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HEALEY ALS platform trial
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| The HEALEY Center for ALS at Massachusetts General Hospital
was established in 2018 with a large philanthropic donation.
One key objective of the Center is to more efficiently test
drug candidates for the treatment of amyotrophic lateral
sclerosis (ALS). To meet this objective, they developed the
HEALEY ALS Platform Trial to facilitate testing of multiple
drug candidates under a single master protocol. It was clear
to patients and other stakeholders that the HEALEY ALS
Platform Trial offered obvious advantages by providing a way
to increase the speed of testing, cut the cost of research,
decrease the number of participants exposed to placebo, and
bring innovative drugs to patients sooner. By leveraging a
platform trial design, they anticipate they will reduce the
cost of research by 30% and decrease trial time by 50%. |
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Duchenne Muscular Dystrophy Platform Trial
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| Similar to ALS, the large number of drug candidates in the
Duchenne muscular dystrophy (DMD) pipeline reinforced the
need for innovative study designs that could speed the
development of new therapies. However, perceived loss of
patient choice and misalignment on target patient population
options and appropriate endpoints introduced challenges in
securing buy-in to the platform trial approach. Parent
Project Muscular Dystrophy, in partnership with the United
States Food and Drug Administration and other key leaders
from the community, generated impactful discussion forums
that ultimately led to alignment on key patient-centered
design considerations that were essential for the progress
of the Duchenne Platform Trial and will strengthen future
clinical development strategies in DMD. |
