A medicine adoption model for assessing the expected effects of additional real-world evidence (RWE) at product launch

Abstract

Introduction:The optimal launch and adoption of novel medicines require effective navigation of the commercial and regulatory landscape, including three key gatekeeper groups: regulators, payors, and prescribers. Traditionally, despite lacking generalisability, the pharmaceutical industry provides Phase III randomized controlled trial evidence to gatekeepers. Despite additional real-world evidence (RWE) improving the generalisability of evidence, the provision of RWE remains in its infancy. The industry may use a multiple stakeholder approach to RWE generation to address each group's priorities and expectations effectively. Methods: The Jandhyala Method observed a consensus of expert opinion on the expected effects of supplementing randomised controlled trial evidence (RCTE) with RWE on the depth and time to maximal adoption. The study recruited participants from two of the three gatekeeper stakeholder groups (prescribers and payors) and their four aligned industry stakeholder groups (market access, commercial, medical affairs, and regulatory affairs) groups involved in the approval or adoption of investigational medicines. Results: The observed consensus predicted a shortened time to maximal adoption by 22% and increased maximal adoption by 31% of a new medicine with additional RWE at launch. The suggested rationales for these effects varied across stakeholder groups and between specific effects. The results found the prescriber gatekeeper to be dominant in driving the predicted shortening of adoption. No such dominant subsystem was identified for the expected increase in adoption depth, although a potential rationale was the broader population observed in the supplementary RWE. Conclusions:The proposed model provides an intuitive temporal framework within which subsystem logic can be further researched to identify and address stakeholder groups' needs and weigh those needs. Preliminary results appear to support adopting a multiple stakeholder approach to RWE for augmenting the uptake of novel medicines.

Development and Validation of the Medical Affairs Pharmaceutical Physician Value (MAPPval) Instrument

“…MAPPs play a critical role in guaranteeing that key messages are informed by sound scientific evidence and no disingenuous assertions are made [ 32 ], which explains their high level of accountability for pharmaceutical company activities. MAPPs also design and implement RWE to answer questions unanswered by registered clinical trials [ 33 , 34 ], therefore their value is not only in their ability to influence successful medicine adoption but to do so in many different ways. Thus, the findings of this study suggested that MAPPs were of key value to pharmaceutical company success and they were the only internal stakeholder of those studied able to deliver this value.…”

Concordance between the schedule for the evaluation of individual quality of life-direct weighting (SEIQoL-DW) and the EuroQoL-5D (EQ-5D) measures of quality of life outcomes in adults with X-linked hypophosphatemia

“…Accurate construct measurement in social sciences can be argued as being just as important as in natural sciences. However, given the key decisions made on the basis of findings from clinical studies particularly in the context of the adoption of new medicines [ 11 ] the consequences of inaccurate findings in this setting can be particularly harmful to the very patients the investigators are seeking to support.…”

“…Each subsystem requires specific evidence to satisfy its internal logic for that medicine to progress in its adoption. Evidence of quality of life improvements in rare disease patients receiving a particular treatment is valuable in payor discussions [ 10 ] and therefore an essential input for the Payor subsystem [ 11 ].…”

Concordance Between The Schedule for The Evaluation of Individual Quality of Life – Direct Weighting (SEIQoL-DW) and The EuroQoL-5D (EQ-5D) Measures of Quality of Life Outcomes in Adults With X-linked Hypophosphatemia.

“…Accurate construct measurement in social sciences can be argued as being just as important as in natural sciences. However, given the key decisions made on the basis of ndings from clinical studies particularly in the context of the adoption of new medicines [11] the consequences of inaccurate ndings in this setting can be particularly harmful to the very patients the investigators are seeking to support.…”

“…Each subsystem requires speci c evidence to satisfy its internal logic for that medicine to progress in its adoption. Evidence of quality of life improvements in rare disease patients receiving a particular treatment is valuable in payor discussions [10] and therefore an essential input for the Payor subsystem [11].…”