8 Fox Focus | Research
Focus on the Patient :
New Thinking on Clinical Trial Endpoints Is Next Boon for Drug Development
By Kat Mehlhorn
When you take a medication , you ’ re likely thinking about how it will improve your daily life . Will it ease your pain ? Will it help you sleep better ? Will it last throughout your workday ? But those considerations — so critical for patients — don ’ t necessarily map to how drugs are approved , especially in Parkinson ’ s disease .
In clinical testing , drugs are approved based on their ability to meet a pre-determined endpoint — an objective measure to determine whether the intervention is beneficial . In Parkinson ’ s drug development , these endpoints have relied heavily on existing scales used in the clinic to measure how patients feel or function , such as the MDS-Unified Parkinson ’ s Disease Rating Scale ( MDS-UPDRS ).
But as many patients know , scales like MDS- UPDRS can be unreliable , and can ignore what matters most to them . “ I ’ m certainly more interested in knowing how a drug can improve my quality of life than that it moved me X points on the UPDRS ,” says Gary Rafaloff ,
MJFF Patient Council member Gary Rafaloff is pushing for patientfocused measures in clinical trials to help drugmakers develop better , more precise therapies for early PD .
who lives with PD and is a member of MJFF ’ s Patient Council . “ And I know from experience that it ’ s not reliable . The same clinician can measure you differently on the same day .”
Despite this common mismatch with patients ’ needs , the accepted endpoints and scale used are a roadmap for how companies design clinical trials and get new treatments approved .
One challenge identified with using these measures in drug development is that they focus on leaps in progression seen in later disease