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EB girl visiting an EB ambulance with his mother.

Overcoming the challenge of clinical trials

Clinical trials and patient access

EB patients willing and able to participate in clinical trials are essential in the process of developing approved treatments, products and diagnostics. 

Life with EB is difficult, and planning for clinical trials needs to take this into account. It is important to integrate patients in the planning of clinical trials, and companies planning trials are encouraged to contact patient organisations early in the process to ensure this happens.

Challenges for EB patients taking part in clinical trials

  • Timeframe
  • Competing for ongoing trials
  • Exclusion criteria
  • Travel & cost
  • Additional clinical tests
  • Family commitments/bereavement
  • Misconception about cell and gene therapies

Recommendations for overcoming some of these challenges:

  • Include patients in the clinical trials design from the start
  • Carefully consider flexibility of eligibility criteria and ensure it is justified
  • Try and open some ‘shared care’ sites such as GP practices or hospitals or meet patients at their home
  • Help patients to book travel and hotels where possible and pay upfront from a study account
  • Have early, late or weekend appointments available where possible to fit around patients' schedules.

Share knowledge about patients' needs & perspectives

Owing to legislation, EB Resnet cannot provide companies with direct access to patient registers or contact details of individual patients. However, for companies planning clinical trials, opportunities to participate in trials can be communicated to the patient community via EB Resnet. EB Resnet does not provide endorsement of specific treatments, trials or products. Patient recruitment for clinical trials can be facilitated through clinical-network partners. EB Resnet can help companies understand patient perspectives.


Publication 2020:Profiling trial burden and patients’ attitudes to improve clinical research in epidermolysis bullosa. 

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