To Help the Terminally Ill, the FDA Must Deregulate

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In a recent press statement, Food and Drug Administration Commissioner Scott Gottlieb announced plans to improve and broaden FDA’s Expanded Access Program. Sometimes called the Compassionate Use Program, the Expanded Access Program provides terminally ill patients with access to experimental medications before they are fully approved. xyz

Current proposed changes to the program include streamlining the submission process that physicians undergo to provide required documentation and allowing individuals (instead of the entire board) to approve treatment requests. The agency also previously commissioned an independent review board to assess aspects of the program needing improvement.

The program has had recent success in expanding access. From 2005 to 2014, the FDA provided approximately 9,000 patients with access to experimental drugs. It has granted access to an equal number of patients in the last five years.

Although expansion has improved and the FDA’s commitment to helping the terminally ill is praiseworthy, current access levels are a far cry from what is needed.

According to the Pew Research Center, nearly 42 percent of Americans have a friend or relative with a terminal illness or who is in a coma as of 2006. In her book The Right To Try, Darcy Olson notes over 25,000 patients with terminal cancer die each year while waiting for the FDA to approve potentially life-saving medication.

When we consider that the FDA has offered similar programs since the 1970s, and the shortcomings of its current program, it’s clear another way to access experimental medication is needed. Fortunately, there is one.

Last May, President Trump signed right-to-try legislation into law. Right-to-try laws allow patients with terminal illnesses to access experimental medication with only the approval of their physician and the drug provider. With limited treatments and time, cutting additional barriers to access (even streamlined ones) is critical.

Shockingly, no one has used the right-to-try process. Why? The most likely reason is the high cost of administering treatment caused by excessive regulation.

Even though drugs accessed through right-to-try are only required to pass the first phase of the FDA’s approval process, regulations dictating research, development, and clinical-trial standards can cost drug companies between $50 million and $840 million. Further, many insurance companies are unable or unwilling to cover unapproved drugs, making potentially life-saving drugs unaffordable for most patients.

In June, Brainstorm Cell Therapeutics Inc. attempted to become the first drug provider to offer terminally ill patients access to experimental treatment for amyotrophic lateral sclerosis (commonly shortened to ALS) through right-to-try legislation. However, even by charging $300,000 per treatment, the company was unable to cover the costs of offering treatment. Its treatment, name NurOwn, will likely be FDA approved in 2019 or 2020. This will be too late for many patients currently suffering from ALS.

To give the terminally ill the best chance to prolong their life, greater access to experimental medication is desperately needed. The Expanded Access Program, although helpful, falls short. Right-to-try can provide a much-needed alternative, and less bureaucratic, method. However, it will never reach its potential without deregulation.