Purpose of Payers’ Advisory Committee
Background
EXCITE International is a not-for-profit organization on a mission to accelerate the adoption of breakthrough technologies and to improve the well-being of patients globally.
To that end, EXCITE International has created a pre-market collaboration consisting of innovators/companies, payers/health systems, expert end-users, regulators, and patients (collectively referred to as “stakeholders”). Overseen by an Executive Board with global representation and advised by a Payers’ Advisory Committee, a Scientific Collaboration and an Advisory Council, EXCITE International selects breakthrough technologies to be evaluated through harmonized studies that anticipate the requirements of its global stakeholders.
A priority for EXCITE International is to bring payer and health system perspectives into the early stages of developing and evaluating new technologies. The present pathway to market is disjointed in that innovators focus on regulatory requirements, only to be subsequently rejected by payers because the evidence generated for regulatory purposes fails to address the concerns of payers and health systems. There are two reasons that innovators focus on regulatory requirements. First, regulatory approval is the inaugural and most immediate step in the pathway to adoption. Second, regulatory authorities are increasingly regarding innovators as customers through actions such as making expectations explicit and meeting with companies to review protocols for planned trials. However, the results from these trials will not necessarily meet the expectations of payers and health systems, leading to downstream rejection despite regulatory approval.
There are ongoing efforts to resolve the disconnected pathway to market. Examples include the Parallel Review by FDA and CMS; the FDA-CDRH Payer Communication Task Force; the NICE UK Scientific Advice program; and, the MaRS EXCITE program. EXCITE International differs from these efforts in that it is taking a global approach that incorporates various components: early selection of potential breakthrough technologies; developing protocols that are both rigorous and feasible; and, conducting clinical evaluations in multiple jurisdictions using common protocols and processes in each region to achieve global reach. EXCITE’s approach aims to drive innovation globally and to stabilize the current innovation drain from countries with more comprehensive approaches to adoption to countries that offer a path of least resistance.
Overarching Considerations
The Payers’ Advisory Committee (PAC) provides advice to EXCITE International regarding the selection and assessment of new candidate medical technologies that are being considered for pre-market evaluation in accordance with the EXCITE International process.
1. The Payers’ Advisory Committee is advisory to the EXCITE International Management Board. In reporting the work of the PAC, contributions by discussants will be anonymized.
2. The Payers Advisory Committee and its members will be indemnified by EXCITE International as outlined in the Terms of Reference. Members of the PAC will be offered an honorarium for their participation according to the policy set by their home organizations.
3. The confidentiality of companies submitting technologies and proprietary information for consideration will be protected.
4. Certain government entities participating in the PAC may have legislative mandates regarding transparency. In such cases, steps will be taken to preserve both government obligations around transparency and confidentiality for participating companies. These steps may include separate convening and reporting mechanisms and providing non-voting observer status to representatives.
5. EXCITE International’s pre-selection process will not prejudice any subsequent review by the payers and health systems contributing to the Payer’s Advisory Committee’s final recommendation.
6. Neither the selection of a candidate technology nor the endorsement of a study protocol will obligate the payer organizations to adopt the technology under consideration.
7. Rejection by EXCITE International does not exclude future post-market consideration by payer organizations. It is understood that medical technology companies will use other avenues to adoption available through respective EXCITE International member organizations.
EXCITE International’s pre-selection process will not prejudice any subsequent review by the payers and health systems contributing to the Payer’s Advisory Committee’s final recommendation.
a. Neither the selection of a candidate technology nor the endorsement of a study protocol will obligate the payer organizations to adopt the technology under consideration.
b. Rejection by EXCITE International does not exclude future post-market consideration by payer organizations. It is understood that medical technology companies will use other avenues to adoption available through respective EXCITE International member organizations.
6. Companies are advised in their agreements with EXCITE International that the intent of the EXCITE International process is to mitigate the risk of regulatory, payer, and health system rejections through the early involvement of the relevant stakeholders, without any guarantee of adoption. Adoption entails policy decisions made by each independent payer through established processes as legislated or through their organization’s Board-approved processes.
Advisory Functions
The PAC is one of several advisory groups to the EXCITE International Board, including the Scientific Collaboration and expert end-users. The PAC has three primary functions:
i. It provides advice to the EXCITE International Board on selecting technologies for evaluation by EXCITE International, based on pre-defined criteria and processes;
ii. It provides input into protocol development to ensure that the quality, design, and outcomes are consistent with the needs and expectations of payers;
iii. It offers advice on implementation, including identifying barriers to and facilitators of adoption; and
iv. It offers early advice to industry on the potential relevance of a technology to payers. This advice may be sought as early as the proof-of-concept stage of product development and occurs through PAC’s contribution to the EXCITE International Early Technology Review offering
Guidance for Developing Advice
In fulfilling its advisory functions, the PAC is guided by two overarching principles. First, that the mission of EXCITE International is to accelerate adoption of breakthrough technologies in order to improve the well-being of patients. Second, that the role of the PAC is to provide a payer and health system perspective. That perspective often differs from the regulatory view with respect to prioritizing the outcomes of importance, the representativeness of populations, the relevance of pragmatic and real-world approaches, and the impact on population health and system efficiencies.
Succinctly, regulators oversee the entry of technologies into the marketplace. Payers and health systems must choose from the marketplace in order to effectively and efficiently fulfill their commitment to the health of their beneficiary populations.
Outlined below is guidance for each of the three primary advisory functions of the PAC: technology selection; protocol development; and implementation. The guidance supports a qualitative review and is intended to guide to deliberations of the Committee to consensus and consistency of approach.
