Frameworks for promising therapies with less mature evidence base to secure adoption

Recent reforms in regulation (e.g. Adaptive licensing, Conditional Market Authorisation, Early Access to Medicines Scheme) hold the promise of faster and more efficient regulatory approval processes but can also result in technologies reaching the market with a less mature evidence and therefore with greater uncertainty about their clinical and cost-effectiveness than previously. Current assessment frameworks used by health technology assessment bodies and UK payers are not geared to deal effectively with such uncertainty, therefore the existing frameworks present a hurdle to the reimbursement and adoption of these technologies. This is further exacerbated in the case of cell and gene therapies which are often suggesting long-term benefits arising from finite number of treatments, however their supporting data at launch tends to cover shorter-term horizons.

Fortunately in the recent months, UK key market access stakeholders with input from the industry have engaged in numerous and complimentary initiatives that provide the opportunity for a more favourable adoption environment for promising therapies with immature evidence base at launch. These include:

  • The development of frameworks by the Decision Support Unit (DSU) of The National Institute for Health and Care Excellence (NICE) for analysing risk in health technology assessments (published January 2016). These proposed frameworks built further on the existing NICE methods guide by including quantification of the magnitude of the uncertainty and impact to the NHS in health and monetary terms; they also inform how Managed Entry Agreements (also known as Risk Sharing Schemes or Patient Access Schemes) can be leveraged to mitigate risk through price modulation and further research/evidence generation.
  • A study of the appraisal of a hypothetical exemplar regenerative medicine product was commissioned and published by NICE in collaboration with the Centre for Reviews and Dissemination/Centre for Health Economics, University of York. The study establishes the appropriateness of current NICE methods as a basis for the evaluation of regenerative medicines and makes suggestions on how the same methods can be leveraged to also quantify the impact of uncertainty to the NHS and identify the optimal strategy for risk mitigation; the ways that uncertainty is quantified and mitigated in this NICE report aligns to the proposed framework by the DSU described above. It should be emphasized that this study is not meant to inform on typical pricing benchmarks for cell and gene therapies as it was based on a hypothetical highly efficacious therapy that prevents young sufferers of acute lymphoblastic leukaemia from dying and enables them to recover; therefore this hypothetical therapy results in a remarkably large gain in quality-adjusted life years.
  • NHS England and NICE have proposed a new Cancer Drug Fund (CDF) operating model (consultation period closed February 2016). According to this proposal novel oncology therapies which appear promising but where NICE indicates that there is insufficient evidence to support a recommendation for routine commissioning, would be given a conditional reimbursement and their use will be enabled by the CDF for a pre-determined period whilst further evidence is collected. At the end of this period the therapy would undergo NICE appraisal, using this additional evidence so that its suitability for ongoing reimbursement is established.
  • The Accelerated Access Review, when it is published later this year is also expected to make recommendations around conditional reimbursement.

The Cell and Gene Therapy Catapult has actively contributed to both the appraisal of the exemplar regenerative medicine product and the Accelerated Access Review; we are in support of the arising recommendations and recognise the importance of the proposed reforms at multiple levels including: reducing market access hurdles and time to UK adoption for cell and gene therapies, strengthening the UK cell and gene therapy industry and attracting overseas manufacturers to establish their operations in the UK.

For more information, read the press release issued by NICE and view the full report.