The Struggle to Access Experimental Rare Disease Therapies Through Compassionate Use

Accessing treatments can be difficult, especially if one is impacted by a rare disease. In fact, only 5% of rare diseases have an FDA-approved treatment. Because of this, many patients have turned to clinical trials in order to receive care. Unfortunately, this is not an option for everyone. Many are turned away from clinical trials, as they do not meet enrollment requirements. For these people, compassionate use, also referred to as expanded access, is the only remaining option. While further research needs to be conducted on this topic, existing studies show that it is more difficult to utilize compassionate use for rare disease therapies than it is for treatments for more common conditions.

Compassionate Use for Rare Disease Patients

Treatment access is an issue within the rare disease community, and expanded access to experimental therapies is no exception. One reason behind this is pharmaceutical companies’ reluctance to provide patients outside of clinical trials their drug before the trials have finished. The belief is that allowing this access could put future FDA approval in jeopardy. But for patients who will die without treatment, the remaining research does not matter. In addition, research has demonstrated that allowing compassionate use does not put approval at risk.

Because there are already so many obstacles standing in the way of rare disease research – small patient populations, lack of funding, low interest, and a lack of knowledge are just a few – companies are much more cautious when approving expanded access. They’re already hesitant to conduct rare disease research at all, much less take action that could possibly harm their efforts.

In contrast, companies are much more willing to offer compassionate use for experimental treatments of common diseases. The leukemia drug Gleevec was administered to thousands before FDA approval, as were Videx for HIV and Iressa for common forms of lung cancer.

An FDA patient representative, Melissa Hogan, has pointed out that this may be due to the tight-knit communities that exist within the rare disease sphere, along with the staggering cost of treatments. Drug companies may not be able to provide their medication to many patients outside of the trial due to manufacturing restraints. Because rare disease patients have formed communities and advocacy groups, they often share information and treatment advice. Essentially, if one patient has received expanded access, others will most likely find out and request the same. Drug companies don’t necessarily want this.

Current Examples Within the Rare Disease Community

Many rare disease patients have finally been able to request compassionate use after clinical trials wrapped up for multiple drugs. Examples include Enspryng for neuromyelitis optica spectrum disorder (NMOSD), Evrysdi for SMA, and Viltepso for certain DMD patients. These trials wrapped up just last year. Similarly, a myasthenia gravis drug was only allowed to be provided through expanded access last year. No programs exist yet for pridopidine, an investigational treatment for ALS and Huntington’s.

Roche, who is currently working on a Huntington’s disease treatment and also developed Evrysdi and Enspryng, has stated that this is because of their company policy: no expanded access until phase three trials have been finished. In most studies, this phase is the final one.

There has been an exception to this trend. Biogen provided an experimental ALS treatment to certain patients before the results of their clinical trials were known. While this situation is positive, there are still many patients who do not receive compassionate use. In fact, a 2015 estimate put the number of ALS patients in the country at 16,000, while only about 125 patients have been able to access treatment through compassionate use.

Looking Forward

There must be a change in expanded access, as many rare disease patients are unable to access any viable treatment options. For patients who will die without treatment, further research and completed data analysis do not matter.

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