidance earlier this month expressing its intent not to enforce Class I device regulatory controls for
medical device data systems (MDDS), medical image, storage and medical image communications devices. Thompson
said then that the agency was applying the lightest regulatory touch appropriate for software. Before that, the agency
released another guidance defining what a “wellness product” is and its intent to deregulate those products as well. In
addition, Thompson said, FDA cleared a de novo submission by Dexcom that led to another deregulation of an entire
category.
Thompson said in each instance, someone in industry was advocating for the deregulation of those technologies. The
next category, he said, can be from anywhere. But FDA’s actions since the start of the new year indicate a commitment to
spurring innovation in technology for medical devices. — David Hood
Hospitals, Rural Health Lobbyists Support HRSA In Orphan Drug Lawsuit
Hospitals and the National Rural Health Association backed the Health Resources and Services Administration’s
interpretation of orphan drug discounts in the 340B program in court, telling the federal district court judge in recently
submitted briefs that striking down HRSA’s latest 340B orphan drug rule could be devastating for rural hospitals.
Pharmaceutical Research and Manufacturers of America sued HRSA twice over its interpretation of the 340B orphan
drug exclusion in the Affordable Care Act, one of which is still pending. The law excludes orphan drugs from the discount, but HRSA says that discount applies when orphan drugs are used off label or to treat common conditions. The
Safety Net Hospitals for Pharmaceutical Access and NRHA and the American Hospital Association in a separate brief
supporting the administration argue that for the ACA’s expansion of the 340B drug discount program to cancer centers,
critical access hospitals, rural referral centers and sole community hospitals for outpatient drugs to be useful for hospitals,
HRSA’s interpretation must stand.
“The ACA’s expansion of the 340B drug discount program enables rural hospitals to provide more patients much
needed access to lower-cost medications. Interpreting the ACA to exclude all uses of drugs with an orphan designation,
including indications for non-orphan diseases and conditions, would nullify the benefits of the expansion of the 340B
Program,” the AHA brief says.
The AHA argues that the expansion of the 340B program benefits hospitals and Medicare. Medicare reimburses
critical access hospitals based on their allowable costs, and drug discounts lower allowable costs so Medicare
spends less.
The courts overturned HRSA’s first orphan drug rule, but the judge in the first lawsuit didn’t consider the rule’s
interpretation of the law. Rather, the judge ruled that HHS did not have the authority to issue a binding, legislative rule.
The second interpretive rule, which is essentially the same interpretation, should stand because the plain language of the
ACA limits the orphan drug exclusion to only orphan uses, SNHPA and NRHA say, “and does not sweep in the many nonorphan uses for which medications may be administered.”
PhRMA, however, called the interpretive rule a “power play” and said in its complaint that this is the second time
HHS has tried to re-issue a flawed rule. PhRMA says HRSA’s interpretation violates Congress’ intent because all that
matters is the orphan designation, not how the drugs are used.
The hospitals, SNHPA and NRHA note that many orphan drugs are extremely profitable for drug makers. AHA says
PhRMA is ignoring that the purpose the Orphan Drug Act is to encourage manufacturers to invent drugs for rare diseases
and conditions. PhRMA’s argument would “jeopardize the financial sustainability of those hospitals, while at the
same time providing a windfall to drug manufacturers for uses of the drug unrelated to the rare disease or conditions
for which it was designated; as it is inconsistent with the text and purpose of the legislation that it purports to
construe,” AHA says.
HHS’ interpretation promotes both the goals of the 340B program — lower costs for hospitals — while also
incentivizing the production of orphan drugs, SNHPA and NRHA say.
PhRMA says the 340B program has grown beyond congressional intent, but hospitals say lawmakers intended
discounts to be available for hospitals to not only pass on to patients but to also help keep their doors open and provide
extra services. Former Rep. Henry Waxman (D-CA), who chaired the House Energy & Commerce Committee when the
ACA was passed, said HRSA is interpreting the law correctly, and brand drug makers want to undermine the 340B
program.
If PhRMA wins the debate, SNHPA and NRHA say outpatient drug costs will increase 19 percent over the next year
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for their members, based on member surveys. They also say many hospitals would consider dropping out of the 340B
program.
