Fast-Tracking Innovative Medicines
This Rare Disease Day, there is a lot of attention being paid to the Innovative Medicines Fund (IMF) announced by the Government last year and how it will benefit patients across the UK. The IMF aims to help fast-track cutting-edge drugs to patients, giving them early access to some of the most clinically promising treatments in need of further data for NICE to recommend routine use in the NHS. Designed to build on the Cancer Drugs Fund (CDF), the IMF is designed for the purpose of improving access to rare disease treatments, as well as others beyond.
In a recent White Paper entitled The Innovative Medicines Fund – A Catalyst for Access to Rare Disease Medicines produced by IQVIA, Alexion and AstraZeneca, thought leaders across the UK medicines and rare disease community came together to consider how the IMF can transition from the CDF and question how the fund can be effective in its goal of getting promising treatments to patients.
access to rare disease treatments
As it stands, patients with rare diseases in the UK generally find the medicines approval process for rare disease treatments to be far too slow and consequentially unfair on those living with rare diseases. One of the key reasons for this are the stringent requirements under the Highly Specialised Technology Programme, wherein large numbers of treatments fail to secure NICE approval for wider roll-out in the NHS. Consequentially, the uptake of innovative medicines in England is much worse than that of our European counterparts.
Turning Policy into Practice
While no single initiative will be able to solve the issue of uptake in England, there can be little doubt that the IMF is one step in the right direction. If it successful in its ambition of providing greater clarity to the data for rare disease treatments when they come to be assessed by NICE, then it could have transformative impacts for patients with rare diseases in the UK.
To this end, the White Paper makes a series of recommendations, to ensure that doctors across the UK can use the most advanced, life-saving treatments that are available to them, and to ensure that patients suffering from rare diseases are not left behind. The expansion of the CDF into the IMF represents a fantastic opportunity for the rare disease community – it provides a new route for funded access to promising rare disease medicines that was previously only available to cancer drugs. For this reason, the first recommendation of the paper was unsurprisingly that:
“Rare disease medicines must have the same opportunity for IMF funded access as a medicine for any other disease.”
A further issue to consider with the IMF, if it is to be truly equitable in the opportunities it offers to the rare disease community, revolves around the funding mechanisms according to which it operates. It is the recommendation of the White Paper that available funding is neither ringfenced according to medicine type or disease area, nor operating on a ‘first-come, first-serve’ basis.
“For this to work, the fund must be sufficient to accommodate all qualifying medicines and must not operate on a ‘first come, first served’ basis which would disadvantage medicines on the basis of their development timing.”
Final Thought
The IMF clearly presents an opportunity to catalyse access to rare disease treatments. However, the IMF does not exist in a vacuum, and much will depend on how it is aligned with other initiatives such as the Accelerated Access Collaborative, the Early Access to Medicines Scheme as well as the review processes of NICE.
Similarly, if the IMF is to be truly equitable in its roll out, concerted attention will need to be paid to Rare Disease Action Plans across the four nations and the ways in which they can work together to ensure that promising new treatments can be accessed, regardless of where a patient lives. It will be centrally important that the four nations work to share learnings such that they can navigate decisions across their respective Health Technology Assessments.