The Innovative Medicines Fund (IMF) is a new initiative to fund access to novel therapies across multiple indications, including rare conditions and smaller patient populations.

It was introduced by NHS England on 6 June 2022, following a promise by the Conservatives to reform access to novel medicines in their 2019 election manifesto. It has been designed as an extension to the existing Cancer Drugs Fund (CDF), a pot of £340 million for accelerating access to novel cancer therapies established by the Coalition Government in 2011.

Since its inception in 2016, the CDF has been used to fund 91 medicines, treating over 73,000 patients across 205 different cancers. It was established as a response both to the increasing complexity and price of oncology drugs, the impact of delays in treatment with effective therapies, and the risk associated with the authorisation of therapies with limited data. The IMF is anticipated to mirror this success, whilst breaking the barrier of perceived bias for cancer patients over other therapy areas.

The proposition has received a warm welcome among stakeholders and will undoubtedly have a positive impact on patient access to novel treatments, particularly in the domain of rare diseases, where the treatment options are severely limited. It will also reduce risk associated with the reimbursement of innovative medicines by collecting real-world data during the five-year managed access period. However, is a fund which mirrors that of the CDF appropriate for non-oncology indications? The CDF, although successful, has certainly experienced setbacks – some of which led to its complete reform in 2016.

There remain several nuances in the management of rare diseases that may compromise the integrity of the IMF

There are fundamental differences in the treatment of oncology vs. non-oncology indications that mean certain approaches taken by the CDF are not translatable to the IMF. For example, gene therapy treatment is based on a single infusion of a drug that may lead to cure. Cancer is a dynamic and evolving disease, so patients benefit from multiple different therapies with shorter treatment intervals. The wholesale acquisition price of a single dose of Zolgensma, a gene therapy used to treat spinal muscular atrophy, is £1.8 million. This compares to a two-year course of Libtayo, an immune checkpoint inhibitor reimbursed via the CDF, which costs £161,754. Such treatment patterns are not unique in the oncology/non-oncology divide, it is therefore likely that the £340 million fund for the IMF is not sufficient and will limit the drugs available on the scheme.

Similarly, the diseases that innovative medicines are intended to treat are often rare. As a result, the collection of sufficient data for such treatments that will ease the clinical uncertainty will take longer than for the more common oncology indications. Therefore the proposed five-year time limit for data collection within the IMF may not be suitable for medicines intended for rarer conditions or those that provide long-term responses. Instead, the robustness of such data may be augmented by simulation modelling to provide longer-term trajectories.

As mentioned in its briefing on the IMF, the Association of the British Pharmaceutical Industry (ABPI) has suggested that a simple managed access scheme – as operated in the CDF – will not suffice for the IMF. Many innovative medicines have more challenges than a lack of clinical data, including the long-term uncertainty associated with drugs such as ATMPs. Therefore, the IMF represents an opportunity to explore the functionality and sustainability of innovative and adaptive reimbursement mechanisms to improve patient access and determine the true value of innovative medicines.

Innovative reimbursement mechanisms to enable faster access to innovative medicines, reducing inequalities in access

An important aim of the IMF is to reduce inequalities in access to innovative treatments through its use of diverse reimbursement mechanisms. By using the CDF as a comparator, we can understand how to model the IMF to optimise the reduction of inequities in access through the use of managed access agreements. One study showed that there was no difference in access to the CDF by deprivation, however, patients who were female, and those older than 85 years, were less likely to receive treatments from therapies within the CDF. The report also showed varied access to anti-cancer therapy according to regional cancer networks.

Akin to the CDF, the IMF is to be rolled out in England alone. Patients in Wales, Scotland, and Northern Ireland are not set to benefit from the fund – although the Scottish Medicines Consortium (SMC) has established a ‘new medicines fund’ which secures access for patients with rare or end-of-life conditions. Therefore, in its current form, regional inequalities are likely to persist with the inception of the IMF. Here at LCP, our Health Analytics team places a particular significance on reducing health inequalities and specialises in developing innovative approaches for this purpose.

The inception of the IMF is in line with the Government’s Rare Diseases Action plan, a framework detailing the priorities of patients whose lives are affected by rare diseases. Whilst the IMF is overwhelmingly positive, it is important that policy makers learn from the examples set by the CDF and ensure equitable access to patients across England, whilst providing a sustainable fund that targets the unmet need of patients who would benefit from an innovative medicine.