PPMD’s Duchenne Drug Development Roundtable
PPMD’s Drug Development Roundtable (DDDR) is comprised of the majority of industry partners engaged in developing therapeutics for Duchenne, that come together in the pre-competitive space to address shared challenges and opportunities in Duchenne therapy development. The goal is to accelerate the development of meaningful treatments for Duchenne muscular dystrophy through open discussion and collaborative engagements. Membership is open to all sponsors within the Duchenne space; an annual fee structure to support DDDR meetings and collaborative DDDR activities is required of participants.
2021 Drug Development Roundtable Members
The DDDR Steering Committee
DDDR Steering Committee members are selected by PPMD Executive Leadership, are of a voluntary capacity, and represent the following categories all relevant to Duchenne: a small industry sponsor organization, a mid-size industry sponsor organization, a regulatory policy expert, a representative from the Duchenne patient community, and representatives from different research modalities.
- Cliff Bechtold – Biohaven Pharmaceuticals
- Joanne Donovan, MD, PhD – Catabasis
- Roxana Dreghici, MD – Roche/ Genentech
- Cartier Esham – Biotechnology Innovation Organization (BIO)
- Diane Berry – Sarepta
- Rose Juhasz – Patient Community Rep
- George Mulligan – Astellas
- Stu Peltz, PhD – PTC Therapeutics
- Jodi Wolff, PhD – Santhera
The current emphasis areas for the DDDR includes:
PPMD and the DDDR have focused on working to facilitate a favorable access environment for Duchenne patients in a post-approval space. As our therapy development pipeline has continued to gain momentum and we anticipate the approval of the first gene therapies in Duchenne, PPMD has developed a strategic plan forging partnerships and actively engaging payers and valuators to inform decision making in the access environment. Recent activities impacting access and including DDDR members leadership and participants have included:
- New ICD-10 Code
- Patient Focused Impact Assessment (PFIA) Act Implementation
- Duchenne Patient-Focused Compass Meeting, March 5 2018 (“PFDD 2.0”)
- Patient Preference Studies
- Newborn Screening Pilot Consortium & New York State Pilot launch
- Duchenne DDDR Access Meeting – February 2019
- PPMD Gene Therapy Policy Forum – March 2019
- Duchenne Outcomes Meeting – May 2019
Upcoming DDDR activities will continue build upon these efforts — and will also begin to focus on clinical trial site capacity.
Building on Momentum
The past several years of DDDR engagements have focused on assessing our Duchenne community’s regulatory learnings and applying them to our current pipeline.
2018 DDDR Small Meeting Series:
- Clinical Trial Readiness – Patients, Families, and Treating Physicians
- Optimizing Clinical Trial Design
- Clinical Trial Readiness – Sites & Investigators
The yield from this meeting series was 2 distinct efforts currently being moved forward by PPMD team and workgroups comprised of representatives from industry:
- Clinical Trial Harmonization
- Planning for a Platform Trial in Duchenne
The three meetings yielded two distinct efforts that are each being moved forward by PPMD team members, and workgroups comprised of representatives from industry, clinicians, and the Duchenne community.
Clinical Trial Harmonization
A cross-organizational work group comprised of members of the DDDR together developed a series of seven guidances that reflect ‘best practices in conducting clinical trials in the Duchenne space’. These guidances are being prepared for publication and are intended to help promote efficiencies within trial design and implementation, as well as harmonization across clinical trials.
Developing a Platform Trial for Duchenne
The Duchenne Platform Trial (DPT) is one that is facilitating the exploration of a master protocol concept for Duchenne. Currently, each trial in the Duchenne space is designed, executed and governed as an individual exercise, yet many steps that need to be completed are done for each trial, often at the same sites who run multiple trials at once. While each protocol is slightly different, most are sharing many sites and using similar screening processes and outcome measures. A single platform that leverages the shared aspects of a trial could enhance efficiency greatly. Additionally there is the possibility to share placebo patients across the platform thus reducing the overall number of patients needed to enroll.
The Platform Trial design team, a group of 10+ individuals representing industry, patients, and a biostatistics expert, has been working to create a master platform trial protocol that has the capacity to test multiple therapeutics at one time, eventually testing combinations of these therapies as well. In 2018, PPMD entered into a collaboration with the Institute for Advanced Clinical Trials in Children (I-ACT) to form a Consortium of interested parties to help catalyze this effort. This platform trial protocol was presented and discussed at a Public Workshop in September of 2019.
Annual DDDR Meeting – Convening Our Community
Each June, we convene our Annual Duchenne Drug Development Roundtable meeting which brings together all Duchenne industry sponsors, as well as global Duchenne advocacy partners. The meeting allows for a robust exchange of information about challenges before us, as well as novel opportunities for coming together to work through them.
We are confident the efforts of the DDDR will result in more efficiency, less redundancy and better science, leading to faster therapeutic options for our community.
For more information please contact PPMD’s Chief Business Officer, Kaylan Moitoso.