Timely, consistent, transparent assessment of market access evidence: implementing tools based on the HTA Core Model® in a pharmaceutical company

Abstract

ObjectivesEvidence requirements and assessment methods access differ between health technology assessment (HTA) agencies. The HTA Core Model® provides a standardized approach to HTA, targeting evidence sharing and collaboration between participating HTA bodies. It is fit for purpose from an industry perspective and was used by pharmaceutical company Roche to develop a framework for internal assessment of evidence required for market access and coverage/reimbursement (“access evidence”).MethodsTools were developed to systematically scope, assess, plan, and summarize access evidence generation. The tools were based mainly on the first four HTA Core Model® domains and rolled-out in selected development teams in 2017. Five months after full implementation, the impact of tools was assessed in an internal survey.ResultsSystematic access evidence generation started with the Access Evidence Questionnaire, to scope evidence requirements and identify evidence gaps. Findings were summarized in the Access Evidence Metric, which assessed the alignment of available/planned evidence against HTA bodies’ requirements and developed scope mitigation strategies. The Access Evidence Plan was then used to plan and document (additional) evidence generation. Once generated, evidence was summarized in the Access Evidence Dossier. A survey of twenty-seven Roche employees involved in evidence generation showed that the tools made discussions around access strategies and evidence more efficient and transparent.ConclusionsThe HTA Core Model® provided a useful framework around which to optimize internal evidence generation and assessment. The benefits of using a standardized HTA approach in industry mirror those expected from implementing the HTA Core Model® in HTA agencies.

A Review of the Evidence on Attitudes, Perceived Impacts and Motivational Factors for European Member State Collaboration for Pricing and Reimbursement of Medicines: Time for the EEA Member States to Apply Their Experience and Expertise in Evidence-Based Decision Making to Their Current Pharmaceutical Policy Challenges

“… Makady et al (2017) made a case for real world collaboration for real world data ( Makady et al, 2017 ). One paper described the benefit of the use of the HTA Core Model by a pharmaceutical company ( Ducournau et al, 2019 ).…”

“…Four papers described initiatives for national HTA in different countries, mainly at the time when the specific country was undertaking initiatives for improvement/development of their national system e.g., Belgium: Cleemput and Van Wilder ( 2009 2017) made a case for real world collaboration for real world data (Makady et al, 2017). One paper described the benefit of the use of the HTA Core Model by a pharmaceutical company (Ducournau et al, 2019). Some papers included in this review were used to update the Logic Model, for example: horizon scanning systems in Douw and Vondeling (2006) and Wild and Langer (2008); reference pricing in Leopold et al (2012); horizon scanning in Packer et al (2015), and experience of HTA agencies in Loblova, (2016).…”

Challenges and Opportunities for Companies to Build HTA/Payer Perspectives Into Drug Development Through the Use of a Dynamic Target Product Profile

“…The EUnetHTA HTA Core Model has also been utilized by companies; it has been viewed as a useful framework to standardize the domain of HTA questions and understand the common terminology ( Gyldmark et al, 2018 ). In addition, one company has developed internal access evidence generation tools based on the HTA Core Model, which has a direct impact on drafting the TPP ( Ducournau et al, 2019 ).…”

DAC Target Policy Profile (TPoP)

“…19 Such statements quantitatively establish that (and set forth 'how') a proposed change will be significantly superior to the status quo or at least non-inferior to it. That is, in a fashion much like the TPP and Minimum Clinically Important Difference (MCID) in clinical trials, [20][21][22] the TPoP ought to be accompanied by a statement of a Minimum Policy-Important Difference (MPID) in terms of policy impact. Unlike the Target Product Profile (TPP) and conventional clinical trial design and clinical MCID, the TPoP encourages the investigator and other stakeholders to jointly set forth one or more MPID value-points and their rationale in the relevant dimensions, such as economic value added (EVA), DALYs averted, or employment rate improvement.…”