ABSTRACT: Regulatory approvals of, and subsequent access to, innovative cardiovascular medications have declined. How much of this decline relates to the final step of gaining reimbursement for new treatments is unknown. Payers and health technology assessment (HTA) bodies look beyond efficacy and safety to assess whether a new drug improves patient outcomes, quality of life, or satisfaction at a cost that is affordable compared to existing treatments. HTA bodies work within a limited healthcare budget, and this is one of the reasons why only half of newly approved drugs are accepted for reimbursement, or receive restricted or “optimised” recommendations from HTA bodies.
All stakeholders have the common goal of facilitating access to safe, effective, and affordable treatments to appropriate patients. An important strategy to expedite this is providing optimal data. This is demonstrably facilitated by early (and ongoing) discussions between all stakeholders. Many countries have formal programmes to provide collaborative regulatory and HTA advice to developers. Other strategies include aligning regulatory and HTA processes, increasing use of real-world evidence, formally defining the decision-making process, and educating stakeholders on the criteria for positive decision making. Industry should focus on developing treatments for unmet medical needs, seek early engagement with HTA and regulatory bodies, improve methodologies for optimal price setting, develop internal systems to collaborate with national and international stakeholders, and conduct post-approval studies. Patient involvement in all stages of development, including HTA, is critical to capture the lived experience and priorities of those whose lives will be impacted by new treatment approvals.
INTRODUCTION: Regulatory approvals of, and subsequent access to, innovative cardiovascular (CV) medications have declined, with CV drug approvals by the US Food and Drug Administration (FDA) dropping from 16% of the total new approvals (7/45) in 2015 to 2% (1/53) in 2020 . Similarly, CV drugs accounted for just 3 (3%) of 97 new medicine, and only one of 39 new active substances, approved by the European Medicines Agency (EMA) in 2020 .
Regulatory approval of a drug does not ensure patient access – there remains the step of pricing the drug, and getting reimbursement agencies, third-party intermediaries, or patients to agree to pay for it. Evidence-based medicine remains at the heart of all treatment, guideline, and regulatory decisions. In addition, payers ask how well a treatment works compared to existing treatments, and whether it reflects value for money . Does it improve patient outcomes, safety, or satisfaction at a reasonable and affordable cost [3,4]? All stakeholders must cooperate to provide the evidence that payers need to determine the value of new treatments.
This paper builds on discussions among clinical trialists, industry representatives, regulators, and patients, which took place at the 17th Global Cardiovascular Clinical Trialists (CVCT) Forum in December 2020 (www.globalcvctforum.com). The goal was to review the decisions of regulatory, health technology assessment, and pricing and reimbursement organisations, and to suggest ways to improve patient access to evidence- and value-based therapies. Our paper reports on the views of senior representatives of these and other stakeholders from around the world attending the Forum. Throughout, we attempt to capture what emerged from extended discussions at the Forum. This paper is not a consensus statement from those present at the Forum or the organisations they are associated with. It is a contribution to the debate, aiming to stimulate discussion within and between the various stakeholder groups, nationally and internationally.
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