Meeting the Standards of Normal Science in Health Outcomes and Market ccess
Meeting the Standards of Normal Science in Health Outcomes and Market ccess
Meeting the Standards of Normal Science in Health Outcomes and Market Access for Pharmaceutical Products and Devices
Meeting the Standards of Normal Science in Health Outcomes and Market Access for Pharmaceutical Products and Devices


The Minnesota Guidelines (GFE): Program in Social and administrative Pharmacy, College of Pharmacy, University of Minnesota (Version 2.0 December2016)
  • Current evidence standards and recommendations from professional organizations fail to meet the standards of normal science: they are best seen as pseudoscience supporting intelligent design rather than natural selection
  • Formulary submission standards such as those proposed by the Academy of Managed Care Pharmacy (AMCP) in the US are inadequate and potentially misleading as they put the role of credible, evaluable and replicable claims to one side
  • The proposed GFE standards focus on the credibility of value claims and provide a rigorous and transparent framework for evaluating and reporting on the credibility of value claims
  • In the absence of guidelines that focus on linking credible and replicable claims to prices, there are few constraints on the trajectory of drug pricing behavior in the US
  • Key Features (Version 2.0 December 2016) 
  • To link pricing negotiations and the willingness to accept a manufacturer’s price to the evaluation replication and reporting of credible claims in target patient populations
  • To require replication studies to include comparative claims for product value for therapies in targeted populations
  • To assess whether or not the initial primary and secondary outcomes detailed in phase 3 protocols are consistent with value claims made for the product
  • To link all submissions for formulary approval to a commitment by the manufacturer to underwrite comparative studies to replicate claims in real world treating populations
  • To integrate replication studies of value claims in ongoing disease area and therapeutic reviews
  • To put to one side unevaluable claims for products (e.g., cost per quality adjusted life year claims) in favor of disaggregated value claims that can link back to trial primary and secondary outcome claims
  • To ensure that modeled or simulated claims for comparative effectiveness and product value are meaningful to a formulary committee and can be supported by available data
  • To take explicit account of adherence and persistence in value claims
  • The Submission Protocol 
  • The Minnesota guidelines are unique in requiring all formulary submissions, if requested by the committee, to be accompanied by a protocol to support claims evaluation in the indication for the product and its comparators
  • Version 2.0 of the Minnesota Guidelines taked account of the emergence of next generation sequencing (NGS) in the assessment of claims that link mutations to recommended drug therapies

  • The protocol structure meets required evidentiary standards and ensures continuity from phase 3 study designs to replication studies in proposing (i) a format for product assessment and (ii) a format for the assessment of competing NGS platforms
  • Key requirements are:
    • Systematic reviews to establish the study context for comparative claims and the contribution of the study in meeting these claims
    • A statement of all comparative clinical, cost and other value claims to be made for the product
    • A profile of the target population
    • A description of the data sources to be used to support the study
    • A rationale and description of the study design (e.g., concurrence with good practice, treatment comparators, statistics and analysis plan, statistical hypotheses, sample size)
    • Budget and reporting timeframe
  • Conclusions
  • Given the trajectory of drug launch and post-launch pricing over the past 20 years, it is time to re-visit the basis on which prices link to their expected value in clinical and cost-effectiveness terms
  • Rather than attempting to model claims and address nominal pricing thresholds, the Minnesota approach asks for claims to be validated in target populations as an integral part of the price setting and negotiation process
  • To establish a transparent process to support aligning a ‘fair’ price for a drug with its benefits


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