The Department of Health and Social Care has announced pilots to test changes to the UK’s approach to access to medicines. The headline for our community is a new measure that will speed up access by permitting a set of treatments to go straight into a funded managed access agreement directly following their market authorisation. This could save months of waiting for communities urgently awaiting treatment for their unmet needs.
The Innovative Medicines Fund – £340 million to support managed access agreements has been under-used and felt out of reach as a solution to the access challenges our community face. This pilot could unlock the potential of the fund.
Genetic Alliance UK has welcomed the opportunity to be involved in the policy sprints that have led to this policy announcement today, and have used that opportunity to share the experiences our members have been facing advocating for access to innovative treatments.
Background
The major headline of the UK-US Trade agreement is the shift from the original single technology appraisal (STA) threshold of £20,000 to £30,000 per quality-adjusted life year (QALY) gained, to a new threshold of £25,000 to £35,000. NICE has said this is likely to lead to approximately five medicines a year being made available that otherwise would not have been approved. The original threshold has been in place since 2006, and adjusting that original threshold for inflation would put it at £35,559 to £53,338 today.
That change is significant, but is unlikely to change the picture for access to therapies for people living with genetic and rare conditions. The threshold for the process designed for very rare conditions affecting fewer than 300 people in England, the highly specialised technology programme (HST), has not changed, and the yawning gap in capability to assess rare condition treatments between HST and STA remains.
There is other news from the trade agreement coming though today, and Genetic Alliance UK has been participating in the ‘sprint’ policy development programme undertaken by the Department of Health and Social Care (DHSC) and Association of British Pharmaceutical Industry (ABPI). The announcements today will probably do more to address the needs of our community than the threshold shift alone.
What is the new approach?
The rapid policy development process means that four pilot approaches will be trialled between September 2026 and September 2027, each on a small cohort of selected treatments. If these prove valuable, then they will be transitioned to standard practice. Selection of medicines to go through the programme, and methods for assessing treatments remain to be confirmed. It is feasible for all four pilots to succeed.
Increasing adoption
What’s happening: For piloted medicines, there will be ring-fenced funds for regional spending, this will release local budget constraints.
Our view: This does not appear to be a key issue for rare condition treatments, which are normally delivered through nationally commissioned services.
Accounting for productivity
What’s happening: For piloted medicines, a new approach will be used to measure and understand the full benefit that a new medicine might offer society, such as enabling people who receive treatment to return to work.
Our view: Depending on the methodology and how uncertainty is managed, this could make a difference to Health Technology Assessments (HTA) for new treatments for rare conditions. Especially if this is the beginning of a programme to broaden the scope for the definition of the value of a treatment for rare conditions. The ability to recognise benefits to carers, changes in mobility and what that means to an individual’s independence, are essential considerations for how a new medicine should be valued that our community have felt to have been missing in past evaluations.
Speeding up access to new medicines
What’s happening: This would remove the requirement for a complete NICE evaluation in advance of a managed access agreement (MAA) for piloted medicines.
Our view: This is the pilot that stands to benefit people living with rare conditions most, providing rare condition treatments are prioritised for the trial period. The time between MHRA approval and a completed NICE appraisal for a rare condition treatment can be many months, during which time some people with progressive conditions will become ineligible for treatment. With this change, medicines could in theory go straight from MHRA approval into an MAA, saving time and saving lives.
The Innovative Medicines Fund (IMF) exists to fund MAAs, but the NICE appraisal process has been a significant hurdle and in some cases deterrent from setting up MAAs. This would unlock that unused source of funding for earlier access to medicines. In cases where MAAs are possible, this would also render the early access programme challenges that many of our members have been facing, moot, as the period between MHRA approval and NICE approved Managed Access would become negligible.
We would be keen to see clinician-led pragmatism be a major part of decision-making governing access to this pilot. We also understand that extra measures will be put in place to mitigate other less popular aspects of the Innovative Medicines Fund.
Recognising industry service
What’s happening: The budget impact test is a means by which the roll-out of medicines which have a high budget impact at a population level can be delayed. This pilot would permit investment in NHS infrastructure to deliver medicines effectively to be considered as a mitigating factor in applying the budget impact test.
Our view: This pilot scheme would only make a difference to our community with respect to a treatment for the most prevalent genetic conditions.
How we were involved
Genetic Alliance UK attended both the patient organisation face to face meetings with the DHSC to influence and inform the sprint policy development programme. We are also members of the Charity Medicines Access Coalition (CMAC) and have been participating in monthly meetings with DHSC and NHS England in that context.
Bigger picture
As we carry out our Future for Rare work, we are interested in how things fit together. How does the whole system work for individuals? How does the complete innovation pathway work? And is there an overview being taken to plug gaps and balance inequities?
While four pilots are never going to be a magic wand for system-wide challenges, the approach taken here addresses a major gap for our community. We understand there is potential for a NICE HTA Lab programme to address other concerns for our community too, and we are keen to continue to work to keep connecting our community’s voice with these programmes.
