- Representatives from Rare Cancers Europe (RCE) have asked the EMA to allow patients with rare cancers to gain earlier access to experimental drugs.
- In general, medical decisions are usually risk-averse; however, rare-cancer patients are more willing to try experimental therapies in spite of the risks.
- RCE advocates are asking for a variety of approaches that would relax the protocols, including low-power, randomized clinical trials for these patients and other nontraditional approaches.
The old adage "higher risk means higher returns" is especially true for patients with rare cancers, who are facing a bleak future without some type of curative intervention. According to RCE, these patients can benefit from being involved in clinical trials and gaining access to treatments that might otherwise be unavailable to them.
Some approaches beyong low-power clinical trials include the use of surrogate endpoints in lieu of clinical endpoints; trials that rely on various types of biomarkers; and information from electronic medical records gained from other trials.