“Affected hospitals are willing to bear the administrative complexity and costs of complying with 340B requirements, but withdrawing the critical discounts for non-orphan uses of orphan drugs would severely undermine the benefits,
leading many to leave the program altogether,” SNHPA and NRHA say. This, in turn, would mean higher drug prices for
patients and less services provided by these hospitals, they argue. — Michelle M. Stein
FDA Scientist: WGS Is ‘Revolutionary’ . . . begins on page one
“The Salmonella that come out of the Salinas Valley in California do not look at all genetically that come from the
East Coast right here or from the Salmonella in Southeast Asia, Mexico, Europe or other parts of the world,” said Eric
Brown, director of the Division of Microbiology at FDA’s Center for Food Safety and Applied Nutrition. “This is a huge
breakthrough too, because now it means there’s phylogeographic signal in the pathogens themselves. We should have
known it. Charles Darwin knew it. He knew it 150 years ago, it’s just he didn’t have the cool tools to do it.”
Brown said FDA realized it could use WGS in 2009 when it used it to identify the cause of a Salmonella outbreak
caused by salami in New England.
The “watershed moment” came in 2012, he said, when the agency was able to use WGS to trace a Salmonella
outbreak in spicy tuna that effected the East Coast — particularly New York and Maryland — back to Moon Fisheries in India.
“For us this was a forensic watershed moment because it meant that now we can start building a massive
database much like the FBI does for human DNA samples for their forensic data base,” Brown said. “We want to do the
same thing for Salmonella and other bacterial pathogens.”
Since then FDA has worked with the Centers for Disease Control and Prevention (CDC), the National Institutes of
Health (NIH) and 10 state health agencies to develop the GenomeTrakr database housed at NIH, he said.
The development of GenomeTrakr allowed the agency to quickly identify the source of an outbreak caused by cheese
and suspend registration of the food processing facility in Maryland where the product was made in March 2014.
This was quickly followed last summer with FDA being able to identify early in the process based on just a few
people getting sick the source of an outbreak in the Pacific Northwest caused by a small nut butter producer. This allowed
FDA to become involved in the tracing process 10 days out, which is much earlier than normal, he said.
“That was a big deal because that meant in many ways you now have surveillance or a radar system that runs
from the surface of the ocean to the edge of space,” Brown said. “So if you have two illnesses you can pick up
whether they’re related in any way you know of or not.”
Brown said the agency is able to sequence a sample in a matter of hours cheaply. Where the process cost $600 in
2008 it now costs $40 to sequence a genome.
“That’s almost as cheap or as cheap as field gel analysis,” he said.
As the GenomeTrakr data base grows and next generation sequencing technology advances, Brown said, the agency
believes in the next five to 10 years it will be able to manage a whole outbreak using WGS.
“It’s a revolutionary technology; it’s quite disruptive — it’s made people stop and think about where we want to
go from here,” Brown said. “It can limit the scope of outbreaks and link back like never before. When you link it to
open source outbreak data bases it’s going to empower it for sentinel work on a global scale unlike you’ve seen
previously.” — Todd Allen Wilson
Alexander: WH Needs Someone ‘On The Flagpole’ For Health Threats
Senate health committee chair Lamar Alexander (R-TN) said at a hearing Thursday (Feb. 26) that the administration
needs to ensure someone is “on the flagpole” during future health threats, saying there was confusion as to who was in
charge during the “near panic in the United States over Ebola,” according to a release from his office.
Alexander said that if the assistant secretary of preparedness and response can’t be seen in charge during an
epidemic, then maybe the law should be changed. “First it was [Centers for Disease Control and Prevention director
Tom] Frieden, next it was the [National Institutes of Health], then the president appointed a czar who became the
phantom of the White House and disappeared and was never seen again,” said Alexander. “Now maybe he did a
good job, but none of us would know it...This is something we should think about before we have another incident
like that.”
