According to a story from Charcot-Marie-Tooth Disease News, the U.S. Food and Drug Administration (FDA), under the leadership of commissioner Scott Gottlieb, has begun to change clinical trials by accepting new treatment measures and making trial methods more flexible. This has earned him praise from rare disease patient advocacy groups.

Gottlieb appears to be more eager than his predecessors to work more closely with patient advocacy groups to develop trials methods that take into account “real world” data that has been gathered outside of the trial environment, as well as further incorporation of new treatment endpoints. These new measures also take into account the natural disease histories, which can often vary widely among rare diseases and helps account for the unusual circumstances that they often carry.

The state of clinical trials in the U.S. has long been subject of controversy. The trials process has come under scrutiny due to various inefficiencies, such as redundant trial requirements that can prevent patients from participating and a one-size-fits all approach to statistical endpoints. Traditionally, the FDA has favored randomized, placebo controlled trials, and while this is still the standard, the more recent efforts signal a willingness to investigate different approaches.

The FDA has been moving with record-breaking speed in its approval of new therapies and drugs, which has allowed for treatments to help patients at a rapid rate. This has partially been achieved by using alternative endpoints and changes to trial measurements. However, this new efficiency must be balanced by maintaining safety and effectiveness.

An example of an adaptive endpoint is the adoption of event rate of pulmonary exacerbations in a trial funded by the Cystic Fibrosis Foundation in testing a new treatment for cystic fibrosis called lenabasum. The traditional forced expiratory volume per second (FEV1) was less useful for this treatment, which was meant to reduce the loss of volume overtime instead of a cause an immediate change.

The agency is also accepting data from long term natural disease history studies that will reduce the need for the use of placebos as a part of trials. The FDA is helping fund studies for Friedrich’s ataxia, sarcoidosis, sickle-cell anemia, muscular dystrophy and Angelman syndrome. The agency also accepted natural disease history data for its approval of a treatment for spinal muscular atrophy. The willingness to accept more patient-focused endpoints and take into account disease history should be regarded as positive signs by the rare disease community, and will hopefully help develop safer and more effective treatments in the future.

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