By Olivia Woodford-Berry, '19
Despite impressive advances in medical technology over the last century, there is little hope for those diagnosed with Duchenne Muscular Dystrophy (DMD). DMD, which is a form of the broader muscle-wasting disease muscular dystrophy, is a genetic condition that primarily affects boys and causes the atrophy of muscle, primarily in the chest and legs. This eventually leads to detrimental muscle loss and death . Like many relatively rare genetic diseases, there has been very little
medical research into DMD. Thus, there are often no Federal Drug Administration (FDA)-approved treatments for such conditions. For those suffering from such diseases, the system has seemingly failed them. However, there has been a shift in drug approval toward a more patient-focused process.
Much like the legislative process, the drug approval process is based on a rigorous set of requirements, with careful speculation regarding the potential implications of any decisions. Companies submitting new drug applications (NDAs) to the Federal Drug Administration are responsible for proving the efficacy and safety of their products . A team of physicians, chemists, and other qualified experts will then review the application and make a recommendation on the drug’s status. When analyzing applications, they consider the both the results and the level of rigor demonstrated in the study. Afterwards, given this recommendation, the FDA makes a decision. However, these rulings are not always as clear-cut as one would think.
At the end of last year, this process resulted in a public outcry for sympathy. In a case showcasing eteplirsen, one of the first promising drugs for the treatment of DMD, the FDA received several conflicting perspectives on how they should rule . The committee recommended that the drug be denied approval based on the fairly sound basis that the trial group had been too small and the research methods flawed. The notable aspects of this case enter with the influence of public opinion, as the FDA has more recently been pushing to include more patient voices in these decisions. Despite scientific shortcomings, many patients and parents participating insisted that this drug mitigated the effects of the disease. In a highly controversial decision, the FDA approved the use of eteplirsen despite committee recommendations .
Although the FDA did approve the drug with the conditions that further studies be completed to maintain this status, this decision sets a unique precedent for the pharmaceutical industry. Some argue the ruling allows pharmaceutical companies to make unethical cuts on patient trials and still get FDA approval. On the other hand, certain patient advocate groups feel that this ruling leaves room for cases involving rare conditions where research is difficult to conduct on a large scale, such as this one. The FDA has also created the Orphan Drug Design Program, which creates grants that incentivize companies to research drugs for rare genetic diseases. Thus, this case has demonstrated the FDA’s commitment to increased patient inclusion and to improved access to treatments for groups who previously felt abandoned by the pharmaceutical industry. The approval of eteplirsen has opened the door for several more drugs that attempt to treat DMD to begin the FDA approval process in the near future.
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