Life Science Compliance Update

August 03, 2017

US Senate Passes FDA User Fees, Right to Try and Opioid Legislation

Senate Floor
After many months of debate, both chambers of Congress passed the FDA User Fee Package. This comes after the Senate passed its bill (S. 934) which cleared the Senate HELP Committee in May on a bipartisan basis. The House passed its bill on July 13 (H.R. 2430). The FDA Reauthorization Act (FDARA) renews and enhances the FDA drug, medical-device, biosimilar, and generic-drug user-fee provisions. The bill was uniquely tied to “Right to Try” legislation, and the Senate also passed bipartisan legislation on the opioid epidemic.

Senate FDARA Bill

The politics of the bill were intricately locked in with another member of the Senate’s own legislation. Majority Leader Mitch McConnell (R-KY) committed to taking up an amended version of Sen. Johnson’s right-to-try legislation to the floor in a separate vote, which cleared the way for Senate consideration of the user fee package.

FDA Commissioner Scott Gottlieb informed agency employees via email on July 24th that he would not be sending out any layoff notices to user fee-funded staff “unless and until September 30 had passed without reauthorization.” The publicizing of this policy decision by the Commissioner may have been intended to signal to the Senate that the sky is not falling (yet), but that they need to get to work.

"This program is critical to speeding up the drug approval process, and that's important for everyone frustrated by the time and cost of bringing life-saving drugs to market," McConnell said in floor remarks on FDARA. "Without it, the important work of ensuring that drugs and devices are safe and effective would come to a screeching halt."

User Fees

Specifically, the FDA Reauthorization Act of 2017 addresses several aspects of the FDA’s user-fee provisions:

Prescription Drug User Fee Amendments of 2017

The bill amends the Federal Food, Drug, and Cosmetic Act to extend through FY2022 and revise Food and Drug Administration user fees for new drug applications. User fees are eliminated for supplements to new drug applications and drug manufacturing facilities.

Medical Device User Fee Amendments of 2017

The bill extends through FY2022 and revises FDA user fees for medical devices. A user fee is established for requests to classify devices that are not substantially equivalent to marketed devices. The FDA is no longer granted the discretion to waive or reduce fees in the interest of public health. The FDA must establish a pilot program to accredit testing laboratories to determine whether medical devices conform to performance standards. The bill also revises the types of medical devices that the FDA may accredit third parties to review.

Generic Drug User Fee Amendments of 2017

The bill extends through FY2022 and revises FDA user fees for generic drugs. User fees are eliminated for supplements to generic drug applications. An annual fee is assessed on holders of approved generic drug applications.

Biosimilar User Fee Amendments of 2017

The bill extends through FY2022 and revises FDA user fees for biosimilars. (Biosimilars are biological products approved by the FDA based on their similarity to an already-approved biological product.) User fees are eliminated for supplements to biosimilar applications and biosimilar manufacturing facilities. An annual fee is assessed on holders of approved applications for biosimilars. The bill sets the annual amount of revenue that must be generated by fees. The bill also extends through FY2022 programs and policies including Critical Path Public-Private Partnerships and support for development of medical products for rare conditions.

Industry Supported

The House and Senate language is very similar, and the House bill was widely supported by industry when it passed in July: “This legislation ensures that the FDA continues to have the resources necessary to carry out its critical human drug review programs, while advancing important patient-centered policies that will help streamline the clinical trial process—the most time-consuming, complex, and expensive step in the drug development process. It also takes important steps to advance patient involvement in drug development and bring patient perspectives more clearly to bear on regulatory decisions. The reauthorization of the biosimilars user fee program, including steps to improve communication throughout application review, will move the needle on getting more affordable biosimilar products to patients in a timely way, once the IP protection expires for new biologics,” BIO President and CEO James C. Greenwood said in a statement.

PhRMA president and CEO Stephen J. Ubl issued the following statement: “Timely reauthorization of the Prescription Drug, Biosimilar and Generic Drug User Fee Acts is crucial to patients in need of life-saving treatments and enhancing the competitive market in biopharmaceutical innovation. The House took an important step in reauthorizing the user fee programs and we look forward to swift action in the Senate on behalf of America’s patients.”

