Risk-sharing schemes to finance expensive pharmaceuticals: Interdisciplinary analyses

Excerpt

Limited resources for healthcare need to be allocated effectively and efficiently and in accordance with the respective value of medical interventions. Recent developments in the pharmaceutical market and a rise in the cost of drugs pose a challenge for healthcare systems. This is particularly the case for so-called “personalized medicine” and drugs for small numbers of patients (e.g. orphan drugs). Research and development costs are high, while there are risks in terms of financial return, given that these drugs only address a very small patient population. Challenges to assess the value of these drugs are considerable, especially considering the lack of robust data due to small clinical studies. Accordingly, there is a high uncertainty regarding the real-world effectiveness, leading to significant discrepancies between the pharmaceutical company's list price and the cost payer's willingness to pay.

One approach to control pharmaceutical expenditures in these cases is value-based pricing. On a microperspective level, this is employed as risk-sharing agreements between payers and pharmaceutical companies, whereby the uncertainties, i.e. risks, regarding the clinical and economic performance are shared and the remuneration for the drug is dependent on its real-world value. Depending on the inclusion of an outcome element into the scheme, we can distinguish financial-based risk-sharing schemes and performance- or outcome-based risk-sharing agreements.

This chapter will analyse the strengths and weaknesses of performance- or outcome-based risk-sharing agreements (PBRSA). In addition to a theoretical analysis, we will substantiate it by reference to a case study of a PBRSA involving the evaluation of drugs for a multiple sclerosis risk-sharing scheme in the United Kingdom.