Addressing Key Challenges in the Setting of Tumor-Agnostic Drugs: Meeting an Unmet Need?


Discussion Leader: Beth Devine, PhD, PharmD, MBA, The Comparative Health Outcomes, Policy, and Economics (CHOICE) Institute, University of Washington, Seattle, WA, USA
Discussants: Emma Mackay, MA, MSc, Inka Health, Toronto, ON, Canada; Yilin Chen, MPH, PhD, Curta, Inc., Seattle, WA, USA; Dan Ollendorf, PhD, Institute for Clinical and Economic Review; Tufts University School of Medicine and Tufts Medical Center, Boston, MA, USA


Tumor-agnostic or histology independent therapies are cancer therapies that target specific genetic or molecular alterations, regardless of tumor histology. Often evaluated using ´basket trials´, the assessment of tumor-agnostic therapies presents stakeholders with many challenges, including small sample sizes due to rare conditions, lack of a counterfactual, immature endpoints at the time of drug approval, and heterogeneity in treatment effect across histologies. This workshop will focus on how to leverage established biostatistical and economic modeling methods to assess novel therapies to inform reimbursement decisions.


Workshop attendees will obtain a working knowledge of existing methods for evaluating tumor-agnostic therapies. The workshop will review (a) current challenges faced by health technology assessment (HTA) agencies when considering tumor-agnostic therapies by context and across jurisdictions: (b) how biostatistical and economic modeling tools can be leveraged to address these challenges; (c) perspectives of the National Institute for Health and Care Excellence Technology Appraisal Committees and the Institute for Clinical and Economic Review (ICER). Dr. Devine will chair the session and introduce the topic in the context of existing frameworks and approaches taken by different countries (5min.). Ms. Mackay will show how Bayesian hierarchical models can handle small sample sizes and heterogeneity in basket trials (15min.). Dr. Chen will describe potential approaches to addressing challenges in cost-effectiveness analysis, providing a case example using pembrolizumab in 8 MSI-H/dMMR metastatic cancers in the US (15min.). Dr. Ollendorf will offer an ICER perspective and discuss the policy implications of these methods for assessment of product value, fair pricing, and reimbursement decisions in the US (15min.). Each speaker will incorporate real-time audience polling to highlight learning opportunities and focus on take-away messages (10min.). This interactive and informative workshop will be valuable to HTA policy makers, payers, and industry analysts who are considering tumor-agnostic indications in healthcare decision-making.




Methodological & Statistical Research