CEO Executive AssistantPharma CEO HEOR time management health economics has become a strategic discipline that pharmaceutical CEOs can no longer delegate entirely to the medical affairs and market access teams. Health economics and outcomes research (HEOR) now determines whether a drug receives adequate payer coverage after FDA approval, whether it survives cost-effectiveness challenges from organizations like ICER, and whether the company can demonstrate value to an increasingly evidence-demanding payer community. The CEO who is not personally invested in HEOR strategy is ceding control of a major determinant of commercial success.
Why HEOR Is Now a CEO-Level Obligation
A generation ago, HEOR was a specialized function within medical affairs that produced cost-effectiveness models and published clinical outcomes analyses. The payer community used HEOR evidence, but it was rarely the determining factor in formulary decisions for innovative medicines.
The landscape has fundamentally changed. ICER (the Institute for Clinical and Economic Review) publishes independent cost-effectiveness assessments that directly affect payer formulary decisions and negotiating leverage. Large payers and PBMs have built internal HEOR capabilities that scrutinize manufacturer-provided evidence with increasing rigor. CMS increasingly references HEOR frameworks in its IRA negotiation value assessments. International health technology assessment bodies (NICE in the UK, IQWiG in Germany, HAS in France) use cost-effectiveness thresholds that directly determine market access and pricing in those countries.
The CEO who treats HEOR as a support function will find that the company’s HEOR evidence program is not adequately resourced, not strategically positioned relative to the payer evidence expectations, and not generating the real-world evidence data that payers require for formulary decisions on high-cost innovative therapies. Correcting these deficiencies after approval is far more expensive and time-consuming than building the evidence program correctly from Phase 2 forward.
HEOR Evidence Generation Governance
The CEO’s most important HEOR governance decision is the investment framework: how much capital is allocated to HEOR evidence generation, what types of studies are prioritized, and how HEOR investment is balanced against clinical trial investment.
HEOR evidence generation encompasses a wide range of study types: prospective observational studies using patient registries, retrospective database analyses using insurance claims or electronic health record data, patient-reported outcome (PRO) studies that capture quality of life and functional status, indirect treatment comparisons that position the drug’s clinical benefits relative to alternatives, and health utility studies that generate the utility weights used in quality-adjusted life year (QALY) calculations.
The CEO must govern HEOR investment with explicit attention to the payer evidence requirements for the therapeutic area and patient population. For oncology drugs, payers are increasingly sophisticated about progression-free survival versus overall survival data, and HEOR investment in long-term survival registry studies is essential. For chronic disease drugs, medication adherence and patient-reported quality of life are primary payer evaluation criteria. For rare diseases, natural history evidence that establishes the disease burden is often the most important HEOR investment.
ICER Engagement Strategy
ICER’s therapeutic area reviews produce value assessments that are increasingly referenced by payers in formulary decisions and by state Medicaid programs in coverage policy. ICER’s comparative effectiveness reviews use a cost-per-QALY framework that has been controversial in rare disease and pediatric contexts where small patient populations make QALY calculations methodologically challenging.
The CEO must invest time in ICER engagement strategy that encompasses both the pre-assessment phase (providing evidence to ICER during its scoping process) and the assessment phase (engaging with ICER’s technical review through the public comment process, participating in ICER public meetings, and responding to final assessment publication).
The strategic question for the CEO is not simply whether to engage with ICER but how proactively to invest in the relationship. Companies that engage with ICER early, provide data transparently, and participate constructively in the review process typically receive assessments that better reflect the drug’s actual value, because ICER’s analysts are working with more complete information. Companies that resist ICER engagement and treat the assessment process as adversarial tend to receive assessments that rely on conservative assumptions about clinical benefit and cost-effectiveness.
NIH’s National Institute for Health Care Management Foundation resources on comparative effectiveness provide context for the evidence standards that HEOR evidence programs must meet to be credible in comparative effectiveness assessments.
Payer Dossier Preparation Oversight
Payer dossiers, formal evidence packages submitted to payers and pharmacy and therapeutics committees to support formulary placement decisions, are the primary commercial deliverable of the HEOR function. The quality of the payer dossier directly affects formulary placement outcomes, particularly for high-cost specialty drugs that receive active P&T committee review.
The CEO must invest time in overseeing payer dossier preparation as a commercial priority, not as a medical affairs deliverable. Payer dossiers should be developed with the following CEO-level questions answered: What is the payer’s primary concern about this drug (cost, appropriate use, long-term outcomes uncertainty)? What evidence does the dossier need to address that concern specifically? Who are the key P&T committee decision-makers at the top payers, and has the medical affairs team engaged them before the formal P&T review?