Right To Try

Sen. Ron Johnson's Trickett Wendler, Frank Mongiello and Jordan McLinn Right to Try Act of 2017 (S. 204 (115)) authorizes the use of unapproved medicines by patients diagnosed with a life-threatening illness as long as the drugs in question have already been tested in the first phase of human clinical trials and are continuing on in further FDA-overseen research. Patients must have exhausted other treatment options and be unable to participate in ongoing clinical trials. Reps. Andy Biggs and Brian Fitzpatrick have a similar bill pending in the House.

Johnson's measure aroused concern from the drug industry, public health advocates and some at the FDA. But Johnson threatened to hold up the reauthorization of the FDA user fee programs if he did not get a vote on the bill. A compromise reached by Senate leadership and leaders of the Senate HELP Committee gave Johnson a vote on a revised version of his bill, separate from the FDA user fee bill. The compromise included requiring the FDA to receive reports of safety events that occur in right-to- try situations and forbids patients from being charged more than the cost of production for the medicines.


Additionally, lawmakers passed a bipartisan opioids bill (S. 581), from Sen. Joe Manchin (D-WV), requiring HHS to develop standards for hospitals and physicians to denote a patient's history of opioid addiction in medical records. The legislation directs the FDA to develop standards that: (1) consider the potential for addiction relapse or overdose death if a patient recovering from addiction is prescribed opioids; (2) require that a history of opioid addiction be displayed in a manner " similar to other potentially lethal medical concerns" such as drug allergies; and (3) require medical professionals have access to the opioid information to ensure they can prescribe medically appropriate medication.

July 14, 2017

PhRMA Report Shows More than 240 Immuno-Oncology Treatments in Development

ACS CAN logoACS CAN logo

In early June 2017, the Pharmaceutical Research and Manufacturers of America (PhRMA) – in partnership with the American Cancer Society Cancer Action Network (ACS CAN) – released a report that found there are over 240 immuno-oncology medicines and vaccines currently in development.

Immuno-oncology treatments are found through research into the role of the body’s immune system in fighting cancer. New immuno-oncology treatment options are allowing the patient’s own immune system to fight cancer similar to the way it fights disease-causing viruses and bacteria. The treatments can help the patient’s own immune system to work against cancer, with the potential for lasting results.

According to the report, there is no single accepted definition of immuno-oncology. However, this report includes many of the most recognized classes: adoptive cell therapies (including CAR-T therapy), bi-specific antibodies, cytokines, immune checkpoint modulators, oncolytic virus therapies, and vaccines. Former President Jimmy Carter was a notable recipient to immunotherapy treatment in his battle to fight metastatic melanoma.

In addition to the medicines currently in development, researchers are working to understand the full potential of each individual medicine, seeking approval for new indications for currently-existing immunotherapies, as well as new uses in combination with other cancer medicines.

“Cancer continues to be one of the most complex and vexing diseases of our time and it will impact an estimated 1.6 million Americans who will be diagnosed this year,” said Stephen J. Ubl, president and chief executive officer of PhRMA. “As our understanding of the root causes of cancer grows, we are expanding the types of treatments we are able to bring to patients. The idea of harnessing the body’s own immune system to fight cancer is not new but recent breakthroughs are making it a reality, bringing hope to patients.”

“We are at a moment of tremendous opportunity when it comes to developing therapies that can address even the most vexing cancers we see today,” said Chris Hansen, president of ACS CAN. “To fully leverage our potential to reduce suffering and death from cancer, robust and sustained federal investment in basic research is critical to provide the necessary building blocks that together with privately-funded innovation lead to advances in immunotherapy and other targeted treatments.”

