The latest event in the LexHealth breakfast series brought together over twenty-five stakeholders, including patient groups and industry representatives, to discuss the changing environment for rare disease in the UK. Alastair Kent OBE and Sheela Upadhyaya shared their thoughts and engaged with attendees on a range of issues including priorities for patients, the commissioning landscape and complex challenges to market access.

Key themes raised at the breakfast roundtable included:

  1. The important role of patients – Services must fit around the patients that industry seeks to support, and patients also play an important role in the collection of real-world evidence and humanising data.
  2. Industry needs to demonstrate a deep understanding of patient needs – Companies must engage with patients to understand their experience, as well the outcomes that matter most to them.
  3. Collaborate to tackle rare diseases – Companies should capitalise on opportunities to share insights and help foster new ways of working to improve services for rare disease.
  4. How to navigate the commissioning ‘cliff edge’ – As the relationship between the NICE process for Highly Specialised Technologies (HST) and the Standard Technology Appraisal (STA) will likely remain complex and imperfect, companies should focus on simple discounts and a clear value proposition.

Companies must not neglect patient insights

Attendees began by discussed the rapid rate of change in specialised commissioning.  We have evolved from a system where very few drugs were manufactured for rare diseases. The NICE HST process originally envisaged 3-4 evaluations per year but this has been overtaken as more come forward for evaluation.  In fact, this has now increased 200 per cent, with eight to nine drugs coming through per year.  These new drugs provide very real opportunities.  However, while services for rare diseases have improved, too many patients are still left wondering whether they will be able to see a specialist or access a new treatment following diagnosis.

The reasons for this are varied but if companies want to provide new treatments, patient experience and the outcomes that matter most to patients must play a central role in product development. Companies need to demonstrate the value of their product, which should be based on understanding and responding to the priorities of the relevant patient population, including their perspective on what is important and what change they want to see. This should involve incorporating patient-reported outcomes as part of a wider process of collecting evidence on condition experience. These insights are critical to developing a clear story with a strong value proposition and unique offer to patients.

Collaborate to improve rare disease services

Brexit uncertainty has already weakened the UK’s previously close working relationship with the EU on scientific research. This has only strengthened the need for collaboration within the UK to improve service provision for rare diseases. While NICE is responsible for establishing the value a product delivers for the NHS, there are opportunities to share insights and suggest new ways of working. For example, attendees highlighted how the Office for Market Access and NICE’s Scientific Advice team are always keen to hear from industry before phase 3 trials, to help build the case for a new product. Beyond NICE, there are other partner organisations within and beyond NHS England, like Clinical Reference Groups, that also have an important role to play.

Challenges and opportunities in the commissioning system for rare diseases

Attendees discussed how the relationship between the NICE HST and STA processes is perceived to be imperfect and complex, particularly regarding the QALY threshold. There is a gulf for patients with orphan diseases because there exists a cliff edge between HST and STA. Attendees highlighted how often ‘orphan status’ does not mean that a product will be appraised through HST as it can be very challenging to meet the HST criteria. It was suggested there should be a sliding scale between £20,000 and £300,000 per QALY, however there was also a recognition of the pressures this would place on NHS budgets. Notably, there may be opportunities for flexibility in the new voluntary scheme. For example, there is commitment in this scheme to review NICE methods but it’s too early to say what the scope will be.

It was also discussed – in line with recent comments by NHS Commercial Medicines Director Blake Dark – how companies should first approach the discounting process by offering a straight discount. There still remains hesitance towards complex schemes and it was discussed how companies would be best placed offering a simple discount, while emphasising the value of the new product in terms of benefits to patients and the wider health system. Attendees also explored the need to build a disinvestment narrative, where new products that improve outcomes are adopted quickly and replace older, less effective products, which are removed from the system. This would likely also require education and/or training for healthcare professionals so they are aware of the changes to the prescribing process and stop prescribing ineffective treatments.

However, the future is promising

The breakfast event included discussion of a number of encouraging developments that suggest we should feel optimistic about the future of rare disease services in the UK. The appointment of Nicola Blackwood, herself a rare disease patient, as Health Minister, was welcomed by all attendees, as was her commitment to developing a National Genomic Healthcare Strategy and an overarching framework to replace the soon-to-expire UK Rare Disease Strategy. Whilst the NHS Long Term Plan does not mention rare diseases, it does encouragingly stress the importance of related issues including service integration, genetic testing, technological innovation and research. This is all in addition to ongoing UK rare disease collaborations, the evolution of Centres of Excellence and more.

Lexington Health is running a series of exciting events for the life sciences sector. To find out more, get in touch with our Director and Head of Health, Emily Cook: