Join our webinar: Rare diseases – Regulatory and reimbursement submissions, the challenges

Increasing numbers of rare diseases are being recognised and more medicines to treat them are being developed. There are several unique challenges for developing evidence packages in rare diseases such as the paucity of information on patient populations, disease progression and appropriate Clinical Outcome Assessments (COAs). As with all clinical development programs, disease-specific COAs are an important part of assessing signs and symptoms of rare diseases and treatment response.

On Tuesday 22nd March at 3:00pm GMT Adelphi Values, in association with PMGroup, will host a webinar to look 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.

Click here to register

Throughout the webinar we will explore:

  • Evidence needs for defining value in rare diseases
  • The overall challenges of collecting data to support registration and reimbursement, the challenges around creating evidence and reimbursement evidence packages, and special considerations associated with clinical trials and regulatory approaches in rare diseases.
  • The regulatory evidence needs and requirements for HTA/payer bodies for a positive value assessment and 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.
  • How patient access to rare disease treatments varies across regions and how to incentivise development.

The webinar will be hosted by Nikki Bonner, Associate Director, Patient-Centered Outcomes and Louise Heron, Director, Value Insight & Communication at Adelphi Values. We have also partnered with Alexandra Bowden PhD, Senior Manager at Ultragenyx Pharmaceuticals Inc, Denise D’Alessio MBA, Manager, Patient Reported Outcomes and Global Market Access & Policy at Novartis Pharmaceuticals Corporation, and Anne Kilburg MSc, Principal Consultant at Wellmera AG.

This webinar will be useful to professionals working in the pharmaceutical industry, academia, and consultancy who are involved in clinical trials, outcomes research, regulatory, commercial aspects of the drug development process, especially within rare diseases.  It will also be useful to those with an interest in these areas.

To attend the free webinar please register here – the webinar can either be watched on the day or later as a recorded version.

For more information please contact: contact us or email