BIA members come together to improve patient access to medicines for rare and very rare diseases

A rare disease is defined as one that affects less than 5 in 10,000 of the general population and an ultra-rare disease affects less than one patient per 50,000 of the European population.1,2 In the UK, it is estimated that 1 in 17 people – approximately 3.5 million people – may be affected by a rare disease at some point in their lives.1 The impact that rare diseases have on patients, their families, and society is profound, as many are severe, chronic and progressive, with high mortality rates.3 

In England, there is no dedicated process to assess orphan medicines and uptake of these medicines is varied. According to the Office of Health Economics of the 143 orphan medicines that were available in the UK, only 68 of these medicines were reimbursed and made available to NHS patients in England.4 In contrast, 116 orphan medicines are reimbursed in France and 133 are reimbursed in Germany.5  

Commenting on the launch of the group, BIA CEO and Chair of the Rare Disease Industry Group, Steve Bates OBE said:

“The BIA has long been a vocal advocate of the need to improve access to treatments for patients with rare and very rare diseases. In 2014, the BIA published the report Very Rare Diseases, Complex Issues, calling for a separate approach for evaluation and commissioning of ultra-orphan medicines.6 The RDIG will now continue and build on the BIA’s previous work in this important area.” 

Peter Kuiper, General Manager UK and Ireland at Sanofi Genzyme, the specialty care global business unit of Sanofi, and Vice-Chair of RDIG said:

“There is strong public support for treating patients with very rare diseases as part of the comprehensive offer of the NHS. Almost three quarters of the UK public agrees that patients with a very rare disease should have the same access to treatment based on clinical need as patients with common diseases.7 The RDIG looks forward to developing recommendations and initiatives that can help move the dial on this important issue.”

 

Notes to editors

About the Rare Disease Industry Group

The Rare Disease Industry Group (RDIG) was established in July 2017. The members of the RDIG include Alexion Pharmaceuticals, Alnylam, PTC Therapeutics, Sanofi Genzyme and Vertex.

References

  1. Rare Disease UK. What is a rare disease? Available at: https://www.raredisease.org.uk/what-is-a-rare-disease/
  2. Regulation (EU) No 536/2014 of the European Parliament and of the Council of 16 April 2014 on clinical trials on medicinal products for human use, and repealing Directive 2001/20/ EC. Available at: http://eur-lex.europa.eu/legal-content/EN/TXT/PDF/?uri=CELEX:32014R0536&qid=1421232837997&from=EN
  3. EURORDIS. What is a Rare Disease? Available at: http://www.eurordis.org/sites/default/files/publications/Fact_Sheet_RD.pdf
  4. Office for Health Economics, Comparing Access to Orphan Medicinal Products (OMPs) in the United Kingdom and other European countries, March 2017 - https://www.ohe.org/news/important-variations-access-orphan-drugs-france-italy-germany-spain-and-uk
  5. Shire Report. Equity and Access: Making the UK a rare disease leader p.12. March 2017. Available at: http://www.haeuk.org/wp-content/uploads/2015/07/Shire-Equity-and-Access-Making-the-UK-a-rare-disease-leader.pdf
  6. BIA Report. Very Rare Disease, Complex Issues. March 2014. Available at: 

  7. BIA Report. Strong Public Support for Equal Access to Treatment for Patients with Very Rare Diseases. February 2014. Available at: 

     

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