Commercial impact of adding real-world evidence to clinical trials at regulatory approval: A Markovian-like transition model

Regulatory use of real-world evidence (RWE) has been recognised as a useful supplement to clinical trial evidence and could benefit patients by reducing time to treatment. However, commercial benefits have not been documented. The aim was to determine commercial impact of regulatory RWE, using ambrisentan as an illustrative example. A Markovian-like transition model was constructed to simulate the drug development workflow across a simulation time of t = 20 years. RWE was assumed to be incorporated at pII - pIII and pII - pIII - pIV, and its multiplicative median transition rate was determined by biopharma expert opinion. Each model was subjected to ‘with’ and ‘without’ RWE rates. Commercial impact was estimated using potential decrease in time to launch. Time to first medicine adoption and potential lives saved were also estimated. Based on cumulative first prescriptions for ambrisentan among pulmonary arterial hypertension patients (N = 487), in comparison to standard drug development, RWE incorporation has the potential to expedite first medicine adoption by 10.4 weeks. The duration of market launch was estimated at 2.5–3.0 years earlier than standard, and approximately 9% of patients would benefit in survival. Potential earnings for an earlier launch would be GBP £43,597.86 per patient, with launch being brought forward from 2009 to 2007. Regulatory RWE has the potential to increase overall survival rates and potential earnings by reducing time to launch. This study provides further support for industry efforts to generate RWE in time for regulatory approval.