PPMD’s Duchenne Drug Development Roundtable

PPMD’s Duchenne Drug Development Roundtable (DDDR) is composed of the majority of industry partners engaged in developing therapeutics for Duchenne and Becker, that come together in the pre-competitive space to address shared challenges and opportunities in drug 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.

2024 PPMD Duchenne Drug Development Roundtable Members

The DDDR Steering Committee

DDDR Steering Committee members are of a voluntary capacity, and represent the following categories all relevant to Duchenne: industry sponsor organizations from various therapeutic areas, a regulatory policy expert, a representative from the Duchenne patient community, and representatives from different research and clinical modalities.

  • Jennifer Hodge, PhD – Pfizer
  • Abby Bronson, MBA – Edgewise
  • Siobhan Fitzgerald– Sarepta
  • Rose Juhasz – Patient Community Rep
  • Ashish Dugar, PhD, MBA – Dyne Therapeutics
  • Diana Castro, MD

Current Focus

The current emphasis areas for the DDDR includes:

Harmonization of Clinical Trial Experience

A major driver of the DDDR has been to establish best practices for designing clinical trials with the patients and families in mind. This work has culminated in the development of the Harmonization Recommendations which cover broad topic areas such as:

  • Protocol Development Guidance for Duchenne Clinical Trial Study Sponsors
  • Informed Consent Guidance for Duchenne Clinical Trial Study Sponsors
  • Travel Guidance for Duchenne Clinical Trial Study Sponsors
  • Commitment to Non-ambulatory Duchenne Community
  • Communications Guidance for Duchenne Clinical Trial Study Sponsors
  • Optimizing Contract Research Organization (CRO) Engagement
  • Clinical Trial Welcome Packets

The Harmonization Recommendations are a foundational living document for the DDDR. Updates to the guide are focused on:

  • Best practices for trial communication to the community with sensitive material
  • Strategies for return of patient data
  • Improving the informed consent process to better standardize delivery at site and ensure patients and families are empowered in their decision making process

Access

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
  • PPMD Gene Therapy Policy Forum
  • Duchenne Outcomes Meeting

Upcoming DDDR activities will continue to build upon these efforts — and will also begin to focus on clinical trial site capacity.

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. A Type B meeting was held with the FDA in 2020 to gain feedback on the design of the Master Protocol for the Duchenne Platform Trial.

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.

2020 DDDR Small Meeting Series:

  • Use of external controls in clinical trials
  • Lessons learned from COVID-19 and applicability to decentralized trials
  • Patient experience with COVID-19, trial communications, and return of patient data

The yield from this meeting series was 2 distinct efforts currently being moved forward by PPMD team and workgroups comprised of representatives from industry:

  • Improving and standardizing trial communications
  • Establishing best practices for return of patient data

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.

Learn More

For more information please contact PPMD’s Chief Business Officer, Kaylan Moitoso.