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25 senior stakeholders with an interest in rare diseases met yesterday morning for an informative and interesting BIA-led and organised roundtable discussion in the House of Commons, chaired by Andrew Miller MP, Chair of the Science and Technology Select Committee.

Joining Andrew Miller were the Rt Hon Stephen Dorrell, Chair of the Health Select Committee, Iain Wright MP, Shadow Business Minister, and Lord Turnberg who were able to share their expertise, views and pose a number of interesting policy questions to the attendees.

The meeting was called to discuss “rare diseases, complex issues” with a particular focus on the new arrangements for evaluating and commissioning Highly Specialised Technologies (HSTs) across England and the devolved administrations following changes to the structure.

The depth and quality of discussion, and the wide range of issues covered, was certainly helped by having all the key stakeholders present including representatives from the HST teams in NICE and NHS England together with industry, patient groups and other policymakers.

A wide range of important issues were discussed. Some of the key points touched upon included:

  • A shared and agreed belief that NICE should adopt best practice from AGNSS (the body which formerly evaluated HSTs) to ensure, amongst other issues, that patients are adequately communicated with and engaged. Joining together the service delivery element of the HST programme (dealt with by NHS England) and the evaluation (handled by NICE) is an important factor
  • A belief that patients need to be suitably empowered to engage with the process and make their voice heard. This is particularly important in rare diseases where we have small, often identifiable, patient populations. Accurately reflecting patient need and patient choice needs constant attention and refining
  • The research and development of orphan and ultra-orphan products has a number of unique factors and characteristics that need to be taken into account. Recouping of significant expenditure can only be achieved over a small patient population, manufacturing processes are complex and often need to be entirely bespoke, and there is often a paucity of disease data and knowledge
  • There is a continuum of diseases affecting differing patient population sizes – not just HSTs and more general conditions. Rare diseases offer a window into the world of the challenges that will be faced as more personalised medicines are approved targeting specific subsets of wider populations.