The Senate health committee held a hearing Thursday on the medical and public health preparedness for future
threats. Invited to testify were HHS Assistant Secretary for Preparedness and Response Nicole Lurie; HHS Biomedical
Advanced Research and Development Authority Director Robin Robinson; Centers for Disease Control and Prevention
Office of Public Health Preparedness and Response Director, Stephen Redd; and FDA Director of the Office of
Counterterrorism and Emerging Threats Luciana Borio. — Erin Durkin
FDA Week - www.InsideHealthPolicy.com - February 27, 2015
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Advisors Discuss Non-Compounding List . . . begins on page one
The Drug Quality and Security Act requires FDA to consult with a new Pharmacy Compounding 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.
The drugs that advisors agreed be put on the list are: chloramphenicol oral drug products, alatrofloxacin mesylate,
aminopyrine, astemizole, bromfenac sodium, cerivastatin sodium, cisapride, esmolol hydrochloride, etretinate,
gatifloxacin, grepafloxacin, methoxyflurane, novobiocin sodium, oxycodone hydrochloride, pemoline, pergolide
mesylate, phenylpropanolamine, polyethylene glycol (PEG) 3350, propoxyphene, rapacuronium bromide, rofecoxib,
sibutramine hydrochloride, tegaserod maleate, troglitazone, trovafloxacin mesylate, and valdecoxib. The advisors also
agreed to the entry for adenosine phosphate to be updated to state: “All drug products containing adenosine 5'-monophosphate (AMP), adenosine 5'-diphosphate (ADP), and adenosine 5'-triphosphate (ATP).”
While advisors unanimously agreed that most of the suggested drugs be listed, there was 9-2 split for chloramphenicol oral drug products, with a pediatric clinician and a dermatologist voted against inclusion on the list.
Chloramphenicol is currently available as an injection, and is an antibiotic that treats serious infections when other
antibiotics cannot be used, according to Medline Plus. However, FDA says that oral drugs containing chloramphenicol
were withdrawn from the market for safety and effectiveness reasons.
Katherine Pham, a Neonatal Intensive Care Unit Pharmacy specialist for the Children’s National Medical Center,
said the fact that an intravenous solution of the drug is on the market may lead some physicians to think it is feasible to
prescribe an oral use version.
“Due to the widespread use of broad spectrum antibiotics now, it remains a very good anti-infective as it retains its
susceptibility in a lot of organs due to its under use…What if some extrapolate it as oral as it already comes into solution?
It would be prudent to anticipate some of the further consequences of withdrawing an oral combination,” she told the
committee prior to voting. She added that there should be further clarification on the box warning to prevent such use of
the IV drug.
Jane Axelrad, associate director for policy at FDA’s drug center, noted that the oral form of this drug had been
removed from the market and said it is “not appropriate” to change the label of the IV drug.
John DiGiovanna, staff clinician for the dermatology branch of the Center for Cancer Research, also asked if there is
a way to provide individual patient access to a listed drug in unusual cases without opening the drug up to being widely
bulk prepared and sold.
Axelrad said that if a physician thinks the drug should be used for an individual patient, they can make a case through
FDA’s IND process, pointing out that the agency made the application process much easier in the past month.
FDA earlier this month put out a simplified application that the agency says will take physicians 45 minutes as opposed to
days to fill out when requesting experimental drugs for patients under the agency’s expanded access program. The move is part
of an effort to make it easier for physicians to use the IND process, originally designed for manufacturers to begin human
testing of experimental drugs, to obtain experimental drugs for individual patients. FDA said in draft guidance issued Feb. 4 that
the new form offers an “important tool” for physicians who treat patients with serious or immediately life-threatening diseases
or conditions for which there are no comparable alternative treatments. The original application, as designed for drug companies, called for 26 separate types of information and seven attachments. — Erin Durkin
FDA Can’t Track Whether Pharmacists Compound For Specific Uses
It would be tough for FDA to track whether a traditional compounding facility, which is not required to register with the
agency, is limiting its compounding of drugs to specific indications, said an FDA official at a Pharmacy Compounding Advisory
Committee meeting Tuesday (Feb. 24). The comment came up as the committee weighed whether to add six drugs to a list of
bulk drugs that, when compounded by a traditional compounder, would be exempted from key FDA drug law requirements.
