Economics to the Rescue: Balancing Innovation and Access to High-Cost Therapies

This habilitation thesis explores the intersection of health economics,
regulatory policy, and innovation, focusing on the real-world challenges
posed by high-cost, potentially transformative medical therapies. As
global healthcare systems grapple with aging populations, increasing
complexity of care, and the rising costs of pharmaceuticals—particularly
those with uncertain clinical effectiveness—this work applies economic
frameworks to inform equitable, sustainable, and evidence-based access
strategies.
The thesis comprises five core studies, each contributing to a
nuanced understanding of market dynamics and policy design for novel
therapies. First, it presents a theoretical foundation for alternative
payment models (APMs) for pharmaceuticals, demonstrating that while
uniform pricing can be efficient under ideal conditions, market failures—
such as payer fragmentation, asymmetric beliefs, agency problems, and
treatment adherence issues—can justify the use of value-based pricing
and performance-based agreements.
Second, the thesis includes an economic evaluation of single-dose,
high-cost gene therapies under a fixed budget constraint. Using a case
study of congestive heart failure, it demonstrates how deferred payment
models (DPMs) can improve clinical outcomes and access by enabling
earlier treatment, without increasing overall costs. A Markov model
shows that DPMs can significantly reduce mortality and hospital
admissions, especially when upfront payments are minimized, while
preserving manufacturer revenue and operating within fixed budgets.
Third, the work addresses population-wide therapies for
Alzheimer’s disease (AD), applying a microsimulation model based on
U.S. Health and Retirement Study data. It reveals substantial
misalignment between the timing of therapeutic costs and the accrual of
benefits across public and private payers. The analysis finds that
conventional reimbursement discourages early treatment in
commercially insured patients nearing Medicare eligibility. Simulations
show that installment-based or pay-for-performance payment models
can mitigate this misalignment and support more equitable access.
Fourth, the thesis investigates how non-binding U.S. FDA guidance
influences clinical trialist behavior. Drawing on a regression
discontinuity in time analysis of AD trials, it demonstrates that FDA draft
guidances significantly altered the selection of primary endpoints, particularly the adoption of the CDR-SB composite measure. These
findings underscore the substantial influence of regulatory soft law on
evidence generation and, indirectly, on pricing and reimbursement
decisions.
Fifth, a systematic review of endpoint selection trends in AD clinical
trials complements this analysis by highlighting evolving methodological
preferences among trialists. This research shows how trial design is
shaped by both scientific and policy-driven forces and raises
implications for health technology assessment and payer evaluations.
Collectively, this body of work offers a coherent framework for
understanding and addressing the access challenges posed by high-cost
therapies. By integrating economic theory, empirical modeling, and
policy analysis, it contributes to the design of reimbursement models
that can promote both innovation and equitable access. The insights of
the presented work support policymakers, payers, and other
stakeholders in crafting efficient and fair solutions in an era of rapidly
advancing but expensive therapeutic options and serve as foundation for
ongoing scholarship in the field.