• Many countries are looking to reform their pricing and reimbursement (P&R) systems for rare diseases and are soliciting input from manufacturers as part of this process

The Challenge

  • Lack of consistency in payer assessment systems for rare diseases across countries globally
  • Client needed a coherent policy position on the most appropriate mechanisms and processes for value assessment and P&R decisions for orphan medicines

Our Solution

  • Reviewed existing value assessment and P&R systems for rare diseases across key markets globally
  • Deconstructed different components of systems and assessed patient access
  • Identified components of P&R processes that help facilitate patient access and those that act as barriers to successful outcomes
  • Reviewed rare disease policy literature for recommendations on best practice for orphan drug value assessment
  • Worked in collaboration with key client stakeholders to define a framework of key principles for value assessment and funding for orphan drugs
  • Work was summarised and published in a peer-reviewed journal


  • Based on this work, our client engaged with a European-wide policy initiative to establish a common set of principles for assessment of rare disease treatments
  • At a cross-industry meeting in 2016, Dolon assisted in facilitating workshops on the topic and assisted in driving European-wide consensus