Open research questions in Pharmaceutical Economics and Policy
38 unresolved questions extracted from the limitations and future-work sections of 1,005 Pharmaceutical Economics and Policy papers in our library. Each links back to the study that raised it.
What the literature leaves open
Introduction Dose administration aids (DAAs) support medication taking, yet little is known about how pharmacists perceive DAA benefits and challenges.
Community Pharmacist Preferences for Providing a Dose Administration Aid Service in Australia: A Discrete Choice Experiment · 2026 · DOIABSTRACT Rationale Medication wastage is a persistent challenge in healthcare systems, yet the economic impact of syringe dead space (DS) and residual volume during routine medication administration remains underexplored outside the vaccine context.
Unexplored Factors in Medication Wastage: Investigating Economic Impact in a Brazilian Teaching Hospital · 2026 · DOIOPEN QUESTIONS AND THE FUTURE OF THE REPUBLIC The emergence of the Republic model presents society with options. In doing so, it takes some of the first steps towards filling a 30-year gap in the literature, explaining the incentives and resources that underpin the Republic and situating it within a potential shift towards a new model of translational medicine.
Future work will focus on three areas that are essential for facilitating the tool’s implementation and consistent, standardized use in routine daily practice: (i) large-scale dissemination, (ii) development of structured training programmes for pharma- cists, and (iii) integration of the tool into pharmacy infor- mation systems.
Validation of an updated French national tool for reporting pharmacist interventions for all healthcare products · 2026 · DOIFuture research should explore broader green finance measures as firm-level green credit data becomes more systematically available through enhanced environmental information disclosure requirements. This points to possible policy tools for pursuing the dual goals of environmental sustainability and health equity envisioned in the Sustainable Development Goals (SDGs), although direct evidence on health outcomes remains to be established. The mechanisms found in this study are primarily rooted in the context of China, and the cross-country generalizability the findings still needs to be validated. The research sample is limited to A-share listed pharmaceutical companies.
Digital transformation, green finance, and pharmaceutical affordability in China: a health economics perspective · 2026 · DOIIn this research, only approved new drugs were captured. In other words, it should be noted that products for which approval was denied, applications for which approvals were withdrawn, and applications for which approvals were abandoned in Japan were not captured. It should be con- sidered the possibility that sponsors submit only products with high likelihood of approval in Japan. In addition, the impact of the difference in safety profiles between Japan and overseas on the approval in Japan was outside the scope of this research. Author contributions All authors contributed to the conception and design of the research, and to the analysis and interpretation of the data. All authors contributed to the critical revisions for publication. Funding None. Data Availability No datasets were generated or analysed during the current study.
Impact of Japanese Subpopulation Data and Overall Survival in Pivotal Trials on Oncology Drug Approvals in Japan · 2026 · DOIA lack of data and financial and human resources inhibits evaluation of the impact of the EML and exacerbates the influence of external actors, including which products are reviewed and how they are recommended.
The Political Economy of the World Health Organization Model Lists of Essential Medicines · 2025 · DOICONCLUSIONS: In our scoping review of the 340B program, the highest-quality evidence indicates nonprofit, DSHs may be using the 340B program in margin-motivated ways, with inconsistent evidence for increased safety net engagement; however, this finding is not consistent across other hospital types and public health clinics, which face different incentive structures and reporting requirements.
Assessing the Impact of the 340B Drug Pricing Program: A Scoping Review of the Empirical, Peer‐Reviewed Literature · 2024 · DOILiterature with greatest internal validity leaned toward evidence of margin-motivated behavior at the external environment and covered entity levels, with inconsistent findings supporting mission-motivated behavior at these levels; this was particularly the case among participating disproportionate share hospitals (DSHs).
Assessing the Impact of the 340B Drug Pricing Program: A Scoping Review of the Empirical, Peer‐Reviewed Literature · 2024 · DOIThe highest-quality evidence indicates that nonprofit, disproportionate share hospitals may be using the 340B program in margin-motivated ways, with inconsistent evidence for increased safety net engagement; however, this finding is not consistent across other hospital types and public health clinics, which face different incentive structures and reporting requirements.
Assessing the Impact of the 340B Drug Pricing Program: A Scoping Review of the Empirical, Peer‐Reviewed Literature · 2024 · DOIPolicy Points Regulatory agencies may have limited evidence on the clinical benefits and harms of new drugs when deciding whether new therapeutic agents are allowed to enter the market and under which conditions, including whether approval is granted under special regulatory pathways and obligations to address knowledge gaps through postmarketing studies are imposed.
