HealtH tecHnology assessment – implications for national policy: an industry perspective

Introduction and objectives: Due to limited resources, health systems are increasingly looking to health technology assessment (HTA) as a tool to guide health policy decisions. There are a range of models used in HTAs across the European countries including the “relative effectiveness” followed by Germany and France, and the “cost/quality adjusted life year (QALY)” used in the UK. Pharmaceutical companies need to navigate different systems and value assessment criteria to ensure patient access to new medicines. There are examples of good practice in a number of HTA systems; however, current systems often suffer from complexity, lack of transparency and limited focus to patients. The objective of this review was to provide an industry perspective on the key principles of a national HTA system that would balance the needs of payers, healthcare professionals, patients and industry. Dear readers, Following the successful proceedings of the first Health Technology Assessment Conference, organized in Athens on November 5th, 2015 Health Daily and Boussias Communications are proud to present in this special edition the first paper as a result of the work conducted by one of the main speakers Mr. Martin Price, Vice President, Market Access EMEA, Janssen on the implications of HTA for national policy from an industry perspective. In light of our forthcoming HTA Conference on February 7, 2017, this paper is most important as it sets a precedent and food for thought for the imminent discussion on building trust and identifying national barriers as a basis for creating an integrated HTA platform in our country. We would like to thank Mr. Price for keeping his promise and following through with this important work. Discussion: From an industry perspective, the key principles for a nationally relevant, rational, effective and patient-focussed HTA system should include transparency, stakeholder engagement, independence of HTA assessment bodies and payers, accountability, a locally relevant scope and an appropriate evidence framework. Dialogue between decision-makers and industry is key to discuss the value assessment, pricing and patient access arrangements. As evaluating affordability is not the remit of HTAs, patient access schemes (e.g. risk sharing, confidential discounts or price capping), are valuable in addressing potential affordability issues. A system that values new therapies on the basis of clinical benefit and subsequently enters a negotiation period with payers may lead to better outcomes for patients compared with systems driven by rigid costeffectiveness thresholds. Conclusions: There is no single “right” HTA model, but good practice elements exist across different systems in Europe. Although sharing HTA experience across countries is beneficial, decisions based on HTA need to remain a national responsibility. Countries who are considering introducing HTA need to define processes that reflect national competence and address local priorities. introduction Health systems, however diverse, share a common goal to improve the health of their population. The financing and delivery of health services is a critical aspect in being able to achieve this goal of health gain. With limited resources, health systems are increasingly looking to health technology assessment (HTA) as a means to guide health policy decisions. HTA, a multidisciplinary process that summarises the medical, social, economic and ethical issues related to the use of a health technology in a systematic, transparent, unbiased and robust manner, informs the formulation of safe and effective health policies that are patient focused and achieve best value for the jurisdiction. This article provides an industry perspective on the key principles that should be considered inherent to the establishment, conduct and implementation of national HTA. current situation Looking at established HTA processes in Europe, two main models have evolved around the value framework, namely the “relative effectiveness” and the “cost/quality adjusted life year (QALY)” approaches (Figure 1). The “cost/QALY” (CPQ) model provides estimates around the incremental costs per QALY gained and aims to assess the cost-effectiveness of new interventions against an acceptable incremental cost-effectiveness ratio (ICER) threshold (e.g. £30,000 per QALY gained for the National Institute for Health and Care Excellence [NICE] in the UK). One potential benefit of this model is its explicit robust methodological criteria. Drawbacks are said to include its reliance on often complex modelling, resulting in technical complexity, statistical uncertainty, and difficulty for stakeholders to understand results and implications. In addition, there is debate about what an appropriate ICER threshold should be and how clinically relevant the QALY metric is when the focus of HTAs are patients. “Relative effectiveness” models tend to evaluate new technologies in terms of the clinical outcomes and added benefit they provide patients, compared with the current standard of care. Typically, an evaluation process is undertaken during which the added clinical value of the new technology is characterised relative to standard of care. Once this has been finalised, the outputs of the evaluation are used as inputs into price negotiations between the company and the relevant national authority. The more intuitive concept of this model makes it easier for key stakeholders to understand the basis reviews the evidence and decides on the added benefit of the new technology, which is expressed on a six point scale, ranging from “reduced benefit” to “major benefit” compared with the current standard of care. In France, the added benefit of the new technology is rated on a five-point scale of “Improvement of Medical Benefit” (ASMR), with an ASMR rating of I showing major improvement (e.g. demonstrated effect on mortality in a severe disease) and an ASMR rating of V showing no improvement versus standard of care. The Haute Autorité de Santé (HAS) assesses the evidence, whilst the pricing and reimbursement decision is the responsibility of the Commission de Transparence (Transparency Commission). Figure 1: Value Framework: main models evolved in Europe Industry perspective Key elements of an effective HTA system Figure 1: Value Framework: main models evolved in Europe A number of models of HTA have evolved across different European countries over the last twenty years. Pharmaceutical companies need to navigate these different systems and different value assessment criteria in order to ensure patient access to new medicines. These different systems can result in diverse coverage decisions for the same medicine, based on the same evidence package. Our perspective is that there are examples of good practice in a number of systems, but that none of the existing HTA systems are ideal. For this reason, we have identified the key elements of an HTA system that would balance the needs of payers, healthcare professionals, patients and industry. Over the last decade there have been several publications by academics and international forums outlining key principles for HTA (Drummond et al, 2008; EUPATI, 2015), regarding the structure and methods of HTA, the process for conducting HTA, and the impact of HTA on decision-making. As a major stakeholder in HTA, industry needs to be confident that the key principles around allocation of healthcare resources provide an opportunity for flexible dialogue between industry and decisionmakers within the relevant agencies. To achieve this dialogue, the key elements of an effective HTA system from an industry perspective include transparency, stakeholder engagement, independence of HTA assessment bodies and payers, accountability, a locally relevant scope and an appropriate evidence framework (Figure 2). Figure 2: Key elements for an effective HTA process HTA and the associated decision making processes should be founded on principles of fairness, evidence-based decision-making and transparency. The lines of communication between all • Transparency • Stakeholder engagement • Independence of HTA assessment bodies • Independence of payers