Trump, Biotech, and the FDA Drug Approval Process

During his Joint Address to Congress on the evening of February 28, President Donald Trump denounced the decades-old prescription drug approval process, pledging to “slash the restraints” of the current regulatory landscape at the US Food and Drug Administration (“FDA”).  “Our slow and burdensome approval process at the Food and Drug Administration keeps too many advances…from reaching those in need,” President Trump said, giving a nod to his guest, 20-year-old Megan Crowley. Crowley, a college student who was diagnosed with a rare and typically terminal ailment – Pompe Disease – at 15 months old, was not expected to live past age 5. Since her diagnosis, Crowley’s father went on to launch Amicus Therapeutics, a biotechnology company that aims to be the frontrunner of advancing therapies for rare and devastating diseases.  

To many, the President’s harsh opinion of the FDA drug approval process was seen as misleading. Not only is the drug review process in the United States recognized worldwide as the gold standard, FDA is the fastest regulatory agency in the world. Today, the average total review time of a single drug is 8.5 months, down from an average of 30 months in the 1980s.

While most drug and biologic companies must successfully maneuver their compounds through three phases of clinical trials, proving not only safety but efficacy (via two “well-controlled and adequate studies”), there are several other expedited processes that may be utilized to obtain agency approval, at least preliminarily. Breakthrough, priority review, fast track, and accelerated approvals are all programs created to speed up the market availability.

Perhaps most misleading about Mr. Trump’s statements was the approval process for rare diseases like that of Megan. The term “Rare Diseases” is a term of art, defined as afflicting less that 200,000 people nationally. Drug manufacturers of potential treatments of rare diseases, like Pompe, may participate an expedited approval program depending on the seriousness of the ailment. ON the other hand, they may participate in the Orphan Drug Designation process, by which they are incentivized by the FDA to increase research and development on novel drugs via seven years of patent exclusivity, tax credits, and expanded access to Investigational New Drugs (IND). In other words, those who have rare diseases, to the extent to which therapies are being developed, have access to treatment faster than average.

Moreover, many biotech and pharmaceutical companies are skeptical of the deregulation of the industry. “People often argue that the FDA is too restrictive, [but] [w]e have the sense that the balance is pretty right … you have to have a well-characterized risk/benefit profile,” said Roger Perlmutter, head of Research and Development at pharmaceutical heavy hitter Merck. Similarly, Alnylam Pharma CEO John Maragarone stated what, to some, may be obvious: while deregulation seems like a good idea, “payors are looking for evidence of value.” As it is, insurance companies have already shunned some approved treatments that have not demonstrated strong effectiveness.

In 1962, in response to several children dying from ingesting elixir, Congress enacted the Drug Efficacy Amendment to Food, Drug and Cosmetic Act of 1938, explicitly requiring the pre-market approval process to include not only safety profiles, but proof of effectiveness by a showing of substantial evidence. Deviation from this would deem the product false and misleading if placed on the market, and likely open the manufacturer up to liability.