Skip to content
Page last updated: 21st December 2022

Complex regulatory systems in England haven’t been favourable for rare conditions. Without change, future potential access is limited. We and other rare condition groups have made our voices heard as changes to the systems managed by NICE have been consulted and implemented. We continue to monitor the impact of the changes.

What were the proposals?

Within the last five years, the SMA Community has gone from having no treatment options to having three. This advancement has only been made possible through fast-tracked treatment pathways that have identified innovative drugs in the pipeline. Innovative drugs are drugs that meet an unmet need, treatments which can demonstrate substantial clinical promise. These drugs often have significant uncertainty around their clinical and cost effectiveness due to smaller patient numbers. Managed Access Agreements (MAAs) are established between NHS England and the pharmaceutical companies. The drugs are often purchased at a reduced rate, patients and their clinicians have to agree to data collection activities that will build evidence, assessing the ongoing progress of the drug, how safe, effective and cost effective it is.

The government are now proposing to fund this pathway through an Innovative Medicines Fund (IMF), £680million per year will be ringfenced for innovative medicines. This way of funding medicines is already established and has been tried and tested by the Cancer Drugs Fund (CDF) which successfully fast tracked new medicines to cancer patients. The CDF, that stood at £340million per year will be absorbed into the IMF and an extra £340million will be added supporting patients with any condition, including those with rare and genetic diseases, to get early access to the most clinically promising treatments.

What did SMA UK say?

NICE Systems Review: Innovative Medicines Fund >

What was the outcome?

NHS England’s Consultation Outcome >

In this document, Zolgensma™, the one off gene-therapy for SMA, is referenced as an example of how successful the mechanisms behind the IMF can be at bringing innovative medicines to the public as quickly and as safely as possible.

SMA UK received this response to our submission:

"Thank you for taking the time to engage with the consultation on proposals for the Innovative Medicines Fund (IMF):

Today, NHS England and NHS Improvement (NHSEI), in partnership with the National Institute for Health and Care Excellence (NICE), are publishing our response to consultation and formally opening the IMF.

112 responses to the consultation were received, including from stakeholders in industry (37%), NHS (24%) and patient groups & charities (18%). There was widespread support for the purpose of the Innovative Medicine Fund with 96% of respondents either strongly agreeing, or agreeing, with the purpose of the IMF. Alongside our response to consultation, a new set of IMF Principles set-out the policy approach to the delivery of the Fund, providing clarity on the primary role of the IMF, to enable faster patient access to innovative new treatments

Building on the success of the Cancer Drugs Fund (CDF) that has delivered faster access to cancer treatments for over 80,000 patients, the IMF will support faster access to non-cancer drugs and, alongside the CDF, provide a total of £680 million ringfenced NHS funding for innovative medicines.

Thank you once again for helping to shape the development of the IMF."

We were grateful to the Genetic Alliance UK and Charity Medicines Access Coalition (CMAC) for all their work on this – it was huge. Also the Specialised Healthcare Alliance (SHCA) and many other groups.

We joined many meetings and contributed our voice at them and as follows:

  • NICE Methods Review: 6-week public consultation started in November 2020. We attended a number of consultations and discussions run both by and for Patient Groups. You can read our December response to the consultation.
  • NICE Case for Change Proposals – Processes Review​: In April 2021 we made our response to this part of the consultations.
  • Draft programme manual: 8-week public consultation started in August 2021 – we attended consultations and discussions and submitted our response in October 2021.

New guidance manuals were finalised and came into effect on February 1st 2022.

There were some positive changes but not far enough to meet the needs of the rare disease community. You can read why in these summaries below:

In 2021, we were involved in an international project led by IMPACT HTA looking to formulate best practice.