Director of Value and Access Policy, ABPI (The Association of British Pharmaceutical Industry)
There are many new treatments for rare diseases in the pipeline, which could transform people’s lives and potentially provide cures. Now health systems across the UK need to get better (and quicker) at making these available to patients.
In January, the four UK Governments published the Rare Disease Framework with four important priorities: help patients get a final diagnosis faster; increase awareness among healthcare professionals; better coordinate care; and crucially, improve access to specialist care, treatments and medicines.
The pandemic has shown us how quickly industry, academia, health services and regulators can move to research, develop and approve new medicines and vaccines. We need to build on that energy if we are to truly realise the goals set out in the Framework.
Getting new treatments to patients quicker
In England, right now, we have two golden opportunities to improve access to new rare diseases medicines: the ongoing NICE methods review and the new Innovative Medicines Fund (IMF).
NICE has the incredibly important job of deciding whether new medicines should be made available to patients in England, but the ‘methods’ by which it does this need updating. It is important that the review enables NICE to introduce more flexibilities into its approach for assessing rare disease medicines.
The pandemic has shown us how quickly industry, academia, health services and regulators can move to research, develop and approve new medicines and vaccines.
The Innovative Medicines Fund is aimed at ensuring doctors can use the most advanced treatments, including for rare diseases that affect children.We need the Fund to deliver on that promise.
Commitment from industry to support
The creation of the IMF, together with the NICE Methods Review, could not come at a more important time. Furthermore, as new medicines are developed, industry has committed to ensure their introduction is affordable to the NHS.
Through the ‘Voluntary Scheme for Branded Medicines Pricing and Access,’ NHS spend on branded medicines is capped, with the industry reimbursing all expenditure above agreed growth levels. Since 2019 companies have paid back over £1billion, giving financial certainty to the NHS.
The pharmaceutical industry stands ready to play our part in securing the changes required to ensure those with rare diseases and their families across the UK can benefit from quicker diagnosis, better care and better treatments in years to come. The Rare Disease Framework sets that challenge – together we must deliver it. We owe people with rare diseases nothing less.