In 2016, then-president Barack Obama announced the creation of the Cancer Moonshot Initiative. Immunotherapy research was identified as a priority and a blue-ribbon panel of experts called for the creation of both adult and childhood immunotherapy clinical trial networks designed with the needs of cancer immunotherapy research in mind. As part of the Moonshot initiative, the American Cancer Society – the largest nongovernmental funder of cancer research in the United States – also committed to a goal of doubling its own research budget to help reach the national goal of accelerating progress.

Increases in federal research funding, like those advocated for by ACS CAN, enable continued growth in our understanding of cancer immunotherapy, and allows important investments in research infrastructure such as the Moonshot-recommended immunotherapy trial network. Those federal investments in basic research provide the necessary building blocks to fuel further privately-funded innovation.

The report was released as part of PhRMA’s GOBOLDY campaign, which showcases the bold advancements the industry is making in tackling some of the most complex and devastating health conditions through innovative research. While the biopharmaceutical industry is a leader in the development of innovative treatments, it also plays a critical role along with others in the health care ecosystem to help bring new medicines to patients.

July 13, 2017

FDA Drug Approvals on an Uptick


Already in 2017, the FDA has approved a number of new drugs, which Regulatory Focus points out as a trend away from the low number of new drugs approved in 2016. They caution, however, it is unlikely to match the approval highs from 2014 and 2015. The pace for 2017 may not continue at this level and we could see an average, or perhaps slightly above average year for approvals.

2017 vs. Past

It has been reported that coming into 2017, the environment was looking better for approvals. According to the FDA’s Office of New Drugs, 36 new molecular entity NDAs were received by FDA through mid-December 2016, already beating the average number of 35 for the past decade.

The FDA, which approved 22 treatments last year, has given its approval to 21 drugs so far in 2017 including 3 in May. Key approvals so far in 2017 include Regeneron/Sanofi’s Kevzara (rheumatoid arthritis), Roche’s multiple sclerosis treatment, Ocrevus, Regeneron and Sanofi’s eczema treatment, Dupixent, Tesaro’s PARP inhibitor, Zejula, and BioMarin’s Brineura (treatment of a specific form of Batten disease) among others. Some of these drugs have blockbuster potential.

Why the low number in 2016?

John Jenkins, the now-retired director of FDA’s Office of New Drugs, offered the explanation that the lower number in 2016 may not be a clear signal that industry innovation is stalling.

Jenkins wrote: “For example, CDER approved five novel drugs in 2015 that had PDUFA goal dates in 2016. These early approvals benefited patients by making the drugs available sooner, but also decreased the total of novel drugs approved in 2016. Another factor was the number of Complete Responses (CR), which describe deficiencies in the application, precluding approval, with advice on what the sponsor needs to do for FDA to support resubmission of the application. CDER issued 14 CR letters for novel drugs in 2016, higher than in recent years.”

Jenkins also notes that the primary deficiency in several of the applications was the failure to comply with FDA’s current Good Manufacturing Practice (cGMPs) regulation, whereas by comparison, only four of the 47 new drug applications issued a CR from 2010 through 2015 included a failure to comply with cGMPs as the primary deficiency.

FDA Process

For more background information, it is important to understand how the FDA approves drugs and biological products. As the FDA states, some of these products are innovative new products that never before have been used in clinical practice. Others are the same as, or related to, previously approved products, and they will compete with those products in the marketplace.

Certain drugs are classified as new molecular entities (“NMEs”) for purposes of FDA review. Many of these products contain active moieties that have not been approved by FDA previously, either as a single ingredient drug or as part of a combination product; these products frequently provide important new therapies for patients.

Some drugs are characterized as NMEs for administrative purposes, but nonetheless contain active moieties that are closely related to active moieties in products that have previously been approved by FDA. For example, CDER classifies biological products submitted in an application under section 351(a) of the Public Health Service Act as NMEs for purposes of FDA review, regardless of whether the Agency previously has approved a related active moiety in a different product. FDA’s classification of a drug as an “NME” for review purposes is distinct from FDA’s determination of whether a drug product is a “new chemical entity” or “NCE” within the meaning of the Federal Food, Drug, and Cosmetic Act.


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