A successful practical application of Coverage with Evidence Development in Australia: Medical Services Advisory Committee interim funding and the PillCam® Capsule Endoscopy Register

Abstract

The use of interim funding requires both risk and cost sharing among the key players: industry, government, the medical profession, and the hospitals. Although the characteristics of PillCam Capsule Endoscopy proved to be suited to data collection, this may not be the case with other emerging health technologies. If interim funding coupled with data collection is to become an effective mechanism for bridging the evidence gap, work needs to be carried out by health technology assessment agencies to provide guidance on the design of registers so that they cater for the unique characteristics of individual procedures.

Coverage With Evidence Development and Managed Entry in the Funding of Personalized Medicine: Practical and Ethical Challenges for Oncology

“…15 Evidence generation in CED also differs from traditional premarketing clinical research in that RCTs are not necessarily preferred over other research methods. There are two main alternatives to RCTs that can provide useful information to inform CED and that may be particularly important in CED for PMs: observational cohort studies and prospectively designed single-arm studies.…”

“…A similar CED approach has been used since 1998 by the Australian Medical Services Advisory Committee, which funds medical devices and diagnostics. 15 The ethical and sociopolitical justifications for CED programs are that they balance the interests of clinicians and patients, who want early access to new diagnostic tests and medicines; payers, which want to address genuine health needs but do not want to pay more for medicines than they are worth; and pharmaceutical companies, which want to be paid fairly for their products. As Pearson et al 23 argue, such programs "can play an important, ethically legitimate role in balancing needs for better evidence .…”

“…Because these studies also rely on real-world data sources, such as patient registries or administrative databases, and well-matched historical or contemporary comparative cohorts, 15,26 they also must be supported by funding agencies, sponsoring companies, clinicians, administrators, and insurers. 15 These studies should also complement, rather than replace, RCTs.…”

Usefulness of a short-term register for health technology assessment where the evidence base is poor

“…For example, the Medical Services Advisory Committee (MSAC) of Australia have used information from the PillCam Capsule Endoscopy Register to validate assumptions used in their economic modeling and influence health technology appraisal decision making (11). For example, the Medical Services Advisory Committee (MSAC) of Australia have used information from the PillCam Capsule Endoscopy Register to validate assumptions used in their economic modeling and influence health technology appraisal decision making (11).…”

Safeguarding injured Victorians: development and implementation of an evidence-informed system to manage therapeutic uncertainty and decision making in a compensable environment

“…6,7 A Type 2 error occurs when the agency denies funding of a treatment that is later found to be beneficial and safe (high value). 7,8 Accordingly, the delivery of high value care to appropriately selected patients requires the consideration of funding NENETs with rigorous evaluation of risk and benefit. A transparent and consistent decision making process is also desirable to reduce the risk of conflict and litigation, factors associated with suboptimal recovery in compensable systems.…”