Early Dialogue Between the Developers of New Technologies and Pricing and Reimbursement Agencies: A Pilot Study

Abstract

It is common practice for developers of new health care technologies to engage in early dialogue with the major regulatory agencies; such discussions frequently center around the proposed clinical trial designs to support the registration of new interventions and suggestions on their improvement. Pricing and reimbursement agencies are increasingly using the results from health technology assessments to inform their decision making for new technologies. Such assessments are invariably underpinned by the phase 3 clinical trial evidence which may not provide answers to the key questions. Technology developers are beginning to realize that direct, early dialogue on the evidence requirements of the major pricing and reimbursement agencies, before phase 3 clinical trial designs for their key development compounds have been finalized, may be beneficial. This article reports on the pioneering efforts of one technology developer in seeking early dialogue with seven pricing and reimbursement agencies in five countries globally in 2007-2008 on their likely evidence requirements for a new oral treatment for patients with chronic plaque psoriasis. The pilot project demonstrated that a feasible process of early dialogue could be established, through a face-to-face meeting with prior circulation of a briefing book. Although there was some variation in the advice the similarities far outweighed the differences. More experience of early dialogue needs to be accumulated, involving a wider range of pricing and reimbursement agencies and compounds. The conclusion of this study, however, was that early dialogue can be a worthwhile process for all parties and can lead to a common understanding about evidence development for market access.

Improving the relevance and consistency of outcomes in comparative effectiveness research

“…Medicare declined to provide reimbursement for cervical artificial discs in part because they viewed the outcomes reported in the trials as uninformative for key aspects of patient functional abilities [6]. The primary outcome reported for most trials of drugs for psoriasis, which is also the one that is required for regulatory approval in the USA, is based on a clinician's judgment of the extent of disease, while payers, clinicians and patients view the distribution of the plaques and impact on functioning as most meaningful for their quality of life [7]. In their 2012 methodology committee report, PCORI provides a standard for patient outcomes that instructs researchers to, 'measure outcomes that people representing the population of interest notice and care about', to be identified with input from patients and decision makers through meetings, surveys or published studies [8].…”

Investigating the health-economic profiles of biomarker-driven immunosuppresion (BIO-DrIM) following solid organ transplantation

“…Another advantage of early integration of health-economics is the ability to provide healtheconomic data for authorities at early stages and making the authorities familiar with the intervention under consideration. Engaging in early dialogue with payers and regulators proved to be useful from a developer`s perspective [17,18] .…”

“…The generic EQ5D-5L questionnaire is a widely accepted QoL instrument, and has been recommended by various healthcare payers [17] . It will be administered at baseline (randomization), at Months 1, 3, 6, 12, 18, and 24.…”

“…The mean and median values of accumulated QALYs will be determined for each IS strategy and compared against each other. The generic SF-36v2 questionnaire is also a widely accepted QoL instrument, and has likewise been recommended by various healthcare payers [17] . It will be administered at the same time-points as the EQ5D-5L and the other QoL questionnaires.…”

Healthy Decisions: Towards Uncertainty Tolerance in Healthcare Policy

“…Such 'mapping the decision situation' informs the assessment, and might encourage stakeholders to acknowledge the necessity of information about uncertainty [17]. Although initiatives of early dialogue already exist in healthcare, they do not explicitly address the issue of uncertainty [18]. In the recent Ab PET example, the CMS decided to cover the imaging technique only in clinical studies [coverage with evidence development (CED)] [11].…”