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Economic challenges and possible policy actions to advance stratified medicine
Mark R Trusheim1, Ernst R Berndt2
1Massachusetts Institute of Technology Sloan School of Management, Cambridge, MA, USA. trusheim@MIT.EDU.
Abstract:
In this article, we present a simple economic model to illustrate the economic challenges facing an oncology stratified medicine developer when scientific discoveries lead to ever smaller, targeted patient populations.We provide preliminary empirical evidence suggesting that at least some developers and their investors are retreating. We then examine the armamentarium of policy actions beyond higher reimbursement that may be employed to enhance the economic incentives for developing stratified medicines. In the absence of significant pricing and total oncology outlay flexibility by payers, our analysis suggests that private sector investment in small oncology segments, and in stratified medicine generally, may not prove economically sustainable, thus endangering the translation of scientific advances into bedside medicines. Beyond increasing reimbursement, decreasing development cycle time and costs, or both, would most directly improve the economic incentives facing developers. By contrast, extending exclusivity periods, or initiating advance market commitments and awarding prizes would likely have less impact and involve greater implementation challenges.
Insights
Developing targeted oncology medicines faces economic hurdles due to shrinking patient populations. Policy solutions are needed to ensure the sustainability of stratified medicine investment and innovation.
Area of Science:
- Oncology
- Health Economics
- Pharmaceutical Development
Background:
- Scientific advances are creating smaller, targeted patient populations for cancer therapies.
- This trend presents significant economic challenges for oncology stratified medicine developers.
- Preliminary evidence suggests a potential retreat of developers and investors from this sector.
Purpose of the Study:
- To model the economic challenges in developing stratified medicines for small patient populations.
- To evaluate policy interventions aimed at enhancing economic incentives for stratified medicine development.
- To assess the economic sustainability of private sector investment in niche oncology markets.
Main Methods:
- Development of a simple economic model for stratified medicine.
- Analysis of policy options beyond reimbursement adjustments.
- Examination of empirical evidence on developer and investor behavior.
Main Results:
- Economic sustainability is threatened without significant payer flexibility in pricing and oncology spending.
- Decreasing development time and costs offer the most direct economic improvements for developers.
- Policies like extended exclusivity or advance market commitments may have limited impact.
Conclusions:
- Private sector investment in stratified oncology medicine may be unsustainable without supportive policies.
- Ensuring the translation of scientific advances into new medicines requires addressing economic viability.
- Policy interventions should focus on reducing development costs and timelines for stratified medicines.
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