The committee followed FDA’s advice, voting to add to the list four drugs — thymol iodide, squaric acid dibutyl,
diphenylcyclopropenone and cantharidin — and voting against inclusion of silver protein mild and piracetam. Drugs that
appear on the list are exempted from sections of the law covering current good manufacturing practice, labeling of drugs
with adequate directions for use, and new drug application approval requirements.
In a briefing document for the meeting, the agency said over 2,500 substances were nominated for inclusion on the
list, but many of those nominations were for a substance that was the subject of a USP monograph or component of an
FDA-approved drug. The committee only weighed in on the six drugs presented by FDA.
During discussions around the drugs’ inclusion on the list, Jane Axelrad, associate director for policy at the Center
for Drug Evaluation and Research, told the committee that decisions about whether to add drugs cannot rest on the idea
that the FDA would be tracking whether the pharmacies were compounding drugs for particular indications, saying “it
would be hard to police that.”
“I did not want people to place a weight on the fact that we would be able to go out there and police,” said
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FDA Week - www.InsideHealthPolicy.com - February 27, 2015
Axelrad. “That being said, if the committee decided that they wanted to limit these [drugs] in some ways, we could
explore how one would do that.”
Katherine Pham, a Neonatal Intensive Care Unit Pharmacy specialist at the Children’s National Medical Center,
agreed, saying there was only so much FDA can do in terms of enforcement once a drug is on the bulk compounding list,
adding that prescriptions that get sent to the pharmacies don’t necessarily have the diagnoses noted.
“Where we typically see more direct diagnosis is the third party payer, if they are going to cover the compounds,”
said Pham, adding that prior authorizations sometimes can ask for that information.
But Padma Gulur, a professor whose expertise is in anesthesiology at University of California, Irvine, expressed
concerns because the committee makes its evaluation based on safety and effectiveness data for particular indications,
and once it’s on the list it can be used for anything. “I must express my discomfort in the process,” she said.
Jurgen Venitz, an associated professor with an expertise in pharmacology at the Virginia Commonwealth University,
suggested that at future meetings the committee discuss not only how drugs can get on the list, but also how they can be
reviewed and potentially removed. — Erin Durkin
FDA Delays Meeting On Second Biosimilar, Cites Pending Data Requests
FDA has postponed an advisory committee meeting that was slated for March 17 on a second biosimilar application
— Hospira and Celltrion’s proposed biosimilar for Janssen Biotech Inc.’s Remicade — citing pending requests for more
information from the application’s sponsor. The agency said it will announce a new meeting date in a future Federal
Register notice.
The delay comes after Hospira announced last week that data from the Hungarian National Registry showed that 90
patients treated with the infliximab biosimilar, Inflectra, had comparable responses to that expected with the reference
product. “These data add to the body of evidence supporting Inflectra’s use in inflammatory bowel disease (IBD),” states
a Feb. 19 press release.
The March 17 Arthritis Advisory Committee conversation likely would have focused on Celltrion’s data because the
infliximab biosimilar product is a much more complex molecule than Sandoz’s filgrastim — which was the first
biosimilar application to be reviewed by an FDA advisory panel — and with which there is considerably less European
marketing experience, according to an industry expert. — Erin Durkin
Hamburg To Appear At House Appropriations Budget Hearing Next Week
House appropriators plan to hold a hearing on FDA’s budget next Wednesday (March 4), with FDA Commissioner
Margaret Hamburg slated as one of the witnesses. The other two witnesses include FDA budget chief William Tootle and
the HHS budget shop’s deputy assistant secretary, Norris Cochran.
Sources told IHP that Senate appropriators are planning a similar hearing the following week, adding that Hamburg
is also expected to testify.
The president’s 2016 budget seeks a $425 million increase in FDA funding to beef up food safety, combat
antibiotic resistance, address the safety of compounded drugs, improve medical product review and inspections, and
promote the development of reliable molecular and genetic diagnostics in line with the administration’s Precision
Medicine Initiative. — Erin Durkin
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AMA: FDA Should Have Authority Over ‘Black-Box’ Tests, But Not All LDTs
American Medical Association this week weighed in on how it would like to see House lawmakers tackle laboratorydeveloped tests in their evolving 21st Century Cures bill: The physicians group says only a small subset of complex
genetic and genomic tests should be subject to FDA regulation and otherwise opposes the LDT framework developed by
the agency last year. The group urges lawmakers to only give FDA authority over tests for which incorrect results could
harm patients and for which test methodologies are not transparent.
