New report from MAP BioPharma on access to orphan medicines

MAP BioPharma (MAP) has published a report on access to orphan medicines in England. The report, Access to Orphan Medicines: A Case for Change, was developed with support from a Steering Group* of seven MAP Online members.

The report recommends that NICE should adapt its STA processes to enable more flexibility. It found that, whilst the National Institute for Health and Care Excellence (NICE) can accommodate for the rarity of the disease, to an extent, through its Highly Specialised Technology (HST) appraisal, many rare disease medicines do not qualify for an HST.

Furthermore, the STA criteria fails to take into account the nature of rare disease medicines both in terms of the evidence base, unmet need and cost, according to the report.


Other findings from the report include:

  • of the 24 completed STA reviews of rare disease medicines between 2013 and 2017,
    only 13% were recommended for the full eligible population, compared with over two thirds of non-orphan medicines
  • in the same period 50% of rare disease medicines were given a “restricted recommendation”, compared to 21% of other medicines
  • only six non-cancer orphans were reviewed by STA. Of these, only four appraisals were complete and none were recommended within their full marketing authorisation, compared with over two thirds of non-orphan medicines


Christian Hill, MAP BioPharma CEO said:

“MAP’s research illustrates that the current processes are not fit for purpose and we urge NICE, NHS England and the Government to look into how they can improve patient access to rare disease medicines.”

Commenting on the report, Jayne Spink PhD, Chief Executive, Genetic Alliance UK said:

“This report helps to show the systemic failings in our decision-making systems for access to rare disease treatments. This system will continue to fail people living with rare diseases until the fundamental challenges of assessing rare disease treatments are better addressed. We at Genetic Alliance UK, our 220 patient organisation members, and our community look forward to ensuring patient voice plays a crucial role in making positive changes.”

Access to Orphan Medicines: A Case for Change can be accessed via MAP’s publications page.

* The Steering Group members are Amicus Therapeutics, AveXis, bluebird bio UK, Chiesi Limited, Gilead Sciences, Inc, Kyowa Kirin International and Santhera Pharmaceuticals. Each company has made an equal financial contribution to support this work. MAP retains editorial control.

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Published 19. February 2019 in News, News UK