The CEO should review the payer dossier for major products personally before it is submitted to payers, not to evaluate the technical content in detail (that is the CMO and HEOR team’s responsibility) but to assess whether the value narrative is compelling, the evidence is presented clearly for a non-specialist audience, and the cost-effectiveness claims are defensible under challenge.
Real-World Evidence Study Design
Real-world evidence (RWE) studies use data from routine clinical practice to generate evidence about drug effectiveness, safety, and outcomes in patient populations broader and more diverse than those enrolled in clinical trials. FDA’s RWE framework, established in the 21st Century Cures Act and implemented through subsequent guidance, has expanded the potential regulatory uses of RWE. Payers are using RWE to evaluate long-term drug performance beyond the clinical trial evidence base.
The CEO must invest time in RWE study design governance because the design choices (data source selection, study population definition, outcome measure specification, comparison group selection) determine whether the resulting RWE is credible to payers and regulators or subject to the methodological criticisms that frequently undermine RWE credibility.
RWE study design requires explicit attention to the potential for confounding (patients who receive the drug may differ systematically from those who do not in ways that affect outcomes), which must be addressed through appropriate analytic methods (propensity score matching, inverse probability weighting, instrumental variable approaches). The CEO should understand the RWE study design well enough to ask the right governance questions, even without the technical expertise to evaluate the analytic methods in detail.
HEOR Publication Strategy
The HEOR publication strategy determines how the company’s evidence base is communicated to the scientific and payer community through peer-reviewed journals, conference presentations at ISPOR and AMCP, and health policy publications. A well-executed publication strategy ensures that the most important HEOR evidence reaches the relevant audiences before payer formulary decisions, rather than after.
The CEO must govern the HEOR publication strategy by ensuring it is coordinated with the commercial launch timeline and payer engagement calendar. A cost-effectiveness model that is published six months after the drug’s launch is of limited value for the formulary decisions made in the first year post-approval. The publication strategy must be planned reverse-engineered from the payer engagement timeline.
The CEO should also invest time in ensuring the HEOR publication strategy is independent and credible. Payers who view the company’s HEOR publications as sponsored advocacy rather than objective evidence will discount the evidence quality accordingly. Publishing in high-quality journals (Pharmacoeconomics, Value in Health, JAMA, New England Journal of Medicine) and engaging academic co-investigators with genuine scientific independence enhances the credibility of the company’s HEOR evidence base.
For context on managing parallel evidence generation and commercial investment governance, time management for pharma CEOs during post-approval commitments covers the evidence generation governance obligations that continue after FDA approval.
HEOR vs. Clinical Trial Investment Balance
One of the most consequential resource allocation decisions the CEO makes is the balance between HEOR investment and clinical trial investment. Clinical trial investment directly affects the regulatory evidence base and the company’s pipeline progression. HEOR investment affects the commercial success of approved products and their ability to achieve adequate payer coverage.
In practice, HEOR investment is frequently underfunded relative to its commercial importance because the clinical trial budget is managed by the development organization (which has direct CEO attention) while HEOR investment is managed by medical affairs (which typically receives less CEO attention). The result is that companies often arrive at approval with robust clinical trial evidence and insufficient HEOR evidence to support formulary access for high-cost drugs.
The CEO must establish an explicit HEOR investment framework that is governed at the same level as clinical trial investment, with HEOR evidence generation milestones tracked alongside clinical development milestones in the CEO’s program review cadence.
Conclusion: Pharma CEO HEOR Time Management Health Economics as Commercial Strategy
Pharma CEO HEOR time management health economics is now inseparable from commercial strategy for innovative pharmaceutical companies. The payer community’s increasing sophistication about health economics evidence, the influence of ICER’s cost-effectiveness assessments on formulary decisions, and the growing importance of real-world evidence in demonstrating drug value beyond the clinical trial environment all require the CEO to invest personally in governing the HEOR evidence generation program.
CEOs who treat HEOR as a specialist function and delegate its governance entirely to medical affairs will find that their drugs face formulary access challenges that are more severe and more durable than those of competitors who invested in superior HEOR evidence generation. The investment in HEOR evidence is, ultimately, the investment in commercial success after approval.
Related Reading
For further context, explore How Pharma CEOs Balance Regulatory Affairs and Commercial Time and How Pharma CEOs Manage Time During Major Conference Season.