Spring / Summer 2023 | Research 9
MJFF is addressing this need for sensitive , patient-centered endpoints head-on and approaching the issue from two angles — coalescing the field around a shared understanding of what is meaningful to patients and bringing together key stakeholders in a precompetitive setting to develop a roadmap .
MJFF is convening global stakeholders to create a roadmap for more sensitive trial endpoints . At last November ’ s conference in Washington , D . C ., Karen Lee , PhD , president and CEO , Parkinson Canada , and David Dexter , PhD , deputy research director , Parkinson ’ s UK , joined MJFF ’ s Katie Kopil , PhD , senior vice president of clinical research .
rather than subtle , early changes . “ It ’ s like measuring grass growing with a yardstick ,” says Katie Kopil , PhD , senior vice president of clinical research at MJFF . “ The measurement is too big for the change you ’ d expect to see .”
So , how can drug developers better improve testing of potential treatments aimed at slowing or stopping disease progression in early Parkinson ’ s , a time when most individuals aren ’ t seeing significant day-to-day progression of their symptoms ?
In a shift that will address the needs of patients with early-stage PD , and ultimately lead to smarter , faster and less-expensive drug development , regulators in both the U . S . and Europe are turning their attention to what is appropriate to use as a measure of efficacy in trials targeting PD . This is a game-changing opportunity for companies designing late-stage trials of interventions in people with early PD because as it stands today , without sensitive endpoints , there is no way to tell if a drug is working in the typical time a trial is run .
MJFF has established an interdisciplinary patient-centered PD Endpoints Advisory Committee of experts from within and outside of the field , including patients like Gary . “ We need a consensus around how to move forward as a field . And we want to be in the driver ’ s seat alongside other patient organizations that share a similar commitment ,” says Kopil .
Last November , MJFF co-hosted a roundtable with Parkinson ’ s UK and Parkinson Canada to unite industry and academic groups working on new measure development with representatives from the U . S . Food and Drug Administration .
MJFF is also leveraging our Parkinson ’ s Progression Markers Initiative ( michaeljfox . org / ppmi ) infrastructure to collect data and validate novel patient-reported outcomes . One such study is asking patients and care partners about their experience with Parkinson ’ s in three domains : what ’ s bothersome , limiting and important .
At MJFF , we are working to convene the greatest scientific minds and bring them into direct partnerships with patients . Ultimately , it is these conversations that are critical to unlocking the early signs of PD , developing new scales to track changes over time and , ultimately , intervening before the disease ever develops .