Rare disease patients often have a much harder time getting access to experimental drugs through compassionate use programs compared to patients with more common diseases, such as cancer, according to a July 14 Kaiser Health News report.

Following pressure from individual patients and groups, Biogen (NASDAQ:BIIB) has agreed to start a compassionate use program for its experimental ALS (amyotrophic lateral sclerosis) drug tofersen.

In remarks at the daily Coronavirus Task Force press briefing, FDA Commissioner Stephen Hahn pointed out that the drug, remdesivir, is available under compassionate use in addition to the ongoing clinical trials being run by Gilead Sciences and the NIH.

Over the past few months, about 30 people who suffer from Lambert-Eaton myasthenic syndrome, or LEMS, have switched back to the older treatment, known as Ruzurgi. For more than two decades, a small, family-run company called Jacobus Pharmaceuticals made its drug available on a compassionate use basis to a few hundred people while pursuing formal FDA approval.

The controversy over Novartis’ ultra-pricey gene therapy Zolgensma has intensified after it allegedly refused to supply the one-off therapy to a sick toddler in Belgium on compassionate grounds.

FDA will announce at the upcoming ASCO meeting the launch of Project Facilitate, a concierge service to help guide physicians through the process of applying for expanded access, also called compassionate use access.

The Right to Try bill signed into law by President Trump on May 30th this year allows terminally ill patients residing in the U.S. to gain access to medicines that have passed Phase 1 of the FDA approval process but have not established efficacy in post Phase 1 clinical trial testing. Prior to Trump’s signing of the legislation 38 states had already enacted Right to Try laws.

The right-to-try legislation guaranteeing that the FDA cannot deny patients the right to try experimental drugs was both championed and criticized by many groups. The conservative/libertarian think tank, Goldwater Institute, has been in the forefront of those promoting the legislation. The institute drafted model legislation upon which 40 state right-to-try laws and the federal law is based. Christina Sandefur of the Goldwater Institute took the time to provide answers to our questions laying out the Institute's stance on the relevant issues.