Duchenne UK has established Project HERCULES to bring together a consortium of global companies developing therapies for Duchenne Muscular Dystrophy (DMD) to build an evidence base and develop common tools to support better decision-making on the pricing and reimbursement of new treatments of DMD.
Pharmaceutical companies wanting to introduce new treatments for patients in many countries are asked to submit dossiers to processes of Health Technology Assessments (HTA) agencies and payers. For example, in the UK the National Institute for Health and Care Excellence (NICE) and the Scottish Medicines Consortium (SMC) are likely to review all new treatments for DMD. These assessments determine if a new treatment should be funded by the NHS and made available to patients.
Developing evidence for HTA dossiers is particularly challenging in rare diseases such as DMD given the sparsity of available evidence to support the requirements of HTA agencies. Many pharmaceutical companies are developing potential treatments for DMD and are working individually to develop their approach to HTA. The variety of methods in use and the difficulties of generating data can lead to delays in introducing new treatments and inconsistent decision-making.
In January 2017, at a meeting organised by Duchenne UK, several companies expressed an interest in working together with Duchenne UK to develop key tools to support HTA processes. Project HERCULES has since been established to take forward this work. Project HERCULES will have two phases. The planned outcomes for each phase are described below.
- Data sharing platform to share the project outputs including a systematic literature review and burden of illness data.
- Data analysis to support HTA and reimbursement decisions including a review of current data and mapping of trial endpoints to clinical outcomes and natural history data.
- Quality of Life metric - a review of current metrics and their suitability for use in DMD; the development of a de novo metric or add-ons to an existing metric; and the use of the metric to generate scores for the relevant health states.
- A core economic model, adaptable by individual companies, to support HTA and reimbursement decisions.
- A burden of illness study to map the impact of DMD on patients, families and carers.
- Other advice, potentially including guidance for approaching managed entry agreements.