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HDSA Statement on new FDA rule
allowing access to investigational drugs

Jang-Ho Cha, MD PhD

Chair Center Programs and Educational Advisory Committee

HDSA National Board of Trustees

 

The FDA has issued a new rule that potentially allows patients access to investigational drugs. This rule, while potentially empowering for patients, should be greeted with caution.

 

There are currently no treatments that slow the progression or cure Huntington's disease, and so the need to develop new drugs is paramount. In terms of research to advance treatments, there is good news and bad news. The good news is there is no shortage of new ideas that are being advanced for HD treatment. For example, the Coalition for the Cure investigators and other HDSA-supported scientists have discovered treatments that slow disease progression in HD mice. With each of these exciting reports, however, several important questions are raised:

  •           Will this treatment be effective in HD patients?
  •           Will this drug be safe in humans?
              Will this drug be harmful to humans?
  •           What dose is most effective with the least amount of harmful side
             effects?

 

The answer to these important questions can only be obtained through clinical research, that is, research that involves human subjects. In this regards, clinical trials are essential to provide the answers to these important questions that we all want to know. HDTrials.org is a website that is dedicated to increasing participation of HD patients and families in clinical research.

 

As a community, we all need to work together in order for better treatments to be developed. Achieving a treatment and an eventual cure for HD will take the concerted efforts of patients, families, scientists and pharmaceutical companies. For safety and in order to gather the most information possible, persons interested in new treatments should participate in clinical trials.

 

For those for whom clinical trials are not feasible or accessible, the FDA rule may offer another way to try promising but unproven treatments. Most clinical trials have inclusion or exclusion criteria, that is, the clinical trial is recruiting only certain types of patients. The reason for this is that the clinical trial is designed to answer a specific question.  An example would be a trial working to determine "Does this medication reduce movements?"  For such a trial, it is logical to include only those patients that have abnormal movements. As a result, some patients will find themselves excluded from clinical trial participation. For others, geographic barriers prevent participation in a clinical trial; not every clinical trial is held at every location.

 

So, the new FDA rule allows persons to have access to investigational drugs outside the confines of a clinical trial. This development should be seen as a mixed blessing. On the one hand, many more patients will be permitted access, which seems like a good thing. On the other hand, investigational drugs are just that: investigational. For many of these compounds, we simply do not know if the medications are safe or effective, which is the reason for the trial itself.

 

Patient subjects in a clinical trial are subjected to careful safety monitoring in order to detect harmful side effects. It is not clear what kind of similar protection would be available to patients taking investigational drugs outside of a clinical trial. The other sobering reality is that most investigational drugs 'fail,' meaning that many of the medications that people will be trying may not have any benefit or may have some harmful side effects. That's why they call it research. However, even these "failures" add to our understanding of Huntington's Disease, and help us move forward towards successful therapies.

 

In the absence of an effective treatment for HD, many people are willing to take a chance. If a drug has reached the stage of a clinical trial, it is because there is significant evidence that it might work. The new FDA rule may be permissive to those who are willing to take that chance. Finally, it is important that patients inform their physicians about all medications, supplements and vitamins they are taking. One important caveat with investigational drugs is that their drug interactions are largely not known, and so there is no easy way for a physician to check for harmful interaction with the medications already being taken.

 

HDSA will follow the activity that occurs as a result of this new regulation and provide updates about therapies in clinical trials so that through the systems and protections of clinical research we can identify meaningful treatment for Huntington's disease.