Five Important Themes in Late Phase Evidence Development

Five Important Themes in Late Phase Evidence Development

Last week, more than 100 senior Late Phase pharmaceutical professionals and key opinion leaders representing Medical and Scientific Affairs, Health Economics and Outcomes Research, Safety and Risk Management, Regulatory, Market Access and Drug Reimbursement met in Berlin, Germany, at the Third Annual Medical Affairs Leaders Forum. We joined a talented group of industry leaders on February 12th to provide a series of presentations focused on both the opportunities and challenges associated with Late Phase evidence development.

Five themes emerged from different speakers throughout the day:

  • Market access is not an “event” anymore, but rather a process over time. More than ever, a successful market launch requires a strategic and coordinated evidence development plan that serves to reduce the potential uncertainty associated with the real-world value of a drug or device. Evidence development must be viewed as a carefully orchestrated program of research activities rather than standalone studies.
  • The need for evidence of incremental benefit versus the standard of care is increasing the need for comparative study designs such as pragmatic trials. Studies that gather comparative data versus standard of care early in the market uptake phase of a new product, however, can result in selection biases and other methodological challenges. Payers and regulators should discuss these issues openly. 
  • The importance of the Patient Experience in evidence development and its growing relevance to decision makers was underscored.
  • Novel approaches to hybrid study designs, including post-authorisation safety studies (PASS), resulting in a diversity of clinical, safety, health economic, and quality of life outcomes are warranted.
  • Those at the helm of study design activities must offer leadership with respect to:

    • Scientific rigor for small- or large-scale studies
    • Operational efficiencies and streamlining within and across studies
    • Transparency regarding the consequences of trade-offs between science and logistics

The industry is evolving, and leadership in the navigation of evidence development challenges has never been as important. Our UBC presentation at the Berlin meeting on “Navigating the Convergence of Payer and Regulator Evidence Requirements” may be accessed here.