| dc.description.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. | |
