Evidentiary Standards in Health Technology Assessment

Unfortunately, standards and recommendations for modeled claims in health technology assessment (HTA) or pharmacoeconomics seldom meet the standards expected in normal science. Over the past 20-25 years the focus in cost-effectiveness analysis has been on model building, but with little attention paid to the ability of the models to generate predictions for product and device impact that are either testable or testable in a timeframe that is appropriate to formulary decisions.

The result of this focus on model building is that the standards and formats recommended or proscribed by agencies such as the Academy of Managed Care Pharmacy (AMCP) in the US, the National Institute of Care and Clinical Excellence (NICE) in the UK, the Canadian Agency for Drugs and Technologies in Health (CADTH) in Canada and the Pharmaceutical Benefits Advisory Committee (PBAC) in Australia, support modeled yet untestable claims. There is no requirement for claims to be testable, particularly within a timeframe relevant to formulary decisions.

This is an untenable position which has been recognized by a number of authors. Most recently, a supplement to the Journal of Medical Economics (JME: December 2015) made the case that if claims for the impact of products and devices on costs and outcomes in health care systems are to be accepted then they should meet the standards expected in ‘normal science’.

• The only acceptable modeled claims in formulary submissions are those that are testable in a timeframe relevant to the needs of a formulary committee.

• If claims do not meet this standard they should be rejected.

• The supplement proposed a new research agenda that focused on the testable impact of products and devices on patient outcomes, resource utilization and the costs of health care delivery.

• Key elements in this proposed new research agenda are that formulary submissions claims should be evaluated and reported on in a timeframe that is meaningful to the committee.

• In practice, this would mean 2-3 years with results reported on as part of ongoing disease area and therapeutic reviews.

• To support this process the supplement recommends that formulary submissions be accompanied by a validation protocol (PROST) detailing how the claims are to be evaluated and a short list of questions a formulary committee should ask of a manufacturer’s submission.

• At the same time the supplement points to the ready availability of ‘big data’ to support claims evaluation.

It is this new research agenda that Maimon Research supports and advocates to clients in developing formulary submissions and value propositions for products and devices.

Maimon Research can prepare GVD submissions that meet these standards in modeled claims for cost-effectiveness that are supported by testable claims for comparative product impact for:

• Clinical outcomes

• Patient reported outcomes

• Resource utilization

• < >The claims are tailored to the requirements of specific formulary committees and include the PROST protocol for claims validation and reporting.