As biomedical innovation rapidly grows and evolves, it leads to breakthroughs that allow treatments to be developed that will turn fatal diseases of today, into chronic but manageable ones of the future. However, the trials that lead to these new medications being available to patients are dependent on receiving FDA approval – and the trial process does not always closely align with what the FDA expects.

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Clinical trials

The FDA is looking for large and random, placebo-controlled medical trials with professional clinical staffing. However, with such new biomedical innovation, it is becoming necessary for America’s medical regulatory systems to evolve too.

It is agreed that drugs must be thoroughly tested to be deemed safe and effective before being issued to patients. The FDA’s drug review and approval system is exceptional and protects the public from potentially harmful medications.

Eteplirsen, the first FDA approved medication to treat patients with DMD (Duchenne Muscular Dystrophy) has brought about a discussion around how the FDA evolves to meet patient needs. While FDA approval was granted for Eteplirsen in September, the debate lingers over whether there were sufficient clinical trials for approval to be awarded.

Dating back to 1992, the FDA introduced their accelerated approval process which was created to get effective and safe drugs to patients when there are no other options that exist. It has made a huge difference in approving cancer treatments, HIV/AIDS treatments and many other non-curable diseases.

The debate regarding the DMD treatment stems from the fact that rare diseases affect low numbers, making the populating of clinical trials difficult. It is vital when testing and evaluating medicines for diseases that affect a small population, that decisions reflect the altered scope of the arena. Companies such as provide expert staff for clinical trials.


Rare disease drug testing must be recognised as having a much smaller impact on public health than a regulatory error that concerns wider population treatments. It is then the affected patient’s decision whether they wish to accept this risk to gain quicker access to potentially effective treatments.

The FDA’s response to dealing with these evolving issues will have a widespread effect. It has worked in the case of Eteplirsen. It is now time for the FDA to build on this process.