Comparing Drug and Non-drug Technologies in Comparative Effectiveness Research

Abstract

Randomised Controlled trial evidence of efficacy and safety has long been a requirement for pharmaceuticals to gain market access. In contrast, the evidence requirements for the approval of nondrug technologies, in particular medical devices, have traditionally been much lower. However, with increasing health-care costs and pressure of limited budgets, policy-makers are increasingly turning to health technology assessment (that includes consideration of comparative effectiveness and economic value) to inform their decisions on technology coverage and reimbursement. This chapter describes the differences between medical devices and drugs that impact on the assessment of their comparative clinical and cost-effectiveness.

Most international health technology assessment agencies have a common mandate to evaluate both medical devices and drugs. At this time there appears to be little or no explicit consideration by the health technology assessment community of the potential differences between medical devices and drugs either in their assessment or appraisal processes. Comparing medical devices to drugs highlights the challenges in the common assessment – both comparative effectiveness and economic evaluation – of drug versus nondrug technologies.

Appendix

Checklist of items for reporting trials of nonpharmacologic treatments^a

Section	Item	Standard CONSORT description	Extension for nonpharmacologic trials	Reported on page no.
Title and abstract b	1	How participants were allocated to interventions (e.g., “random allocation,” “randomized,” or “randomly assigned”)	In the abstract, description of the experimental treatment, comparator, care providers, centers, and blinding status
Introduction
Background	2	Scientific background and explanation of rationale
Methods
Participantsb	3	Eligibility criteria for participants and the settings and locations where the data were collected	When applicable, eligibility criteria for centers and those performing the interventions
Interventionsb	4	Precise details of the interventions intended for each group and how and when they were actually administered	Precise details of both the experimental treatment and comparator
	4A		Description of the different components of the interventions and, when applicable, descriptions of the procedure for tailoring the interventions to individual participants
	4B		Details of how the interventions were standardized
	4C		Details of how adherence of care providers with the protocol was assessed or enhanced
Objectives	5	Specific objectives and hypotheses
Outcomes	6	Clearly defined primary and secondary outcome measures and, when applicable, any methods used to enhance the quality of measurements (e.g., multiple observations, training of assessors)
Sample sizeb	7	How sample size was determined and, when applicable, explanation of any interim analyses and stopping rules	When applicable, details of whether and how the clustering by care providers or centers was addressed
Randomization-sequence generationb	8	Method used to generate the random allocation sequence, including details of any restriction (e.g., blocking, stratification)	When applicable, how care providers were allocated to each trial group
Allocation concealment	9	Method used to implement the random allocation sequence (e.g., numbered containers or central telephone), clarifying whether the sequence was concealed until interventions were assigned
Implementation	10	Who generated the allocation sequence, who enrolled participants, and who assigned participants to their groups
Blinding (masking)b	11A	Whether or not participants, those administering the interventions, and those assessing the outcomes were blinded to group assignment	Whether or not those administering co-interventions were blinded to group assignment
	11B		If blinded, method of blinding and description of the similarity of interventionsb
Statistical methodsb	12	Statistical methods used to compare groups for primary outcome(s); methods for additional analyses, such as subgroup analyses and adjusted analyses	When applicable, details of whether and how the clustering by care providers or centers was addressed
Results
Participant flowb	13	Flow of participants through each stage (a diagram is strongly recommended) – specifically, for each group, report the number of participants randomly assigned, receiving intended treatment, completing the study protocol, and analyzed for the primary outcome; describe deviations from study as planned, together with reasons	The number of care providers or centers performing the intervention in each group and the number of patients treated by each care provider or in each center
Implementation of interventionb	New item		Details of the experimental treatment and comparator as they were implemented
Recruitment	14	Dates defining the periods of recruitment and follow-up
Baseline datab	15	Baseline demographic and clinical characteristics of each group	When applicable, a description of care providers (case volume, qualification, expertise, etc.) and centers (volume) in each group
Numbers analyzed	16	Number of participants (denominator) in each group included in each analysis and whether analysis was by “intention-to-treat”; state the results in absolute numbers when feasible (e.g., 10/20, not 50 %)
Outcomes and estimation	17	For each primary and secondary outcome, a summary of results for each group and the estimated effect size and its precision (e.g., 95 % confidence interval)
Ancillary analyses	18	Address multiplicity by reporting any other analyses performed, including subgroup analyses and adjusted analyses, indicating those prespecified and those exploratory
Adverse events	19	All important adverse events or side effects in each intervention group
Discussion
Interpretationb	20	Interpretation of the results, taking into account study hypotheses, sources of potential bias or imprecision, and the dangers associated with multiplicity of analyses and outcomes	In addition, take into account the choice of the comparator, lack of or partial blinding, and unequal expertise of care providers or centers in each group
Generalizabilityb	21	Generalizability (external validity) of the trial findings	Generalizability (external validity) of the trial findings according to the intervention, comparators, patients, and care providers and centers involved in the trial
Overall evidence	22	General interpretation of the results in the context of current evidence

1. ^aAdditions or modifications to the CONSORT checklist. CONSORT = Consolidated Standards of Reporting Trials
2. ^bThis item was modified in the 2007 revised version of the CONSORT checklist