1. Selecting Technologies
When selecting a candidate technology, the Payers’ Advisory Committee will first consider whether it has breakthrough potential and, if so, whether it is likely to be feasible to implement.
1.1 The technology has potential to be a breakthrough innovation that will have a major impact on patient outcomes and/or health system efficiencies.
a. Dimensions considered in judging the impact on patient outcomes include burden of disease (prevalence and severity), unmet need, and potential to improve access.
b. The technology addresses an important challenge in the delivery of care to a population or an important challenge to the health system as a whole. Examples include reducing hospitalization, reducing invasiveness, or improving control of a chronic disease.
c. With respect to orphan diseases, the severity and need may be sufficient, irrespective of disease prevalence and/or impact on the health system.
1.2 A potential breakthrough technology should be feasible to implement as judged by the dimensions below.
a. Are there factors that will facilitate implementation?
b. Are there barriers that make adoption unlikely?
• Benefit design, coverage, and payment considerations that may affect the uptake of the technology.
• Infrastructure and resource requirements for delivery of the technology.
c. What is the potential of the technology as an adjunct, replacement, or prompting obsolescence in the context of current and evolving clinical practice?
d. Are there other barriers to or facilitators of adoption?
2. Input into Evidence Development
Regulatory-approved medical technologies often fail to meet payer expectations due to the evidence necessary to make a coverage decision. The reasons largely fall into two categories: 1) the relevance of the evidence; and, 2) the methodological quality and credibility of the evidence.
2.1 Relevance domains
a. Is the study population clearly defined and representative of the population for whom the technology is intended?
Some important elements of defining the population include diagnosis, disease severity, prior treatments, comorbidities, and demographic characteristics such as age and sex. The study population should be specifically stated in the protocol and generalizable to the patient populations for whom the technology will be used in clinical practice. However, there may be some differences across jurisdictions in the populations of interest due to differences in care pathways and patterns of care.
b. Does the technology as designed and used in the study represent the intervention that will be used in clinical practice?
Some important elements of defining the intervention technology include intensity, delivery, operators, setting, as well as pre-and post-procedural care. Refinements to a technology though clinical trial experience may be addressed by an adaptive design approach.
c. Is the comparator intervention appropriate and representative of existing alternative(s)? Is the comparator delivered effectively, with similar intensity, and by capable operators in the appropriate setting?
Choice of comparator also depends on whether the intervention technology is intended as an adjunct or a replacement to established care. In the case of a novel innovation for an unmet need, the comparator may be best supportive care. Natural history is a relevant comparator in the absence of supportive care, but there are few conditions for which there is no supportive care. Caution is merited with "usual care comparators" as usual care may not be optimal. The "usual care" comparator must be fully described. There may be differences across jurisdictions in the preferred comparator due to differences in patterns of care.
d. Are the health outcomes the important health outcomes?
Health outcomes are outcomes patients experience and care about. Broadly, these include length of life, quality of life, and ability to function. In defining important health outcomes, consideration should be given to patient preferences and trade-offs between benefits and harms.
Outcome measures should be validated and vetted measures with a rationale for why the chosen measures are preferred. Minimum important clinical difference should be pre-specified and supported by empirical evidence or expert consensus.
If a surrogate measure is used, its validity and utility as a surrogate must have robust support from an empirical body of evidence.
Harmful outcomes are as important as beneficial outcome. Rigorous methods of eliciting and reporting harms should be used.
Robust measures of health outcomes are a pre-requisite for analysis of economic value. However, analysis of economic value also requires additional measures such as resource utilization and cost.
e. For medical tests, when direct evidence of improved health outcome(s) is not feasible, is there an empirically-based chain of evidence?
For medical tests, it may not be feasible to conduct an intervention study to assess whether use of the test for direct management improves health outcomes.
Indirect evidence of improved health outcomes requires robust evidence of clinical validity, with complete reporting of performance (true positive, false positive, true negative, false negative).
An empirically-based chain of evidence for the condition and treatment outcomes is necessary to derive benefits and harms from probabilities of true and false results.
f. Timing – is the duration of follow-up sufficient to observe the important outcomes?
The duration of follow up includes both the trial itself and provisions for longer-term post-trial observation.
2.2 Quality and credibility domains
The Scientific Collaboration has overall responsibility for protocol methodology and thus provides leadership on quality and credibility domains. In reviewing protocols, the Payers’ Advisory Committee emphasizes the quality and credibility domains below.
a. Control for selection bias, using randomization or an adequate alternative.
b. Control for perceptual bias, preferably using double-blind design. Or, where double-blinding is not feasible, using an independent assessment of outcomes.
c. The statistical design and analysis should include adequate power, appropriate statistical tests, and a plan for handling loss of participants or missing data.
d. Analysis and reporting must permit comparisons to appropriate comparator(s), noting that these might differ across jurisdictions.
3. Advice Regarding Implementation Issues
This topic is intended to alert clinical trials methodologists to implementation issues that should be incorporated into protocol development and tracked as part of the study. Some examples of implementation issues appear below.
1. Analysis of existing databases to gain a better appreciation of patient outcomes related to comparative technologies.
2. Prospective analyses of resource utilization associated with the technology and its alternatives.
3. Training requirements for health professional end-users associated with the adoption of the technology.
4. Early Advice
The Payers’ Advisory Committee will participate in the evolving EXCITE International Early Technology Review by providing further advice to EXCITE International on upstream factors in the lifecycle of the technology. This advice may be sought as early as pre-proof of concept stage of technology development.
