Shaping the changing landscape of clinical evidence: a multi-stakeholder perspective on the feasibility of a pay-for-proof pricing model for improved access to innovative drugs in the Netherlands

Abstract

After a novel drug has obtained market authorization in the EU, decisions must be made on a national level regarding the reimbursement of the treatment. This process is often informed by Health Technology Assessment (HTA) agencies, who are responsible for assessing the effects of the treatment in the context of a specific healthcare system. In these assessments, HTA agencies aim to shed light on the relative effectiveness of the drug in comparison to the therapy that is generally applied to quantify its added therapeutic value. Due to tight healthcare budgets and increasing expenditures, making well-informed reimbursement decisions is becoming increasingly important.

The randomized controlled trial (RCT) is a study design that is often considered the gold standard, as directly comparing the effects of a newly proposed therapy against an existing treatment in one single study provides the highest level of evidence. However, several trends are ongoing that result in a growing number of drugs being developed through study types that deviate from the traditional RCT design. The lack of strong comparative evidence in these study designs hampers an adequate assessment on the relative effectiveness of treatments. Ultimately, this results in significantly delayed patient access or even no access at all.

Payment models have the potential to manage uncertainties that are associated with a drug through flexible agreements regarding its reimbursement. The objective of this study was to assess the feasibility of an innovative pay-for-proof (PFP) payment model and contribute to its development, ultimately advising Roche Nederland B.V. whether the Dutch healthcare system is ready for the implementation of payment models that aim at improving access to drugs associated with new types of evidence. In the PFP model, the price of a drug is based on data obtained through clinical trials as well as the growing data from real-world practice, in which higher levels of evidence are rewarded by declining discounts, thereby reflecting the real-world value of innovations.

Interviews were conducted with internal and external stakeholders of Roche. Results indicated that stakeholders believe current reimbursement procedures are not sustainable, whereas the PFP model was perceived as a fair and robust alternative that enhances flexible reimbursement and enables reflection of the real-world value of innovations. Main challenges include complex methodologies associated with real-world data and the need for a solid data-infrastructure. Further, the differential pricing used in the PFP scheme may possibly result in resistance due to perceived unfairness.

For successful implementation of the PFP scheme in the Netherlands, several recommendations were made. First, (1) ensure the scheme is easy executable for medical specialists. Further, (2) make use of an existing data-infrastructure instead of developing a new platform. Next, (3) propagate the highest price as reference price and clearly link discounts to specific conditions to justify differential pricing. More, (4) before engaging in the scheme, define which uncertainties remain and always ensure a link between these uncertainties and the conditions applied in the scheme. Last, (5) ensure active involvement of relevant stakeholders during implementation of the PFP model. Hence, the PFP model contributes to Roche’s Pharma Vision 2030 to achieve 3-5 times more patient benefits for 50% less costs to society.