PPMD is excited to share the recently developed Clinical Trial Harmonization Guidances. These guidances are the result of PPMD’s Duchenne Drug Development Roundtable working collaboratively in the pre-competitive space to encourage better consistency across companies in planning clinical trials in Duchenne. Our hope is that these documents can provide companies working in or entering into the Duchenne space direction and insight from our collective learnings to date.

In 2010, PPMD formed the Duchenne Drug Development Roundtable (DDDR) in an effort to bring together industry partners developing Duchenne therapies in a pre-competitive space to address shared challenges and opportunities in Duchenne therapy development. The goal of the DDDR is to accelerate the development of meaningful treatments for Duchenne through open discussion and collaborative engagements.

In 2017 and 2018, the DDDR convened a series of small meetings each focused on specific topics areas that were identified as critical priorities by the DDDR Steering Committee and through discussions with DDDR membership. The goal of the series was to identify opportunities to optimize regulatory learnings and explore innovations that could be applied to the Duchenne therapy development space. From those meetings, the Clinical Trial Harmonization Working Group was formed between DDDR members and other key stakeholders in the Duchenne drug development ecosystem. The role of this group is to provide tools, templates, and guidance to encourage and facilitate quality, efficiency, and consistency across Duchenne clinical trials.

A key deliverable of this working group was to create a series of clinical trial harmonization guidance documents to aid in the development of trials and provide more consistency with Duchenne trial planning and process considerations ensuring that families and patients remain at the center of developing these documents.

We are pleased to share the completed guidance document with the community.

Download the Guidance >

The guidance includes:

• Protocol Development & Review
• Consenting/Assenting Processes
• Travel Guidance for Duchenne Clinical Trial Sponsors
• Non-Ambulatory Community Commitment
• Best Practices in Communications
• Formal Engagement of CROs
• Clinical Trial Welcome Packets

We consider these to be living documents where periodically we will need to update and adapt based on the changing environment.

We appreciate the effort of the Clinical Trial Harmonization Working Group leading this effort. Our hope is that these documents can provide companies working in or entering into the Duchenne space direction and insight from our collective learnings to date.

2019 Drug Development Roundtable Members

2019 Drug Development Roundtable Partners