Elsevier

Health Policy

Volume 125, Issue 3, March 2021, Pages 335-340
Health Policy

Impact of a risk-sharing agreement in rheumatoid arthritis in Spain

https://doi.org/10.1016/j.healthpol.2020.11.009Get rights and content

Highlights

  • The effectiveness of new drugs may differ from efficacy in clinical trials.

  • Adequately designed RSAs are useful instruments for the sustainability of healthcare systems.

  • RSAs are a financial tool to control the uncertainty of new drugs.

  • The RSA on certolizumab pegol in Spain implied cost savings for hospitals.

  • We estimated a reduction of at least 16 % in CZP treatment cost.

Abstract

Context and objective

Risk-sharing agreements(RSA) allow decision-makers to manage the uncertainty associated with effectiveness and costs of treatments. Our objective was to estimate the economic impact of RSA implementation on treatment of patients diagnosed with rheumatoid arthritis(RA) with certolizumab pegol(CZP) and assess the potential impact of alternative RSA.

Methods

Under original RSA, treatment with CZP was reimbursed when the response was optimal (DAS28 score <3.2) or satisfactory (DAS28 score ≥3.2 and reduction from baseline ≥1.2) at 12 weeks. Alternative RSA would additionally include a 50 % reimbursement for moderate responders(DAS28 score >3.2 and ≤5.1, and reduction from baseline between 0.6 and 1.2). We estimated average savings per patient for hospital's pharmacy service(HPS) at 12 weeks, taking into account the pharmacological cost of CZP. Uncertainty associated with effectiveness of CZP was assessed through 1000 Monte Carlo simulations.

Results

After 12 weeks of treatment, 57.8 % (n = 52) and 22.2 %(n = 20) of patients had optimal and satisfactory responses, respectively, and average disease activity improved by 1.77 points. Average savings for HPS amounted to 876.9€ and 706.4€ per patient under original and alternative RSA, respectively. Savings in simulated cohort reached 846.2€ and 681.8€ per patient, respectively, leading to estimated net savings for HPS of 846,209€ and 681,790€, respectively.

Conclusions

RSA implementation on patients with RA treated with CZP has generated savings and improved efficiency within HPS.

Introduction

Access to new drugs faces different challenges that must be taken into account: a combination of high prices of new patented medicines and the uncertainty related to their clinical effectiveness and use in real life imply a dilemma for decision-makers [1]. Indeed, on the one hand, the real effectiveness of a new drug is unknown, as it may differ from the efficacy shown in clinical trials, especially when it comes to specific patient sub-populations. On the other hand, the health expenditure associated with the introduction of the new drug in the market is also uncertain, because expenditure will depend on prescription patterns, the dimension of the target population, patients’ demand, and available treatment alternatives.

An increasingly common way of dealing with these uncertainties is through the adoption of alternative financing schemes for pharmaceutical innovations, such as Risk Sharing Agreements (RSAs) [2]. RSAs are among the most commonly used financial tools in this context, and their main objective is to replace traditional payment models based on a fixed price per drug container, for alternative models where payment is linked to clinical results [3]. RSAs have been implemented for years in many high-income countries, especially when it comes to new drugs such as biological agents and other cancer-related drug therapies, drugs targeting infectious diseases (e.g. hepatitis C), rare diseases, and autoimmune diseases like rheumatoid arthritis (RA) [4,5].

RA is a chronic autoimmune disease that entails a significant human and economic burden [6]. Worldwide, its prevalence rates vary between 0.3 % and 1% [7]. In Spain, the estimated prevalence of RA is 0.8 % [8]. In addition, RA has no cure, and approximately 30 % of patients do not respond to traditional disease-modifying antirheumatic drugs (DMARDs) and become candidates for biologic treatments such as tumor necrosis factor inhibitors (TNFi) [9]. In recent years, the availability of TNFi has enabled a better control of the disease, which in turn has allowed for improvements in patients’ functional capacity and quality of life. For this reason, TNFi are recommended by clinical guidelines [10].