Energy and Commerce Chair Fred Upton (R-MI) floated a draft Cures bill in late January that left blank a section on
the hot-button issue of how the government should regulated LDTs. Upton recently indicated he plans to unveil an
updated draft in the next week or so, and it remains unclear if that version will fill in the “Modernizing Regulation of
Diagnostics” placeholder section in the earlier draft.
“The AMA agrees that a small subset of complex genetic/genomic tests, e.g., those that use proprietary and nontransparent algorithms that do not lend themselves to review and refinement by laboratory physicians and professionals, should be subject to oversight, potentially by the FDA,” the group sates in its letter to E&C lawmakers this
week.
But the group continues to say that FDA’s proposed framework goes beyond “black-box” tests and subjects a massive
number of LDT services to “costly and burdensome requirements.”
FDA laid out its proposal to regulate LDTs in a draft guidance last year that calls for such tests to be regulated under
the agency’s medical device risk classification system. The proposal caused a firestorm of controversy, with laboratory
stakeholders charging the move would be burdensome and limit patient access to such tests. The American Clinical
Laboratory Association argues that lab tests should generally be regulated by CMS.
AMA also would like to see CMS play a large role. The group writes to lawmakers that it agrees there is a need to
modernize the existing regulatory framework for LDTs, but adds that such an effort should include modernizing CMS’
Clinical Laboratory Improvement Amendments (CLIA) by mandating third-party accreditation of all clinical laboratories
and increasing transparency of documentation of laboratory clinical and analytical validation.
But AMA does see a need for FDA to step in for a limited number of circumstances.
“In addition, the AMA urges Congress to confer the FDA with explicit authority to regulate direct-to-consumer
tests and testing services where incorrect results could cause harm to patients and the test methodology is not
transparent nor well understood (as in the case of tests that use complex and proprietary algorithms to produce
results),” says AMA.
“The AMA also supports streamlining the oversight for manufacturer commercial kits subject to FDA regulation, including greater flexibilities for manufacturers to incorporate modifications.”
The group additionally expressed interest in the White House’s Precision Medicine Initiative, saying the initiative is
not limited to personalized medicine “but contemplates novel research methods, uses of digital health, and is premised on
a level of data interoperability and databases that do not currently exist.”
AMA says that the final Cures legislation could have a significant impact on the feasibility of the Precision
Medicine Initiative. “For instance, lack of interoperability will be a serious barrier to these efforts as already outlined
during a two day NIH meeting concerning the million genome project,” says the group. “In addition, FDA regulation of
digital health and laboratory developed testing services will have implications for the million genome project’s use of
such tools to advance medical knowledge and patient engagement.”
As part of its effort to limit FDA’s role over LDTs, AMA also signed a letter to FDA Commissioner Margaret
Hamburg in November along with several other groups pushing for the agency to use the formal regulatory process
instead of issuing a draft guidance document to assert its jurisdiction over such tests.
“The draft guidance documents conflict with existing regulations and would impose substantial new requirements on
clinical laboratories, hospitals, physicians, and other health care providers without complying with notice and comment
rulemaking as required under the Administrative Procedures Act (APA),” stated the November letter.
Upton’s draft Cures bill, in addition to leaving the LDT section blank, also left blank a section on precision medicine.
“Following FDA’s proposed guidance altering the regulatory landscape for the review and oversight of laboratory
developed tests, a broader conversation about the need to modernize the regulation of diagnostics has reached a fever
pitch,” says a white paper released with the draft legislation. “The committee is encouraged by stakeholder efforts to
build consensus around what a modern framework should look like and is working toward the inclusion of such a proposal.”
The head of the Advanced Medical Technology Association’s diagnostic arm recently said it was unclear whether the
House would ultimately broach FDA’s role in LDTs as part of its Cures bill, telling Inside Health Policy that lawmakers
could end up sidestepping the issue if key stakeholders do not engage in discussions. — Erin Durkin
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FDA Week - www.InsideHealthPolicy.com - February 27, 2015