On June 22, 2026, PPMD Chief Executive Officer Katherine Beaverson, MS attended the U.S. Department of Health and Human Services (HHS) Clinical Trials Roundtable in Washington, D.C., joining leaders from across government, industry, academia, and the patient advocacy community.
The roundtable, convened by HHS leadership, focused on a central challenge: how to cut the duration of clinical trials in half while maintaining the highest standards of safety and scientific rigor. The conversation underscored a growing urgency across the federal government to ensure the United States remains the global leader in biomedical research, particularly as more early-stage trials move overseas due to speed and cost.
A Shared Goal: Faster Trials Without Compromising Safety
A consistent message emerged: patients deserve timely access to therapies, but not at the expense of safety, transparency, or trust.
HHS outlined a coordinated, department-wide effort to:
- Reduce bureaucracy and streamline regulatory processes
- Launch trials faster and more efficiently
- Expand the number of trials conducted in the United States
- Maintain rigorous evidence standards and informed consent
For the Duchenne and Becker community, this balance is critical. Families cannot afford unnecessary delays, but they also rely on confidence that therapies are safe and supported by meaningful evidence.
What We Heard from Federal Leaders
Leaders from the FDA, NIH, CMS, and other agencies shared concrete steps underway to modernize clinical research:
- FDA (CDER) emphasized simplifying early-stage requirements, including clearer expectations for Investigational New Drug (IND) applications, new guidance on dose selection and trial design, and the exploration of more flexible, rolling review processes.
- NIH highlighted opportunities to use artificial intelligence to improve trial efficiency, expand access to underserved and rural populations, and return results to participants to incentivize engagement.
- NCI and NCATS pointed to the need for standardized contracting, centralized Institutional Review Boards (IRBs), and modernized data monitoring systems to reduce delays.
- CMS reinforced the importance of strong clinical evidence to support patient access to innovative therapies.
- Health IT leaders discussed integrating clinical trials into electronic health records to better match patients with studies and streamline enrollment.
Industry and academic stakeholders echoed these priorities, noting that delays often stem not from regulatory review timelines, but from the time and resources required to prepare trial applications, activate sites, and navigate fragmented systems.
Key Takeaways for the Duchenne and Becker Community
Several themes stood out as especially relevant to PPMD’s work:
- Site readiness and activation matter. Delays in contracting, IRB approvals, and site startup can significantly slow trials. Understanding and addressing these barriers is critical.
- Early engagement with regulators is essential. New models for pre-IND collaboration could help sponsors move more efficiently into first-in-human studies.
- Data and infrastructure are foundational. Investments in trial networks, data platforms, and workforce development will be necessary to sustain innovation.
- Access must be expanded. Trials cannot remain concentrated in major academic centers. Rural hospitals, community sites, and diverse populations must be part of the solution.
Where We Go from Here
PPMD welcomes the Administration’s focus on improving the speed and efficiency of clinical trials. For the Duchenne and Becker community, these efforts are not abstract – they are deeply personal.
PPMD will continue to:
- Advocate for policies that reduce unnecessary barriers to trial initiation and participation.
- Elevate the patient and caregiver voice in discussions about trial design and access.
- Work with federal partners to ensure that reforms reflect the realities of rare disease research.
- Promote solutions that maintain scientific rigor while accelerating meaningful progress.
- Submit timely, substantive comments on draft guidances and federal proposals to ensure Duchenne and Becker-specific considerations are reflected in regulatory decision-making.
As these proposals move forward, PPMD looks forward to continued engagement to ensure that the needs of the Duchenne and Becker community remain at the center of clinical trial innovation.
Because for our families, every day matters – and every step forward brings us closer to a future where every individual affected by Duchenne and Becker has access to effective therapies, optimal care, and the support they need.
The post Inside the HHS Clinical Trials Roundtable: Why Speed, Access, and Rigor Matter for the Duchenne Community appeared first on Parent Project Muscular Dystrophy.