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Improving access to orphan medicines and collaboration

Identifying shared challenges

When I joined MAP in 2017 there was a recurring theme: many of our MAP Online members and clients looking to introduce orphan medicines for use on the NHS were faced with the significant ‘gap’ between the Single Technology Appraisal (STA) and Highly Specialised Technology (HST) programmes. Treatments referred to NICE’s STA programme faced much greater barriers which prevented timely access to new treatments. We worked with several companies individually to try to ensure an appropriate HST referral. It became apparent that so many companies were likely to be affected that we should consider pooling resources.

Why collaborate?

Working collaboratively helps to move the discussion beyond a particular product or disease area and strengthens the message. When I worked in the charity sector, I established a coalition that focused on support for children with health conditions in schools. The campaign eventually led to a change in the legislation, providing formal safeguards for these vulnerable children. It is unlikely wholesale change would have been introduced if we had continued to campaign as an individual charity representing one condition – there is strength in numbers.

MAP’s work on orphan medicines, supported by a number of MAP Online members, has allowed us to highlight systemic issues that affect a range of rare disease medicines. It avoids duplication as companies can support an overarching message outside any product-specific engagement they might be undertaking individually. This provides value for money for supporting companies and, importantly, allows MAP to play a key role in highlighting the collaborative concerns of our members with decision-makers and the wider stakeholder community.

Maintaining close dialogue with other industry groups and patient groups that are campaigning on these issues has also been an essential part of our work programme. Most recently, we have sought to establish a multi-stakeholder group through this work where we can share intelligence with the many patient groups that take an interest in this topic. This has brought to light fresh perspectives on NICE’s ongoing methods review and will inform our next phase of activity.


Our report Access to Medicines: A Case for Change was published in February 2019. The report and subsequent engagement have led to:

  •  One national and six pharmaceutical sector items of news coverage, including priority placement on major pharmaceutical news websites

  •  Eight mentions in Parliament, through questions, debates, Select Committee sessions and APPG meetings including active reference to our report by NICE’s then Chief Executive, Sir Andrew Dillon and NHS England’s Director of Specialised Commissioning, John Stewart

  •  At least eight references to our report in independent stakeholder documents, including consultation submissions, briefing documents, website pages and reports

  •  Meetings or teleconferences with 12 key stakeholders and decision-makers about the report, with 13 health charities, and eight Parliamentarians participating in a collaborative event in Parliament

  •  Recognition that there should be flexibilities in data collection for orphan medicines assessed via the STA programme and explicit reference to managed access agreements as an option for non-highly specialised or non-cancer medicines

    We believe that the collaborative approach has been instrumental in its success.

    What next?

    MAP continues its work to improve access to orphan medicines in the UK. MAP has been directly involved in NICE’s ongoing methods review and hopes that we will be able to ensure that further meaningful change is implemented to support timely access to new treatments.

    We are currently exploring the opportunity for a similar project in Ireland that would seek to address many of the challenges in the system that delay patient access to orphan medicines. Through our network of MAP Online members and clients, we are aware there are a range of overlapping interests. If there is a topic that you would like us to actively consider, please don’t hesitate to get in touch on


Tessa Hughes

Associate Director, Head of Policy and Public Affairs

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