PPMD's Duchenne Drug Development Roundtable Meeting Series, Part 3: Clinical Trial Readiness from the Viewpoint of Clinicians and Infrastructure

Accelerating trials. Protecting our robust therapeutic pipeline. Ensure as many hits on goal as we can.

Priorities shared by all of us.

And to achieve this, PPMD has convened our industry partners through our Duchenne Drug Development Roundtable and held a series of small, targeted meetings this winter, each focused on another unique element of this equation.

We have been so grateful to the sponsors who have come together – checked their IP at the door – and taken part in active discussions to help ensure that our Duchenne community benefits from the intense regulatory learnings of the past 18 months and the rich resources of our collaborations.

Today's Discussion Topics

Today PPMD is convening the third meeting of our 3-part meeting series: Clinical Trial Readiness from the Viewpoint of Clinicians and Infrastructure.

The first meeting focused on Clinical Trial Readiness – Patients and the second on Clinical Trial Optimization.

This third meeting focuses on challenges and opportunities through the lens of the trial sites themselves:

What works? What doesn’t?
With many companies doing the same things over and over, where are the synergies?
What could we do together as a community?
With over 15 trials ongoing and many more getting ready to recruit, patient engagement, recruitment and trial execution must go flawlessly. How do we ensure that we streamline our networks and build a sustainable infrastructure for the future?

Our hope is that this meeting, combined with the direction the first two meetings provided, will help us chart a new course and ensure that Duchenne clinical trials are optimized and available to every single one.

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About PPMD's Duchenne Drug Development Roundtable

Established more than seven years ago, PPMD's Duchenne Drug Development Roundtable (DDDR) is a group of committed innovators representing Industry and relevant stakeholders that has the goal of accelerating the development of meaningful treatments for Duchenne muscular dystrophy through open discussion to minimize duplication and to pool resources in pre-competitive space.

PPMD appreciates the support of our partners which enables the DDDR to continue driving the field forward.

We are extremely grateful to the DDDR Steering Committee and the company representatives who have served as planning committee members for each of the three meetings. Their guidance and leadership has impacted our Duchenne community in immeasurable ways.

The DDDR Steering Committee includes:

Cliff Bechtold (BMS)
Michael Binks (Pfizer)
David Cox (Eli Lilly)
Joanne Donovan (Catabasis)
Cartier Esham (BIO)
Ed Kaye (Sarepta)
Kim McCleary (FasterCures)
Thomas Meier (Santhera)
Jeff Watkins (patient community representative, parent)

This Roundtable series summary will be disseminated to all DDDR members in time for discussion at our annual DDDR meeting in June. We hope that all of our Duchenne partner organizations will continue to join us in this annual discussion and planning meeting.

It is our hope that this DDDR meeting series will assist PPMD and the Duchenne community to work together to drive forward collaborations and projects that will benefit all partners working in this space – and yield effective therapies that are accessible to all who need them.

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