The first RSA on the treatment of RA in Spain was developed in 2013, between the University Hospital of Bellvitge (Catalonia) and the certolizumab pegol (CZP) manufacturing laboratory (UCB Pharma). CZP is a biologic TNF alpha blocker for the treatment of adult RA patients whose response to DMARDs has been inadequate. CZP was authorized by the EMA in October 2009 [11], and has been commercialized in Spain since December 2009 [12]. Efficacy data from three pivotal trials (RAPID-1, RAPID-2, and FAST4WARD) showed that most patients treated with CZP were good responders at 12 weeks of treatment [[13], [14], [15], [16]]. Furthermore, fast improvements in physical function and in symptom reduction were able to predict satisfactory results in the long run [[17], [18], [19]]. Hence, as the decision to continue treatment with CZP depends on the results at week 12, the RSA stated that the laboratory would refund all CZP doses provided to patients who did not reach an optimal or satisfactory clinical response at that time. This agreement has already been implemented in several hospitals across 9 Autonomous Communities in Spain [20].

In 2016, the expected economic impact of this RSA was assessed in a cohort of 81 patients [21]. However, nowadays, there are real effectiveness data regarding the CZP treatment in a larger population of patients. Therefore, the objective of this study was to assess the real economic impact associated with the implementation of the current RSA in a sample of RA patients treated with CZP in Spain, and to assess the potential impact of an alternative RSA.

Section snippets

Patients

We used data from a cross-sectional study carried out in 2017 on a sample of 138 patients distributed across 19 Spanish hospitals where the original RSA had been implemented on adult patients diagnosed with moderate to severe RA. All patients were treated with CZP after an inadequate response to DMARDs, including methotrexate.

Clinical effectiveness criteria

The clinical effectiveness measure was the Disease Activity Score (DAS), which is a continuous measure of RA disease activity that combines information from swollen

Structure of the model

The structure of the study is shown in Fig. 2. Of the 138 patients who participated in the cross-sectional study, data was incomplete in 41 patients. Therefore, the data from 97 adult patients (distributed across 18 hospitals in Spain) were considered for this research. After 12 weeks of treatment, 72 patients showed an adequate response, whereas the remaining patients showed an inadequate response, either in terms of DAS28 (n = 28) or because they prematurely discontinued treatment due to

Discussion

RSAs are an increasingly used innovative financial tool whose purpose is to control the uncertainty associated with the effectiveness and/or real safety of new healthcare interventions, especially in early stages of adoption. Under an RSA, different healthcare stakeholders become jointly responsible for the success and risks associated with a new treatment, which may favor access to innovation, savings generation, rationalization of public expenditure on health, and efficiency in the healthcare

Conclusions

This study sheds light on the savings that have been generated within the hospital's pharmacy service under the current RSA for the treatment of RA patients with CZP in Spain. Beyond isolated pilot experiences, the implementation of performance-based financial schemes is a way of introducing continuous evaluation in healthcare management. Adequately designed and evaluated RSAs may be relevant instruments for reaching efficiency in the healthcare system.

Declaration of Competing Interest

The authors declare that there is not conflict of interest that could be perceived as prejudicing the impartiality of the research reported and there is not any competing financial interest in relation to the work described in this article.

Acknowledgments

The participating investigators of the Study Group Ariesgo collected data. Theywere Rosa Garcia Portales, José Manuel Fernandez Ovies, Antonio Fernández Nebro, Isabel Muñoz, Jose María Salazar, José Luis Álvarez, Juan Francisco Rangel, Manuel Romero, Florentina Horno, Juan Povedano, Javier Bautista, Juan Povedano, Gabriel Ramírez, Enrique Raya, Sara Marsall, Julio Martínez Cutillas, RaimónSanmartí, Ramón Casabona, Inés Gozalo, José Miguel Ruiz, Tomás Casasin, Nuria San Juan, Azucena Hernández,

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