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Novel Access Solutions for Novel Medicines

Payers face a number of challenges when making reimbursement decisions for novel medicines. An aging population, scientific advances, and personalized care are increasing funding pressures, with payers in a conundrum to make decisions on long-term value with limited real-world effectiveness data.

One such area is complex perianal fistulas, a debilitating complication of Crohn’s disease (CD) that has a substantial impact on quality of life.1 Current treatment options are limited, and with high relapse rates and poor long-term outcomes, there is a great unmet need to effectively treat the condition.2

A systematic literature review presented at the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) highlighted that there is no clearly defined standard of care for the treatment of complex perianal fistulas.3 With this, treatment is determined on a patient-by-patient basis, which makes it challenging to measure and understand the true clinical outcomes and costs associated with the management of complex perianal fistulas.4

With an absence of comparative data and inadequate quantification of medical expense, payers may be unable to accurately estimate the costs for new therapeutic options, which may lead to uncertainty and evaluation challenges that potentially impact patient care.4

A potential solution: performance-based risk-sharing agreements
A potential solution to overcome these types of barriers is through the use of performance-based risk-sharing agreements (PBRSAs). For a disabling complication like complex perianal fistulas, a PBRSA can be hugely valuable, giving payers and clinicians the opportunity to provide patients with a new therapeutic option while collecting additional evidence needed for a greater understanding of the disease.4

TTo support a PBRSA, patient registries can be designed to collect disease and healthcare resource utilization data, which is relevant and adaptable for country-specific healthcare systems. This makes these agreements more attractive for payers across Europe.The overall success of a PBRSA will depend on a company’s willingness to assume risk and provide resources upfront, as well as the payers’ willingness to invest in a treatment to deliver the expected outcome.

Collaborate to support patient access
Collaboration between key stakeholders is needed to jointly target improvement in outcomes and fill any data gaps in the management of chronic diseases, such as CD and its complications. The pharmaceutical industry can play a key role in helping absorb the potential risk by introducing flexible pricing and reimbursement strategies and delivering outcome-prediction methodologies that allow the right patient to be treated at the right time at a sustainable cost. In such a way, payers and clinicians may have the opportunity to be equally responsible for improving patient care and outcomes.


1 Mahadev S, Young JM, Selby W, et al., Quality of life in perianal Crohn’s disease: what do patients consider important? Dis Colon Rectum. 2011; 54(5): 579-585.

2 Georgiadou E, Brandes A, Fanter L, et al., P804 Retrospective observational claims data analysis of German statutory health insurance (SHI) patients with Crohn’s disease reveals low prevalence of perianal fistulas (3.4%), but high rates of biologic treatment and inpatient surgical intervention. J Crohns Colitis. 12, S518–S519. 2018.

3 Schmetz A, et al., The role of performance-based risk-sharing agreements in minimising payer uncertainty when standard of care is not clearly defined; the example of Crohn’s disease related complex perianal fistulas, ISPOR Europe 2018, PGI40.

4 Schmetz A, et al., Darvadstrocel in the management of complex perianal fistulas: the role of patient registry data collection to support performance-based risk sharing agreements, ISPOR Europe 2018, PGI39.

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