Dying patients simply don’t have the luxury of time, and many American patients with deadly diseases are being prevented from trying drugs that might extend their lives.

David’s Story

Take, for example, the story of David Huntly, an avid athlete, who was diagnosed with amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s Disease.

ALS is a progressive neurodegenerative disease that causes muscle weakness, paralysis, and ultimately respiratory failure and death. After several years of battling ALS, David was wheelchair-bound and was able to communicate only through the assistance of text-to-speech software.

David, a scientist, became aware that a new drug, GM604, was in the second phase of clinical trials and therefore had not yet been approved by the Food and Drug Administration (FDA), the federal agency that regulates and authorizes the use of new pharmaceuticals after extensive clinical testing. David knew that ALS would take his life and was willing to try an experimental drug in the hope that it would slow the progression of his disease.

Unfortunately, David was unable to receive this drug (nor was it approved in time by the FDA). David lost his battle with ALS, dying on July 4.

Streamlining Is Needed Within the FDA

In recent years, pharmaceutical companies have been responsible for many cures and advances in treatment that have benefited patients’ lives. New cancer treatments, medical devices, and discoveries are being made at a rapid pace but are subject to the FDA’s lengthy approval process that has delayed the widespread deployment of new medications.

Regulation is important and necessary in order for the FDA to promote public safety, yet outdated FDA regulations hinder and slow the innovation process. Reducing the burdens imposed on inventors by the FDA (for instance, by streamlining drug approval processes without sacrificing safety) would speed the trial phase and allow more drugs to get to the market quickly, so that patients such as David would have the opportunity to pursue new treatments. In short, implementing creative ways to reform FDA regulations is a topic of major public policy concern.

FDA Reform Proposals

FDA legislative reform proposals have been put forth recently by lawmakers and several presidential candidates.

One is the 21st Century Cures Act, which was introduced by Rep. Fred Upton, R-Mich., on May 19. This 362-page bipartisan bill touches on reforms for the National Institutes of Health (NIH) and the FDA.

In general, the bill seeks to expedite prescription drug and medical device approvals by the FDA and create more research funding for the NIH.

More specifically, it includes “steps to streamline clinical trials; advance personalized medicine by encouraging greater use of drug development tools, such as biomarkers; and creat[es] incentives for developing drugs for uncommon but deadly diseases.”

The bill also calls for billions of dollars in support for NIH-specific biomedical research and FDA activities, plus the endowment of a multibillion-dollar long-term “NIH and Cures Innovation Fund” would support biomedical research, cures development, and funding for early career researchers.

On Oct. 22, Sen Ted Cruz, R-Texas, unveiled his FDA reform plan in a National Review article. The article proposes legislation that would “provide for reciprocal drug approval, so that cures and medical devices that are already approved in other countries can more expeditiously come to the U.S. It requires the FDA to respond the treatment sponsor within 30 days of its application. The legislation also ensures that the FDA is more accountable to congress, and ultimately the American people, so that the FDA’s risk-averse approach [to drug approval] does not stand in the way of medical advances.”

All of the proposed legislative initiatives center on increasing the efficiency of the FDA and promoting the faster introduction of valuable drugs.

While we want our food and drugs to be safe, we also want to encourage a process that would accelerate the approval time of these products and thus strengthen the incentives for innovators to invest in life-saving biopharmaceutical research. Public welfare and our economy would both be enhanced by legal reforms that accelerate the introduction of new and improved products and (more generally) reduced excessive barriers to private sector-driven improvements in the health care sector.

Minor changes such as these would lead to many patients, such as David, benefiting from the earlier introduction of innovative life-saving drugs.