28 February 2018

Rare Disease Day Sheela Upadhyaya

To mark Rare Disease Day (28 February), Sheela Upadhyaya explains how NICE’s Highly Specialised Technologies (HST) programme has helped to ensure that drugs for very rare diseases are evaluated appropriately and, if approved, are offered to patients who can benefit from them as part of NHS England’s arrangements for specialised commissioning.   

Picture of Sheela Upadhyaya.

Key principles

NICE began evaluating drugs for very rare conditions in 2013 following NHS reforms. And although there are a number of differences between the HST programme and our technology appraisal programme for mainstream drugs, it shares the key principles that define how we work. These include transparency, inclusivity, independence, scientific rigour and timeliness - principles that have been fundamental to our success and which are valued highly by our stakeholders.  

Challenges

There is no doubt that new treatments have brought about major improvements for patients with rare diseases in recent years, but they have brought with them challenges for payers such as NHS England.

The particular challenge for our HST programme is how to ensure decisions on these high cost technologies are made consistently and fairly. New methods for evaluating HSTs introduced last year aim to secure the balance needed between value, access and affordability for the NHS. We now evaluate these treatments against a sliding scale, so that treatments deemed to provide significant QALY gains (a way of measuring benefits to patients) but with an incremental cost of more than £100,000 per QALY, up to a maximum of £300,000 per QALY, can still be recommended for routine funding.  

So far the HST programme has published guidance on 7 treatments, all of which have been recommended for routine commissioning. These include strimvelis for ‘bubble baby syndrome’, only the second gene therapy for an inherited disease to be licensed anywhere in the world, and ataluren for treating Duchenne muscular dystrophy.

Solutions

The very nature of treatments for very rare diseases meant that each one of these evaluations had its own unique challenges calling for innovative solutions. Because they are targeted at such small populations – sometimes only a handful of people - the evidence on how well a drug works can be limited. With the short duration of follow-up studies we also lack the data to show what happens in five or ten years. And in many cases we don’t even have the information about how a particular disease progresses without treatment.

To address these challenges we encourage companies to seek solutions that will help manage both the clinical and the financial risks while at the same time allowing patients to access their treatments within the NHS. These commercial arrangements have typically included discounts to the price of the treatment, a commitment to collect additional evidence on how well the treatment works, are time limited and have to be agreed with relevant stakeholders

The future
Our HST programme has a full pipeline of technologies to evaluate in the future. And like the technologies we’ve already evaluated, each one will call for close collaboration with the companies who develop them, the clinicians who want to use them, and the patients who might benefit from them.

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