Guidance for an Effective Approach to Integrated Evidence Planning in a Dynamic World.

The value of a medicine is defined by its impact on patients, caregivers, health system, and society. A pharmaceutical company will generate evidence to demonstrate this value in various studies, including randomized clinical trials, non-interventional and observational studies, real-world data analyses, modeling, and simulation. The quality and strength of the evidence supporting a medicine's effectiveness, safety and product quality will drive decisions by healthcare system stakeholders for marketing authorization (regulatory authorities). Additional evidence of comparative clinical, humanistic, economic, and societal value of the medicine will be critical for reimbursement coverage by HTA (health technology assessment) bodies and payers, guideline inclusion by clinical societies, and ultimately the treatment decision between a patient and their healthcare provider (HCP). The purpose of this article is to provide practical guidance for an effective approach to evidence planning for pharmaceutical companies. In the first section, we give a brief overview of the requirements for evidence generation from the perspectives of healthcare system decision makers, key functions involved in evidence generation within a pharmaceutical company, and different archetypes of products. We then discuss how a company can implement effective integrated evidence planning across the lifecycle of a product. We also review how requirements are likely to evolve given recent changes in major healthcare system regulations, such as Centers for Medicare & Medicaid Services (CMS) drug price negotiations in the US and EU HTA Regulation (HTAR) in Europe, and finally provide some practical recommendations of how to start implementing a new integrated evidence approach.