What do efficacy, effectiveness, and efficiency have to do with late phase clinical trials? Dr. Michael F. Murphy discusses in his latest blog post, which lays out the necessity to connect the three “Es” in late phase clinical programs.
Prediction #3: Efficiency as Well as Effectiveness Required for Product Approvals
Late phase clinical development programs must strive to connect efficacy, effectiveness, and efficiency.
- Efficacy: product attributes in idealized patients and investigators
- Effectiveness: product attributes in representative patients by representative physicians
- Efficiency: the economic impact of new therapy on a system of care
In an era when balancing care delivery with cost containment is mandatory, either trial-based studies using patient-level data or decision analytic modeling using secondary data are necessary contributors. Trial-based approaches are creditable but are difficult to complete before drug approval and have limitations given protocol structure, eligibility criteria, inability to capture outcomes based on trial duration, and methods of analysis.
Nevertheless, the ability to create “piggyback” programs, which assess the economic value and estimate the impact of novel therapeutics on a system of care as well as their clinical utility, is a requirement. Although the FDA currently does not have the authority to prevent market authorization for novel or repurposed therapeutic entities that have not demonstrated an impact on healthcare utilization, pilot programs between the FDA and the Centers for Medicare and Medicaid Services (CMS) exist. It is anticipated that a decision process for product approval that resembles the United Kingdom’s NICE (The National Institute for Health and Care Excellence) will evolve both within the United States and worldwide. Thus, CROs that can ensure access and approval by creating an integrated development program provide valued differentiation.