Life Science Compliance Update

July 14, 2017

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

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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

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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.

April 21, 2017

Dr. Stossel Corrects a Common Misconception

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Thomas P. Stossel, MD, MD (Hon), is a visiting scholar at the American Enterprise Institute and professor emeritus at Harvard Medical School, who has recently published several articles on how to remove barriers to medical innovation, and how medical innovation actually happens. This article highlights the impressive research by Dr. Stossel, supporting his position that private investment does much more to push the progress of medicine along than people think.

One article, published in the Wall Street Journal on January 5, 2017, addressed the assumption that “the root of all medical innovation is university research, primarily funded by federal grants.” He noted that the assumption is incorrect and that it is the “private economy, not the government,” that “actually discovers and develops most of the insights and products that advance health.”

The article opens with complimenting Congress for passing the 21st Century Cures Act, claiming that it “will promote medical innovation,” while at the same time telling readers to be “wary, however, of the $4 billion budget boost that the law gives to the National Institutes of Health.” In addition to his Wall Street Journal article, Dr. Stossel wrote a more in-depth article in National Affairs, arguing the same points, with more research and information embedded into the article. 

There were few findings in medical science that could significantly improve health until the late 19th and early 20th centuries, with innovation primarily coming from “physicians in universities and research institutes that were supported by philanthropy.” Dr. Stossel notes, however, that things changed after World War II when the National Institutes of Health became the major backer of medical research, changing incentives. Universities that previously lacked research operations started to develop them, and existing programs were largely expanded. As noted in Dr. Stossel’s article in National Affairs, “for decades, Congress allocated generous and growing funds to the NIH that enabled it to provide many research grants to universities. As a result, universities expanded their laboratory facilities and research faculties — and the government-academic biomedical complex, or GABC, was born.”

Since that time, improvements in health have rapidly occurred. Also during that period, funding for the National Institutes of Health has lagged behind the growth of an aging population in need of medical innovation while private investment in medicine has largely kept pace with the aging population and “is the principal engine for advancement.”

In his National Affairs article, Dr. Stossel discussed research papers submitted for publication, noting:

Although revered by academics as a quality filter, “peer review” of research papers submitted for publication (and of grants for research funding) is a flawed enterprise. As scientific journals found success in providing researchers the priority and credit they were looking for, the volume of submissions began to exceed the supply of journals’ publication space. The practice of peer review — having selected experts render opinions regarding the quality of articles submitted to journals — was designed to solve that problem. Today, electronic publication has eliminated the space problem, but a prestige hierarchy of journals has replaced it with a false scarcity. Researchers covet attention in the most prestigious journals, and the high-profile journals sustain their elevated status by arbitrarily rejecting the majority of articles submitted to them. The monopoly power of these journals, fueled by researchers’ vanity, allows indifferent editors to delay decisions about whether to publish research articles until dueling authors and reviewers come to a resolution. The referees of these disputes provide a quality of service that would be expected from the nature of the reviewers: anonymous, unpaid cronies or competitors of a paper’s authors. As a result, research data can languish in obscurity for months or years while authors work their way down the prestige pecking order and finally obtain a place to publish.

According to Dr. Stossel, more than 80% of new drug approvals originate from work solely performed in private companies and such drug approvals come on average 16 years after the beginning of clinical trials, which typically cost $2.5 billion from start to finish. Therefore, it appears even if academics and NIH really wanted to create a new drug, economic reality would get in the way.

The National Affairs article notes that, “achieving innovation requires wanting to innovate more than trying to impress reviewers of research papers or grant applications. It involves trial-and-error efforts that academic-review committees dismiss as “fishing expeditions” and that violate the scholarly premium on ‘hypothesis-driven’ studies. Success in academe also demands sticking to one’s research ‘brand.’ By contrast, innovation usually requires shifting gears to employ different technologies and experimental approaches. Such inconsistency reliably leads grant-application reviewers to discount an applicant’s qualifications.”

Dr. Stossel closes his Wall Street Journal article by stating:

Despite its exaggerated role, basic research in universities does advance human knowledge, train scientists, and contribute to medical advances—albeit uncommonly and inefficiently. But the system is unsustainable. A better approach would be to encourage academics to join with industry, where the financial resources and drive to innovate reside. Unfortunately, the biomedical complex demonizes corporations. If academic institutions stopped demeaning the activities needed to develop medical products, industry might take a greater interest in supporting their research.

Great advances in health care have been made, but there are still important challenges, from obesity to dementia. One step toward addressing them would be for Washington to adopt the right approach to medical innovation—and to stop simply throwing money at the current inefficient system.

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