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Navigating the Highly Specialised Technologies (HST) programme workshop

Our first workshop of 2016 explored NICE’s Highly Specialised Technologies (HST) programme, focusing on the role rare disease patient groups can play.

The number of treatments (orphan drugs) being developed for rare diseases is on the rise. In the USA, FDA orphan designations have steadily increased year on year since 2000 (EvaluatePharma – Orphan Drug Report 2014). While this is great news, these orphan drugs come with large price tags. For example, Soliris (Eculizumab) recently made headlines, costing £10 million per patient. This is highly problematic for payers, as they have to decide whether these treatments are worth the cost.

Since 2013, the National Institute for Health and Care Excellence (NICE) has been doing just this: evaluating high cost drugs for patients with rare conditions. They provide robust and independent assessments, which guide NHS policy. This is usually done through their Highly Specialised Technologies (HTS) programme. To help rare disease patient groups better understand this process, we co-organised a workshop with NICE this past Friday. Over 50 delegates joined us and our five speakers to understand how to navigate the HST programme.

Eli Gajraj - Senior Technical Adviser, NICE Scientific Advice

The afternoon opened with a presentation by Eli Gajraj from NICE, who introduced the work of NICE and the process through which it considers health technologies. He shared the importance of cost-effectiveness of new treatments to NICE: in terms of both the financial cost and the opportunity cost, as funding one treatment may prevent funding for another therapy. He also delved into the other areas NICE values, including innovation, reducing health inequalities, and achieving broader social benefit beyond health objectives.


Sheela Upadhyaya - Associate Director, HST NICE

Eli then handed over to Sheela Upadhyaya, who is an Associate Director on the HST programme. Sheela focused in on the process through which NICE select treatments to consider on the HST programme, and specifically how patients and patient organisations can contribute to the evaluations carried out. She highlighted how patients are crucial to helping the evaluation committee gauge the burden on the disease, the impact of treatment, and the likely uptake of the drug if approved.


Ron Akehurst - Strategic Director, BresMed Health Solutions

Our next speaker was Ron Akehurst, who is a current member of the HST committee. Ron walked us through the elaborate process the HST committee work through when evaluating a treatment. He shared that there is more patient influence on the committee than any other he has experienced. Patients and patient groups are invited to join public sessions, and are given opportunities to present, even after points when clinicians and manufacturers are excluded. However, he stressed that while patient views are important, the committee is not swayed by emotional pleading. Instead, content is vital.


Len Woodward - Trustee, aHUS UK

After a break over tea and cakes, the workshop continued with a presentation from Len Woodward, a trustee of aHUS UK. Len is intimately familiar with the HST programme, as aHUS was the first rare disease to go through the evaluation process for the drug Eculizumab. Their journey started at the first aHUS Patients and Families Conference in June 2011, where it was revealed that Eculizumab was being considered by AGNSS (NICE’s predecessor). It took until April 2015, following aHUS UK’s establishment, the conduction of patient interviews, moving from AGNSS to NICE, and lots of roadblocks around “affordability”, until Eculizumab was approved at £10million per patient. Len’s presentation contained some key learnings and lessons for other groups considering getting involved in the HST programme.


Nick Meade - Director of Policy, Genetic Alliance

Our final speaker was Nick Meade from Genetic Alliance UK, who presented a consolidated patient perspective on the HST process. Genetic Alliance UK conducted a Patient Charter on the process in 2014. The charter was endorsed by 78 patient groups, and held key recommendations for NICE. The Charter found that patients are concerned that, while greatly needed, the HST process is opaque, potentially disruptive to the wider NHS, and risks downplaying the patient voice. However, Nick thought that NICE had responded well to the charter, and already made some changes that it suggested. Nick especially focused on the lack of transparency, showing that of 129 treatments considered by the HST, 123 had identical public justifications for moving forward.

Nick’s focus on the lack of transparency formed the initial point of our final session of the afternoon: the panel discussion. The panel and delegates discussed the importance of patient representation on the HST committee, the need for pharmaceutical companies to be more flexible around pricing, and the influence of media campaigns on the committee (the panel stated this does not influence decisions). It was a very lively discussion, carrying on until the workshop’s completion.

We would like to thank all the speakers for their informative presentations, the NCVO for the venue and catering, and all of our delegates for participating.


Navigating the Highly Specialised Technologies programme

by Libbie Read time to read: 7 min