Reflection on clinical and methodological issues in rare disease clinical trials

1:1
Experts provide a comprehensive reflection on the unique clinical and methodological challenges inherent in designing and executing trials for rare diseases.

This paper discusses the specific hurdles faced in rare disease research, including small patient populations and recruitment difficulties. It reflects on innovative trial designs and statistical methods that can enhance the robustness of clinical evidence in these settings.

Acces this publication (pdf)

Source

Orphanet Journal of Rare Diseases

Link to cite

Acces to Link >

You might also be interested in

Advancement of Treatments for Rare Diseases

Advancement of Treatments for Rare Diseases

The two-day event will focus on translating research and policy initiatives into tangible benefits for Rare Disease patients through better prevention, diagnosis, and treatment.
RealiseD webinars

RealiseD webinar series: Helping re‑shape rare disease clinical trials

13 January – 10 February 2026 | Online webinar series to advance best practices in clinical trial design for rare and ultra rare diseases.
Non-human primate hand via pickpik.com (CC0)

EMA public consultation on non‑human primate use in safety testing

EMA seeks stakeholder input on new reflection paper to reduce and replace non‑human primate use in medicines safety testing through 3Rs approaches.
abstract publication image on science health

Tailored antisense oligonucleotides for ultrarare CNS diseases: An experience-based best practice framework for individual patient evaluation

Outside traditional drug development pathways, clinicians and scientists face the challenge of systematically evaluating whether individual patients with severe ultrarare diseases might be eligible for and potentially benefit from individualized mutation-specific RNA therapies.

ERDERA's

search form