When it comes to reviewing drugs, the Food and Drug Administration’s job seems straightforward: Make sure a drug is safe. Then make sure that it actually works and does what it says it’s supposed to.
It’s an essential mission that this deeply dysfunctional organization appears to be having trouble fulfilling.
FDA officials should be held accountable for being hostile and unprofessional toward private pharmaceutical companies who are trying to help the world.
I have been working in investigational drug development for almost two decades. As a politically conservative medical scientist at the FDA — a supreme rarity, I assure you — who has worked as a medical officer and senior medical analyst at the FDA, and as a former investigational medicine research scientist at Pfizer, I haven’t always agreed with regulatory decisions made by the FDA.
Lately though, in addition to disagreeing, I now fail to understand the FDA’s decisions. One example is the approval of flibanserin (Addyi), designed to improve low libido in women. The drug worked in only 8 to 13 percent of the women in which it was tested. On top of the pathetically poor efficacy, flibanserin can have life-threatening interactions with recreational alcohol consumption and some of the most commonly prescribed antibiotics and antifungals on the market.
The approval of Eteplirsen (Exondys 51), which is supposed to treat Duchenne muscular dystrophy, also sets a terrible precedence, and seems to defy common sense. Eteplirsen was tested in a mere 12 individuals, with no control group — a highly unusual situation when it comes to investigational medicine evaluation. In addition, the drug offered a dystrophin biomarker increase of less than 1 percent overall — which as you can probably guess, is obviously neither clinically nor scientifically meaningful.
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Amid calls to reform the agency, I offer 10 of my many, many suggestions for fixing the FDA:
1) Implement truth in prescription drug labeling.
Sure, paying only $4 for your prescription at Walgreens or CVS seems great. But are you comfortable knowing many of the pills you take come from “sweatshop” countries where falsified records are a serious problem, along with unclean manufacturing environments, and very poor quality control? Many generic drugs are imported from India, China, Mexico, and Taiwan. Consumer prescriptions should be labeled with the country of origin, and patients should have the option to request their prescription drugs are made in the U.S. or a non-sweatshop country such as Japan, Germany, France, or Israel.
2) Fix the broken hiring process.
The FDA’s inability to hire enough qualified staff has been an unaddressed problem for well over a decade. The FDA has a backlog of nearly 4,000 applications to produce money-saving generic drugs, because it doesn’t have the staff to review them.
The hiring system through USAjobs.com is also broken. All applications have to initially go through the website, but low-level human resources managers with little or no scientific training are the ones reviewing the applications of highly specialized scientists. It is impossible for our best and brightest to directly call about a job because there is no publicly available list of FDA recruiters.
3) Negotiate disagreements between reviewers and management.
Reviewers conduct the hands-on component of drug analyses. They sometimes have to summarize tens of thousands pages of data for others at the FDA — including management. But if management (which did not conduct the review) disagrees with the reviewer, the reviewer has no recourse but to obey his supervisor. That reviewer should have the option to contact an outside group of scientists — possibly a Congress-appointed committee. And the facts of such disagreements (which occurred with both flibanserin and eteplirsen) should be made public.
4) Better managed drug pricing.
The FDA currently has no say when it comes to the actual price of drugs. Congress should create an offshoot of the FDA that independently reviews and recommends drugs within the same class, based on their safety, efficacy, and overall merit and benefit, similar to Europe’s model.
5) Stop abusing startups and small drug companies.
The U.S. depends on private drug companies to invent and develop new drugs for existing and emerging diseases. Yet smaller drug companies that need assistance in drug development are often demeaned throughout the process. FDA officials should be held accountable for being hostile and unprofessional toward private pharmaceutical companies who are trying to help the world through legitimate development of pharmacology.
6) Improve workforce quality.
Most FDA staffers and supervisors are highly qualified, dedicated individuals from dozens of different disciplines. But the FDA would also benefit from a good sweep. Some senior employees at the FDA have personal and petty agendas, pitting FDA workers against each other — but are not able to be fired because they are considered “permanent” or “career” employees.
Unfortunately, firing federal permanent employees is difficult compared to any private sector job — FDA employees literally have to be caught breaking the law for there to be any repercussions.
7) Improve the quality of advisory committees.
To be part of an FDA advisory committee, an individual must not have accepted any research funding or speaker’s fees from a drug company or have any conflicts with the drugs being reviewed. Because the best and the brightest scientists are usually doing original or novel research, often funded by industry, this eliminates many highly qualified individuals. As a result, today’s advisory committees are made up of retired physicians and scientists who are seldom leaders in their fields, or up-to-date on the latest clinical developments.
8) Address dangers of “right to try” legislation.
As a conservative, I am all for fewer laws and less government intervention. That said, I am not ready to entrust the average person to make informed decisions regarding investigational medicine, pharmacogenetics, and long-term drug safety of new, unapproved molecules. Americans disheartened with lengthy drug approval times have taken matters into their own hands, and passed right-to-try legislation — individuals can now completely circumvent the FDA in 31 states and get access to unapproved investigational medicine. Most people don’t know that more than 95 percent of investigational compounds fail in Phase I clinical trials and more than 80 percent fail in Phase II trials. Few if any investigational compounds have antidotes and their adverse events have the potential to be permanent.
9) Improve the collection and monitoring of FAERS/MedWatch adverse event reports.
The FDA is doing a very poor job of compiling adverse events and reporting them to health care professionals and the public. Adverse event tracking through the FDA Adverse Event Reporting System (FAERS) or MedWatch has the power to provide valuable real-time tracking of new drug safety reports that may not have emerged during clinical trials. Unfortunately, the time-intensive process of reporting an adverse event to a drug company or the FDA is neither a requirement nor a goal for prescribers, physicians, and pharmacists who are already burdened with overwhelming amounts of paperwork for insurance companies’ EMR requirements and Obamacare requirements.
10) Alert consumers about the abuses and dangers of mail-order pharmacies.
Many patients are now required to get their medications via mail order. Almost all mail-order drugs come from the aforementioned “sweatshop” countries. Even more problematic, it is difficult to store mail-order drugs at the temperatures the FDA requires. Non-refrigerated prescription drugs are required to be kept between 68 and 78 degrees Fahrenheit. Stored outside of this range, the drugs may become inactive. Remember that the next time your mail-ordered medication is left in your mailbox during a heat wave or the dead of winter.
Dr. David Gortler is a professor of pharmacology at Georgetown University and a former FDA medical officer, now a pharmacology expert and FDA Policy expert with FormerFDA.com. He was also the FDA/health care policy adviser for the 2016 Ted Cruz presidential campaign.