AccelerateRARE Working Groups

Monday, September 14, 2026

Attendance: Requires full Summit registration and organizer approval. To request participation, please email jenny@raretrialssummit.com.  Attendance is limited to 40 participants per group, with priority given to senior leadership.

Objectives:

Develop stakeholder consensus on rare disease clinical development topics.

Create briefing documents and community guidelines to meaningfully accelerate rare disease clinical development.

2025 Report:

37 experts from biopharma and patient advocacy convened at the 2025 Summit to draft the priorities of the rare disease clinical development community ahead of the upcoming PDUFA VIII reauthorization.

Download the Report

2026 Working Group A:

Evidence Standards for Rare and Individualized Therapies

Purpose:

Ground the discussion in current FDA evidence standards, then propose approval models for rare and individualized therapies when conventional randomized trials are infeasible.

Key questions:

  • What does the current framework permit, and where are the gaps?

  • How could platform and molecular-taxonomy approvals work?

  • When can non-RCT evidence support approval?

  • What evidence and safeguards should be required?

Proposed Output:

An Evidence Reform Framework for Rare and Individualized Therapies

Working Sections:

  • Regulatory Pathway and Evidence Sufficiency: Current rules, gaps and future approval models.

  • Natural History, External Controls and Statistical Credibility: Standards for credible comparisons.

  • Mechanism-to-Clinical-Benefit Evidence Chain: Molecular taxonomy and the evidence linking mechanism to benefit.

  • Trial Design and Evidence Generation in Tiny Populations: Self-controlled, n-of-1, Bayesian and other non-RCT designs.

  • CMC, Safety and Post-Market Evidence: Requirements for platform and individualized interventions.

Suggested participant profile

  • Regulatory and policy experts

  • Sponsor development and CMC leaders

  • Biostatisticians and trial methodologists

  • Natural-history, genomics and biomarker experts

  • Clinicians, advocates and research partners

2026 Working Group B:

Minimum Readiness Standards for Pediatric Rare Disease Trials

Purpose:

Define the minimum conditions for a pediatric rare disease trial to launch responsibly and successfully.

Key questions:

  • Is the evidence sufficient to justify enrolling children?

  • Are endpoints and assessments fit for the population?

  • Is participant and caregiver burden acceptable?

  • Can sites and decentralized-care partners execute reliably?

  • Which gaps threaten enrollment, data quality or retention?

  • What should trigger a go, conditional-go or no-go decision?

Proposed Output:

Minimum Readiness Standards for Pediatric Rare Disease Trials

What minimum conditions must be met before a pediatric rare disease trial should launch?

Working Sections:

  • Scientific and Ethical Readiness

  • Endpoint and Measurement Readiness

  • Child and Caregiver Readiness

  • Operational Readiness

  • Launch Decision and Remediation

Suggested participant profile

  • Pediatric clinicians, investigators and sponsor leaders

  • Endpoint, biomarker and digital-health experts

  • Patient and caregiver advocates

  • Site, home-health and trial-operations leaders

  • Regulatory and bioethics experts