Rare diseases – Regulatory and reimbursement submissions, the challenges


This webinar, hosted by Adelphi Values, in association with PMGroup, looks at the importance of and challenges associated with the development of evidence such as suitable endpoints, COAs and comparators in rare diseases using real-world examples.

Nikki Bonner, Associate Director, Patient-Centered Outcomes and Louise Heron, Director, Value Insight & Communication at Adelphi Values, host the webinar and are joined by industry partners  to explore:

  • Evidence needs for defining value in rare diseases
  • Data collection challenges in supporting registration and reimbursement, creating evidence and reimbursement evidence packages for rare diseases, and special considerations associated with clinical trials and regulatory approaches.
  • HTA/payer body requirements and associated evidence needs for reimbursement.
  • The challenges associated with COA development and how these can be overcome, including an example of a healthcare professional-administered COA for assessment of physical functioning limitations associated with a rare genetic disease.
  • Regional variations in patient access to rare disease treatments and how to incentivise development.

For more information on the webinar series, or our rare dissease expertise, please contact us or email info@adelphivalues.com

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