Registries for Rare Diseases
A foundation for multi-arm, multi-company trials
White Paper
Jan 15, 2019

Through recent collaborative efforts, the FDA and EMA are hoping to encourage multi-arm, multi-company clinical trials for pediatric rare diseases. This white paper examines how registries can provide a valuable foundation for these types of trials by fostering long-term, collaborative relationships between rare disease stakeholders and improving our understanding of diseases and treatment outcomes.


In July 2017, the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) published a joint proposal on multi-arm, multi-company clinical trials, which would use a single control arm to compare multiple medicines.1 The proposal – focusing on Gaucher disease, but intended as a general roadmap for pediatric rare diseases – would allow for economies of scale as multiple sponsors could share the responsibility and cost of identifying and recruiting patients. This guidance does not eliminate any of the existing requirements for rare disease drug development; however, the FDA and EMA may exercise flexibility in determining the kind and quantity of data required to meet the statutory standard.

The approach could enable a better determination of relative risk-benefit by increasing the quantity and quality of safety and effectiveness data; and lead to smoother regulatory pathways, as regulators incentivize sponsors to consider these trials. While there are significant hurdles to the proposed approach, collaborative efforts to develop registries could be seen as a positive step in the process of multi-company cooperation in rare disease research.


Rare disease registries typically have the following objectives:
  • To connect patients, their caregivers and clinicians
  • To better understand the natural history and outcomes of the rare disease
  • To support research on the genetic, molecular and physiological basis of the disease
  • To establish a patient base for evaluating potential therapies and medical devices.

Data from registries can significantly benefit early-stage drug development, and potentially serve as a starting point for multi-arm, multi-company clinical trials. Registries can help address many challenges inherent in rare disease trials, allowing for a more complete understanding of the disease course and variability; guiding the development of endpoint measures, patient reported outcomes (PROs) and biomarkers; facilitating feasibility assessments for future trials; providing a source of patients for clinical trials; and gathering longer term safety and efficacy data after registration trials are completed.

Rare disease registries can also improve clinical trial design by providing information on disease natural history, informing sample size calculations, being used as a historical control group for post-marketing monitoring,5 or for examining real-life outcomes data from different treatment regimens globally.


Disease-focused, not-for-profit foundations and patient advocacy groups have a proven track record of operating registries in the public interest and can serve as models for future collaborations. Sponsors work in parallel to access specific data sets relevant to their R&D assets, helping to inform clinical development without disclosing commercially sensitive information. This may form the basis for future collaboration between sponsors on multi-company, multi-arm trials.

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