Pursuing Smaller, Faster Clinical Trials For Progressive Neurological Disorders

[Farooqji]

It’s easy to be pessimistic when it comes to investigating Parkinson’s disease. The condition, which causes progressive damage to brain cells over many years, has no therapy to halt its progression, with current treatments focusing only on symptom management.

The challenges in Parkinson’s drug development are diverse. Traditionally, clinical trials for progressive neurodegenerative disease are expensive, are time-consuming, and often require hundreds or even thousands of participants. Patients experience many individualized symptoms, and because of the often-debilitating nature of those experiences, their level of commitment throughout the duration of a clinical trial may waver, particularly as the benefits of any intervention that slows disease progression can take years to be revealed. What’s more, neurology is an area of calculated risk, and every new mechanism taken into clinical development has a high chance of failure, especially with the cost and complexity limiting the number of these trials.

clinicalleader.com/doc/purs...

[Ctime]

This is a great article and worthwhile read particularly for anyone wanting concrete policy steps to jump start rational and coordinated efforts to solve neurological decay. It is not a technical medical article.

It takes a while to get to what I think is the real meat; that technology, wearables, biomarkers etc., can help enable meaningful small trials by differentiating patient groups and creating more meaningful trial outcomes.

Having done some trials that are using occasional (monthly) UPDPRS as a metric and lumping a hugely diverse set of symptoms into one trial, it amazes me that wearables are not already a pre and post screen to drug trials as well as part of massive database efforts like the PPMI study.