ASSESSING THE RETURN ON INVESTMENT (ROI) OF HEOR & RWE TO THE BIOPHARMACEUTICAL INDUSTRY: CASE EXAMPLE OF EXTERNAL CONTROL ARMS FOR CLINICAL TRIALS
Author(s)
David Thompson, PhD1, Sandra Nestler-Parr, MPhil, MSc, PhD2, Craig Roberts, MBA, PharmD3, Christopher M. Blanchette, MA, MBA, MSc, PhD4, Rob Abbott, MA5, Vera Mastey, MS6, Montserrat Vera-Llonch, MD, MSc, MPH7, Murtuza Bharmal, MS, PhD8, Ravinder Dhawan, PhD9, Riad Dirani, PhD10, Jan E. Hansen, PhD11;
1Rubidoux Research LLC, Founder & Principal Consultant, Manchester, MA, USA, 2Biocryst Pharmaceuticals, Weybridge, United Kingdom, 3Merck, North Wales, PA, USA, 4Novo Nordisk, Doylestown, PA, USA, 5ISPOR, Lawrenceville, NJ, USA, 6Regeneron Pharmaceuticals, Sleepy Hollow, NY, USA, 7Ionis Pharmaceuticals, Inc, Carlsbad, CA, USA, 8AstraZeneca, Boston, MA, USA, 9Pfizer, New York, NY, USA, 10Teva Pharmaceuticals, West Chester, PA, USA, 11Sanofi, Scottsdale, AZ, USA
1Rubidoux Research LLC, Founder & Principal Consultant, Manchester, MA, USA, 2Biocryst Pharmaceuticals, Weybridge, United Kingdom, 3Merck, North Wales, PA, USA, 4Novo Nordisk, Doylestown, PA, USA, 5ISPOR, Lawrenceville, NJ, USA, 6Regeneron Pharmaceuticals, Sleepy Hollow, NY, USA, 7Ionis Pharmaceuticals, Inc, Carlsbad, CA, USA, 8AstraZeneca, Boston, MA, USA, 9Pfizer, New York, NY, USA, 10Teva Pharmaceuticals, West Chester, PA, USA, 11Sanofi, Scottsdale, AZ, USA
OBJECTIVES: A single-arm trial (SAT) with external control arm (ECA) derived from real-world data (RWD) may be considered when the viability of a traditional two-arm randomized controlled trial (RCT) is uncertain. While the SAT/ECA design ostensibly offers time and cost savings versus the RCT, the latter remains the evidentiary gold standard and deviating from that could introduce regulatory and reimbursement risks. The objective of this study was to understand the ROI of RWE in this context.
METHODS: We constructed a decision-analytic model to characterize the risks, costs, and potential clinical and commercial outcomes of employing an SAT/ECA design versus RCT for a pivotal clinical trial. We linked each terminal node of the decision tree to a net present value (NPV) calculation that characterizes trial costs and product revenue accrual over time and then discounts their values back to the trial design decision. Data from the published literature were used to estimate model parameters for a hypothetical development compound with projected $1 billion annual revenue potential. Extensive scenario and sensitivity analyses were performed.
RESULTS: The SAT/ECA design, associated with 61.5% lower trial costs versus the RCT design, could yield an expected NPV advantage of $174,333,000 and 44-fold ROI on the $4 million cost of constructing the ECA from RWD. However, if the SAT/ECA has relatively lower probabilities of trial success (by 5.6 percentage points), regulatory approval (9.3%), or favorable reimbursement (24.3%) any of these alone would nullify the expected NPV advantage and drive ROI to zero. Results also are sensitive to estimated time and cost advantages of the SAT/ECA design, but relatively insensitive to the cost of the RWD-derived ECA.
CONCLUSIONS: Use of RWE for external controls can yield a strong ROI, but only if the RWD sources and analytic methods used are of sufficient quality to avoid jeopardizing trial success, regulatory approval, and reimbursement.
METHODS: We constructed a decision-analytic model to characterize the risks, costs, and potential clinical and commercial outcomes of employing an SAT/ECA design versus RCT for a pivotal clinical trial. We linked each terminal node of the decision tree to a net present value (NPV) calculation that characterizes trial costs and product revenue accrual over time and then discounts their values back to the trial design decision. Data from the published literature were used to estimate model parameters for a hypothetical development compound with projected $1 billion annual revenue potential. Extensive scenario and sensitivity analyses were performed.
RESULTS: The SAT/ECA design, associated with 61.5% lower trial costs versus the RCT design, could yield an expected NPV advantage of $174,333,000 and 44-fold ROI on the $4 million cost of constructing the ECA from RWD. However, if the SAT/ECA has relatively lower probabilities of trial success (by 5.6 percentage points), regulatory approval (9.3%), or favorable reimbursement (24.3%) any of these alone would nullify the expected NPV advantage and drive ROI to zero. Results also are sensitive to estimated time and cost advantages of the SAT/ECA design, but relatively insensitive to the cost of the RWD-derived ECA.
CONCLUSIONS: Use of RWE for external controls can yield a strong ROI, but only if the RWD sources and analytic methods used are of sufficient quality to avoid jeopardizing trial success, regulatory approval, and reimbursement.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
RWD86
Topic
Real World Data & Information Systems
Disease
SDC: Oncology, SDC: Rare & Orphan Diseases