Orphan Medicines: Special treatment required?

Leela Barham

This report was prompted by our concern that patients and their clinicians are not always able to access medicines for those with rare diseases. The ongoing reforms to the NHS and development of a strategy for rare disease offer an opportunity to explicitly consider access to medicines for those with orphan or ultra-orphan conditions. As the commissioning of services for those with rare diseases moves to be undertaken by the new National Commissioning Board we hope that this report will be timely.

We analyse the complex framework that influences patients’ access to orphan drugs, in particular focusing on the role of R&D, regulatory and payer/commissioner decisions and the links between decision making along the pathway to access. In doing so we seek to make suggestions for better decision making and how to improve access for all to orphan drugs.

We draw on examples from the literature, explore several case studies in detail and take lessons from the situation in other countries. During this project we also benefitted from a mix of telephone and face to face interviews and discussions with patient representatives, clinicians, commissioners, pharmacists, health economists and companies. 2020health would like to thank all those who freely gave us their data, knowledge and expertise.

This report was funded by an unrestricted educational grant from Pfizer, Shire and Genzyme, giving us freedom to independently draw our own conclusions. We are greatly indebted to our sponsors. As well as enabling our on-going work involving frontline professionals in policy department, sponsorship allows us to communicate with and engage officials and policymakers in the research that we do. Involvement in the work of 2020health is never conditional on being a sponsor.

Impact:

• Contributors felt that this research ‘moved the debate on’ for patient access to drugs for those with rare disease
• An important reference document as the debate on access to ‘orphan’ medicines continues.
• Sara Morgan, Policy and Special Projects, Myeloma UK says: “A very comprehensive analysis of all the factors involved in decision making around orphan medicines. I’m sure its recommendations will be very well received.” (see below)

We just wanted to drop you a line to congratulate you on the excellent report you produced for 2020 Health.

I’m just reading through it now and it’s a very comprehensive analysis of all the factors involved in decision making around orphan medicines. I’m sure its recommendations will be very well received.

Sara Morgan, Policy and Special Projects, Myeloma UK