News & Events

Funding treatments for rare conditions

Last Reviewed 23/01/2017

By Nick Meade

The past few weeks have seen the closure of consultations on two sets of dispiriting proposals from NHS England and the National Institute of Health and Care Excellence (NICE), which combined have the worrying potential to further damage the environment for rare disease treatments in England. These proposals have been compounded by consultations that were in places, flawed and disingenuous.  

NHS England and NICE have conducted a joint consultation looking at NICE’s Highly Specialised Technologies (HST) programme which evaluates highly specialised treatments and decides whether NHS England should fund them. The second consultation was from NHS England looking at their ‘generic policies’ – a particularly confusing name for four policies that describe how NHS England will make decisions on funding for treatments that are not currently routinely commissioned: the In- Year Service Development policy, the Individual Funding Requests policy, the Funding for Experimental Treatment and Unproven Treatments policy and the Continuing Funding After Clinical Trials policy.

Many of the proposals put forward in both these consultations pose a potential for negative impact on the patient community. The proposals for changes to the HST process, as they stand at the moment, may well put us in a situation where no treatment that has gone through the process since its inception in 2013 would have received compulsory funding under the new policy. The generic policies consultation had similar potential for negative impact on the patient community, and ignored the outcome of previous engagement with the patient community over the last few years.

You can take a look at our responses to both the consultation on the HST process and the generic policies for details on these damaging proposals.

The HST consultation put forward three distinct proposals, some with the potential to heavily impact on rare disease treatment, but each only peripherally connected to one another. The decision to consult on them together produced a single consultation more complicated than necessary, and will likely result in a higher number of questions to which respondents answer from a disinterested perspective– as different proposals will be relevant to different communities. This will dilute any message coming out of the process.

More worryingly (as we discuss in more detail in our response) the impact of the proposal to remove the funding commitment from the highly specialised technology programme was not clear in the consultation document. It was only because of the frank answers of the NICE team at a consultation event that the stark impact was fully apparent. The impact of proposals should be a central part of any consultation which examines them. Without this clarity the process is neither open nor transparent.

We were concerned to find a similar level of accessibility in NHS England's consultation on generic policies. The good news that we finally have proposed replacements for a set of interim generic policies first published in 2011 was tempered by the opacity of the new proposals.

Much of the detail on the new proposals was bound up in a standard operating procedure that was neither linked to, nor correctly named in the consultation. (The Individual Funding Request Standard Operating Procedure is currently still interim.) NHS England does not have to consult on standard operating procedures. Many of the other proposals were very vague, with many junctures at which NHS England would ‘decide’ on an issue, with no process, responsible individual or committee specified. All of these concerns are amplified by the gravity of the proposals.

The APPG on Rare, Genetic and Undiagnosed Conditions will be continuing its work on access to medicines this year, and we expect them to examine these new proposals in the context of the wider environment. Genetic Alliance UK will be doing more too, so please get in contact with us if you would like to participate in or shape this work.

We want to keep the debate going on these proposals by NHS England. With Brexit on the horizon, it is a time to look for opportunities and find space to make improvements to current processes. We need the UK to become a positive place for research and development of treatments for rare and genetic diseases. We know though that the funding environment for treatments is a significant portion of the environment that must be built openly, transparently and positively. These proposals take us in the wrong direction.

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