Toward Patient-Centered Policy

A Case-Study on Why Change is Needed at the FDA

Approximately 30 million people in the United States are living with a rare disease, most often genetic in origin. About 95% of these conditions have no treatment approved by the US Food and Drug Administration (FDA). These patients are suffering and dying without hope of a treatment; a direct result of the overly restrictive nature of the FDA drug approval process.

Recently, the Wall Street Journal published a front page article documenting the approval process for a drug to treat Duchenne muscular dystrophy (DMD), a terrible genetic disease that causes those affected to slowly lose muscle function until their hearts and respiratory systems shut down. The story demonstrates that personal health decision-making authority should be shifted to patients and their doctors and away from the agency. Reigning in some of the FDA’s authority will also decrease the incentive for drug companies to curry favor from the organization in exchange for granting marketing approval.

The article chronicles Sarepta Therapeutics, Inc’s efforts to develop a drug designed to raise levels of a missing protein in DMD patients called dystrophin. When the company sought FDA approval for the drug, the medical review panel voted against approval. The medical review panel didn’t find any significant health risks associated with the medication. However, a study done on a similar drug showed that any effect on the levels of the missing protein appeared to be small so that no clinical benefit could be established.

While the drug’s effectiveness was underwhelming from a clinical perspective, when individual cases are considered, the drug literally changed lives. Both of Jennifer McNary’s sons have DMD. Naturally, Ms. McNary desperately wanted to find some treatment in order for her sons to avoid an untimely death before the age of thirty as is common in DMD patients. Sarepta enrolled one of her sons, Max, in an early trial of their drug. The McNary family was overjoyed when Max showed signs of progress. He was able to do more everyday tasks, like opening a milk container, without assistance and could get out of his wheelchair for longer periods of time.

The vote to not approve the DMD drug robs patients like Max of their right to make decisions about their health for themselves. Decisions about the costs and benefits of experimenting with a new treatment ought to be made by a patient provided with the best possible information about that treatment. A government official cannot determine how much risk each patient is willing to accept, particularly in a life and death situation. This is especially true in the case of Sarepta’s drug, because there were no significant risks associated with the drug. Shifting this responsibility to patients means preventing the FDA from making approval decisions based on a drug’s efficacy.

Restricting the FDA’s authority will also reduce the need that companies like Sarepta have to “work the levers of power in Washington.” The article shows the enormous amount of time and money Sarepta poured into its efforts to lobby FDA officials. This process is a huge, inefficient mess. Even Sergey Brin of Google, a company with massive cash reserves, admitted that the web of regulation deterred him and other entrepreneurs from taking up most health related projects. This actually hurts patients because it means that potentially life-saving drugs are not being created that otherwise might. It also means that companies like Sarepta are forced to focus their time and money on navigating the process, instead of producing more drugs and helping patients.

Sarepta’s drug for DMD did eventually get approved when FDA officials overruled the medical review panel and allowed the drug to be marketed with the condition that it contain a label letting patients know that no clinical benefit was established. Making this standard procedure, especially for safe drugs treating rare diseases, will allow those desperate patients like Max to have a ray of hope. Focusing on equipping these patients with adequate information on these drugs will allow them and their physicians to make the best decision in their circumstances.

Getting medical regulation right is of utmost importance because patients’ lives are at stake. Correct FDA reform could mean that patients have access to drugs that improve their lives; inaction means more patients will die unnecessarily. The focus of our health policy framework should be the patient’s well-being. With consultation from physicians, patients are best equipped to make difficult decisions about their health. Congress and the FDA ought to respect and facilitate those decisions. The cost of continuing under the current model is too great.