Adaptive Pathways: Possible Next Steps for Payers in Preparation for Their Potential Implementation

Abstract

Medicines receiving a conditional marketing authorization through Medicines Adaptive Pathways to Patients (MAPPs) will be a challenge for payers. The “introduction” of MAPPs is already seen by the European Medicines Agency (EMA) as a fait accompli, with payers not consulted or involved. However, once medicines are approved through MAPPs, they will be evaluated for funding by payers through different activities. These include Health Technology Assessment (HTA) with often immature clinical data and high uncertainty, financial considerations, and negotiations through different types of agreements, which can require monitoring post launch. Payers have experience with new medicines approved through conditional approval, and the fact that MAPPs present additional challenges is a concern from their perspective. There may be some activities where payers can collaborate. The final decisions on whether to reimburse a new medicine via MAPPs will have more variation than for medicines licensed via conventional processes. This is due not only to increasing uncertainty associated with medicines authorized through MAPPs but also differences in legal frameworks between member states. Moreover, if the financial and side-effect burden from the period of conditional approval until granting full marketing authorization is shifted to the post-authorization phase, payers may have to bear such burdens. Collection of robust data during routine clinical use is challenging along with high prices for new medicines during data collection. This paper presents the concept of MAPPs and possible challenges. Concerns and potential ways forward are discussed and a number of recommendations are presented from the perspective of payers.

Role of Health Technology Assessment in Pharmaceutical Market Access in Developed Countries

“…Alongside this, there are also increasing concerns among payers across Europe regarding issues relating to the potential introduction of adaptive pathways for new medicines to accelerate access to new innovative medicines [98,99]. Key concerns include i) issues of payment, i.e.…”

“…who will pay for the new medicine during its testing phase among patients and at what price, ii) where does the product liability lie prior to full marketing authorization, pricing and reimbursement, iii) how is innovation and unmet need defined, iv) how long are new medicines in the adaptive pathways process prior to full evaluation, v) whether such schemes are needed in reality with fast-track schemes for new medicines already in existence, vi) whether health authorities currently have the necessary ability to monitor the effectiveness and safety of new medicines in routine clinical care, vii) wheth- er payers have the necessary powers to disinvest in new medicines if found not to be costeffective in reality and manufacturers are reluctant to lower prices. Additionaly, if health authorities do not have the necessary IT systems, who would pay for their subsequent development [99]? However, the use of patient registries post launch have helped address issues of appropriateness and concerns with new medicines such as potentially increasing rates of infection and cancer with the use of biological medicines to treat immunological diseases such as rheumatoid arthritis and psoriasis.…”

“…The model begins with pre-launch activities including horizon scanning and forecasting, the potential development of quality indicators for new medicines, as well as including new medicines that could go through the proposed adaptive pathways program especially in Europe [98,[109][110][111]. This will increasingly include in Europe potential new medicines going through the adaptive pathways scheme [98,99].…”

“…Whilst proposed adaptive pathways are welcomed by European payers and their advisers to accelerate early access of new medicines, especially those for debilitating diseases and where currently limited or no therapies are available for treatment, there are still considerable concerns. These concerns have been summarized in a number of published papers [98,99,123,125,126], and include the fact that there will still be inequity in the availability of new medicines across Europe depending on potential prices. Payer HTA considerations for assessing potential prices for new medicines going through the adaptive pathways route will also need to evolve to consider how to effectively deal with increased uncertainty, and build this into their negotiations with pharmaceutical companies [99,127].…”

Key enablers and barriers to implementing adaptive pathways in the European setting

“…Payers were not involved in the early discussions around the six drugs that underwent EMA‐HTA parallel scientific advice as part of the adaptive pathways pilots, and they have already emphasised the potential downstream barriers that adaptive pathways might face across Europe from their perspective . Payers can be involved in early dialogue though a number of ongoing initiatives.…”