The Accelerated Access Review (AAR) is being undertaken by the Department for Life Sciences, with th…
On 3 November 2017, the Government published its response to the Accelerated Access Review. This was a short document, broadly endorsing the recommendations from a year ago, and publishing a timeline for implementation.
In March 2015, the Accelerated Access Review process started. It was responding to a problem – getting innovative treatments into the NHS takes too long – that has become significantly worse in the past 30 months. The final report of the review was published a year ago; a 70 page document that made a broad range of recommendations. Though the recommendations themselves were positive, offering possible solutions to speeding up access to innovative products, the way these sat with the broader system was the key problem.
There are fifteen different routes to access a licensed medicine for a rare genetic condition in the UK – see list at the bottom of the page– and these are used differently by each of the four nations in the UK, making the pathways even more complicated.
A small fraction, around 3.5 million people, of the UK population will be affected by a rare disease at some point in their lives. There are more than 6,000 rare diseases (almost all single gene disorders are rare), but only 200 of these have a licensed innovative medicine in Europe. The reality is that we have fifteen pathways to assess treatments for a small fraction of a small fraction of the UK population. They all work in different ways, and sometimes we have four separate decisions being made on the same treatment – not all of them reaching the same conclusion.
So is the central proposal of the Accelerated Access Review – a new Accelerated Access Pathway – the right solution to our problem? There will be another route added to the list, one that will be treated differently by the different nations of the UK. We will have a new part of an already complex system that will be open to scrutiny and argument. The selection of treatments that go into this route will be delivered by the Accelerated Access Collaborative, while selection for all the other pathways in England is delivered by NICE’s relatively opaque topic selection process. (You can see here that every topic selection decision has a virtually identical basis.) The interaction between the two decision-points will be important.
Worse, the pathway is intended to be cost-neutral or cost-saving. This will almost rule out treatments for rare genetic conditions, not necessarily because there are no cost-neutral or cost-saving treatments for rare genetic conditions, but because current methods cannot show all of the value for these treatments. The full costs and benefits associated with different models of care for patients with rare conditions are unknown. Health technology appraisal methodologies consistently fail to take all of the impacts of a rare condition, and consequently the value of the treatment, into account. Part of the problem is the scale of unmet health need. Rare conditions devastate families for generations. Unless we quantify this devastation and consider it to be a cost to us, these treatments will never be considered cost neutral.
The proposed new route is by no means the only proposal here, there is a welcome streamlining measure to transfer the responsibility of agreeing Patient Access Schemes from the Department of Health to NHS England, for example.
The premise of both the final Accelerated Access Review and the Government’s response are more pertinent now than ever. We need to foster innovation within the UK so that our patient community can participate in research and our health services can evolve with the newest innovations. To do this we need to link innovation with access in the NHS. Genetic Alliance UK believes that the UK needs a bigger shake up to the system than that offered here – we do not think that these changes will truly be felt by patients and families living with genetic conditions.
The fifteen routes for funding approval of a rare disease treatment in the UK:
NICE’s Fast Track Appraisal process has not yet been used for a rare disease treatment.
If you have any questions about this article, please contact our Policy Officer, Louise.