Helping Trump Prioritize His Clinical Trials Agenda

By Ed Miseta, Chief Editor, Clinical Leader

Scrolling through my Facebook and Twitter feed in the days immediately following the presidential election, you would think the world had just ended. I even read articles that seemed to be predicting doom and gloom for pharma and clinical research, even though the president-elect would not take office for more than two months. Perhaps it’s time for us all to relax, take a deep breath, and fight the urge to make wild assumptions that may never come to fruition.

For example, I read an article two days after the election that seemed to imply clinical research may be in danger under a Trump presidency. The missive noted the clinical research industry was bracing for the Trump impact. One interviewee stated there would likely be no impact to clinical research in the short term. But in the longer term, things would be a bit hazier. Why? Because the president-elect did not mention research funding during his campaign. There are countless topics Trump did not mention during his campaign. I would hope we don’t have folks in panic mode over those omissions as well.  

Still, the concerns are understandable. The next four years will certainly be a period of change. For many, change brings uncertainty and fear. But rather than speculate on what we may or may not see over the next four years, I would like to focus on what I hope to see, and what might be best for the industry. I hope we can start with the following:

1.  Ensure Passage of 21st Century Cures

The 21st Century Cures Act sailed through the House with overwhelming bipartisan support. It later stalled when it got to the Senate, where it currently resides. The bill, introduced in May 2015 by Rep. Fred Upton (R) of Michigan, seeks to increase funding for the National Institutes of Health (NIH) by $10 billion over five years and create a special fund for basic, translational, and clinical research at the NIH. It would also implement changes to the FDA approval process in an attempt to modernize the agency’s workflow.

Although a vote was expected in September 2016 when senators returned from summer break, no action has yet been taken. Some worry the window of opportunity may be slowly closing. This, despite Sen. Lamar Alexander (R) from Tennessee and chairman of the Senate health committee, calling it the most important legislation Congress could pass this year. The primary dispute at this point seems to be over funding for the bill. With the election now over, passage is still possible in the lame duck session. 

If passage does not occur, I hope the new administration will find a way to bring the two sides together and pass the bill. The FDA does need to update its approval process, and pharmaceutical research would make needed use of the funding.

Two efforts that are sure to have the support of the American people and also help advance cures for patients are President Obama’s precision medicine initiative and Vice President Joe Biden’s cancer research “moonshot” program. Unfortunately, funding for both initiatives is tied up in the 21st Century Cures Act. Insuring both efforts get the funding they need will also benefit researchers and, ultimately, patients.

2.  Get Involved In Patient Recruitment. Since the inception of clinical trials, I’m sure one of the greatest struggles for researchers has been patient recruitment. Little has changed. Today, even a disease as fatal as cancer still has just four percent of affected patients participating in trials. With the reputation of pharma still far from stellar and lingering distrust of the industry by the American public, clearly a much bigger effort is needed. Government involvement may be the answer. Public service announcements and other educational efforts can bring an understanding of clinical research to the masses. Hospitals, clinics, and physician offices should all be informing patients of the benefits of trials. Regulation making it easier for companies and sites to find needed patients is essential. Support from government could also make it easier for the industry to solve compatibility issues with EHRs and EMRs to further simplify patient compatibility with ongoing trials. 

3.  Finally, we need to have the right incentives in place to get pharma companies to invest in R&D. One effort that seems to be having the intended effect is the Rare Pediatric Disease Priority Review Voucher Program. Under Section 529 of the FD&C (Food, Drug, and Cosmetic) Act, FDA will award priority review vouchers to sponsors of rare pediatric disease product applications that meet certain criteria. The voucher can be redeemed to receive a priority review of a subsequent application for a different product. 

Although a government report released in March concluded it is too early to determine if the program is working, the FDA is now considering nixing the voucher program. The program expired on September 30, 2016, and there is talk of not renewing it. This is despite claims from drug companies and advocacy groups who say there is value in the program. FDA notes it does not see evidence the program increased development of drugs for rare pediatric diseases. While it’s true the vouchers, which can be sold by recipients for tens or hundreds of millions of dollars, will allow firms to fast track drugs that treat illnesses with already available treatments, a rare pediatric disease must be addressed in order to get the voucher.   

Although the bill has not been re-authorized by President Obama or congress, there are two bills circulating which could reauthorize the program. If one thing is clear, it’s that the FDA was never a fan of the programs and hopes to end it. A coming together of congress, the president, and the FDA is needed to find incentive programs that work and will bring needed medicines to patients. Incentives work, and if we do not pass this program, then let’s work together to design a better one.

Will a Trump administration do anything to help promote and fund clinical research? It is clearly too early to say. However, he has yet to call pharma an enemy. That is a step in the right direction. But for now we can set aside the panic and do what I do after every election: Hope for the best, deal with the results, and look forward to the next election. This has been a tough election cycle. Let’s band together and push for those changes that will best help pharma bring new medicines to patients in need.