The Emerging Landscape of Reimbursement of Regenerative Medicine Products in the UK: Publications, Policies and Politics

Abstract

Aim: This paper aims to map the trends and analyze key institutional dynamics that constitute the policies for reimbursement of regenerative medicine (RM), especially in the UK. Materials & methods: Two quantitative publications studies using Google Scholar and a qualitative study based on a larger study of 43 semi-structured interviews. Results: Reimbursement has been a growing topic of publications specific to RM and independent from orphan drugs. Risk-sharing schemes receive attention among others for dealing with RM reimbursement. Trade organizations have been especially involved on RM reimbursement issues and have proposed solutions. Conclusion: The policy and institutional landscape of reimbursement studies in RM is a highly variegated and conflictual one and in its infancy.

Advanced therapy medicinal products and health technology assessment principles and practices for value-based and sustainable healthcare

“…In a recent publication, Hampson et al recommend the consideration of outcome-based agreements and their possible combination with potential methods of leased payments when health benefits are expected over a long time horizon [ 10 ]. Performance-based risk-sharing arrangements (PBRSAs) are sometimes used where there will be significant budget impact and where there is uncertainty in the available evidence base [ 39 – 41 ]. PBRSAs include outcome-based schemes, risk-sharing agreements, coverage with evidence development, access with evidence development, conditional licensing and managed entry schemes.…”

Comparative Lessons in Regenerative Medicine Readiness: Learning From the UK and Japanese Experience

“…NICE is the primary agency responsible for HTA in England and Wales, although NHSE also conducts some of its own assessments where guidance from NICE is absent. Although a higher threshold of cost-per-QALY (see Table 1) exists for rare diseases, this methodology has been seen as challenging to RM, where large datasets from randomized control trials are rare, patient populations are often small and manufacturing costs are high [14,56]. While several alternative measures have been considered (Table 1), the next steps are not yet clear.…”

“…Moreover, HTA evaluations from NICE or NHSE are only one part of the challenge. Advanced therapies must also navigate NHS commissioning procedures, which may operate through regional Clinical Commissioning Groups or NHS Executive-centralized commissioning services, under different tariffs and budgets depending on whether the product treats a common or rare condition and the care setting in which it is likely to be administered [56].…”

Chapitre 2. Les essais cliniques de médicaments de thérapie innovante : quel avenir pour la réglementation européenne ?

“…De même, les MTI reposent sur des procédés complexes. Ils soulèvent des incertitudes de preuves en raison d'un petit nombre de patients pouvant être impliqués dans les essais, comme dans le cas des maladies orphelines (la moitié des MTI ayant obtenu une AMM centralisée à ce jour sont des médicaments orphelins) 41 . Ces difficultés réelles rendent nécessaire la révision du modèle classique d'essais cliniques applicables aux médicaments traditionnels 42 et son adaptation aux enjeux particuliers soulevés par les essais cliniques de MTI 43 .…”