Rare disease companies' existential challenge: Patient enrollment
- Currently, three companies, Vtesse, CTD Holdings, and Orphazyme, are recruiting patients with Nieman Pick Type C (NPC), a very rare and deadly disease, for clinical trials.
- Niemann-Pick Type C is a fatal genetic condition, part of a group of more than 50 inherited disorders where cells fail to process and recycle waste.Affected patients experience the loss of the ability to walk and loss of speech as a result of NPC, and generally die as teenagers. Currently, there is no treatment.
- It is unusual to have two companies vying for particpants for a rare disease, which affects roughly 500 people worldwide, but patients' advocacy groups have been pushing for more clinical study of rare diseases for years.
Rare diseases as a category have come into their own in the last few years, as advocacy groups, such as the National Niemann-Pick Disease Foundation, mount massive education and protest efforts for more funding, clinical support, patient support and overall awareness. In fact, in 2014, 17 of the 41 new drugs approved by the FDA were for rare diseases.
In the case of NPC, the major problem is not just too few recruits, but other barriers as well, including the fact that patients who have previously taken the drug in earlier trials or under the compassionate use act, are excluded. Vtesse, which is based in Gaithersburg, MD, is testing cyclodextrin for treatment of NPC and plans to starting recruiting 51 patients at 20 sites worldwide.
Based on early-phase data from 12 children, cyclodextrin slows progression of NPC, compared with untreated patients. In addition, CTD Holdings, based in Florida, is testing another cyclodextrin-based product called Trappsol Cyclo, which is currently sold in the U.S. for compassionate use. CTD representatives have been meeting one on one with families of individuals with NPC to encourage them to enroll in their trial.
Meanwhile, Orphazyme, a Danish company is recruiting NPC patients in Europe to test a pill called arimocolmol. They are laso petitioning the FDA to allow recruitment in the U.S. With the increased use of priority review and an invigorated focus on rare disease drug development, lack of clinical trial recruits will remain a problem, which will ultimately require an innovative approach to clinical trial design and regulatory approval. However, given the alternative of not having any potential cures for rare diseases, recruitment challenges are a good problem to have.