Approval of Cancer Drugs With Uncertain Therapeutic Value: A Comparison of Regulatory Decisions in Europe and the United States · 2020 · DOIPolicy Points The US Food and Drug Administration (FDA) has in recent years allowed onto the market several drugs with limited evidence of safety and effectiveness, provided that manufacturers agree to carry out additional studies while the drugs are in clinical use.
Pharmaceutical Drugs of Uncertain Value, Lifecycle Regulation at the US Food and Drug Administration, and Institutional Incumbency · 2019 · DOIAlternative strategies for reducing ARV prices, such as streamlining financial management systems, improving demand forecasting and removing barriers to generics, should be explored.
Global strategies to reduce the price of antiretroviral medicines: evidence from transactional databases · 2009 · DOIThese reforms began in the late 1980s, as acquired immune deficiency syndrome (AIDS) advocacy organizations openly criticized the FDA, noting that its policies were insufficient to address the prevalence of AIDS and the lag time for FDA approval of new AIDS treatments.
The FDA’s Accelerated Approval Process: Does the Pharmaceutical Industry Have Adequate Incentives for Self-Regulation? · 1999 · DOIOriginality/value The study provides rare empirical evidence from a fragile context, contributing to limited literature on government–donor–academia cooperation and offering a transferable model for resilient, locally led and financially integrated regulatory reform.
Government–donor–academia collaboration model in the pharmaceutical regulatory system and considerations for sustainability in low- and middle-income countries · 2026 · DOIA second open question is whether the two formulations even produce comparable effectiveness, since observed differences may reflect selection on insurance, baseline characteristics, and adherence rather than the drug.
Systematic facility level evidence on what medicines is actually available, at what prices, and at what cost to patients remains scarce, constraining evidence-based policy reform.
Although prior work has identified the structural and economic drivers of these shortages, little is known about whether pharmaceutical plants accumulate operational knowledge from their recovery efforts—tasks that are often irregular and ill-defined.
Abstract Despite recent attention to the role of competition in determining health outcomes in developed nations, little is known about how market power impedes access to quality care in lower-income countries.
In addition, many areas of the world are still lacking clearly defined regulatory processes or incentives for development of orphan medicinal products.
OVERVIEW OF REGULATORY INITIATIVES IN THE EUROPEAN UNION TO STIMULATE RESEARCH AND ACCELERATE ACCESS TO ORPHAN DRUGS AND OTHER HIGH MEDICAL NEED PRODUCTS · 2019 · DOIThe degree to which advisory committees and their respective agency divisions disagree on recommendations has not been well characterized across product and action types.
Association Between Food and Drug Administration Advisory Committee Recommendations and Agency Actions, 2008–2015 · 2019 · DOINevertheless, despite renewed interest in the drug issue in the development cooperation sphere, alternative development remains scarce in policy discussions.
Same script, different play: policy implications of the conceptual struggles around alternative development · 2019 · DOIIt is questionable, however, whether this effort is really justified, because knowledge about the expected size of these order-of-entry advantages is scarce.
The article demonstrates how expert efforts to produce evidence-based policy advice were constrained by an externally induced sense of urgency, avoidance of controversy, internal disunity about drug policy orientation, limited evidence, and the institutional momentum of traditional drug control.
Regulating New Psychoactive Substances in the Czech Republic: Policy Analysis under Urgency · 2015 · DOIIt is insufficient to rely only on pricing policies to reduce drug expenses, given that physicians could circumvent the policy by prescribing more expensive drugs.
Can Price Controls Reduce Pharmaceutical Expenses? A Case Study of Antibacterial Expenditures in 12 Chinese Hospitals from 1996 to 2005 · 2013 · DOI
Most-cited papers in Pharmaceutical Economics and Policy
- Estimated Research and Development Investment Needed to Bring a New Medicine to Market, 2009-2018 · JAMA · 2020 · 1,322 citations
- Costs of Drug Development and Research and Development Intensity in the US, 2000-2018 · JAMA Network Open · 2024 · 198 citations
- Looking Inside the Black Box of Drug Courts: A Meta‐Analytic Review · Justice Quarterly · 2010 · 181 citations
- Comparing Generic Drug Markets in Europe and the United States: Prices, Volumes, and Spending · Milbank Quarterly · 2017 · 120 citations
- Global strategies to reduce the price of antiretroviral medicines: evidence from transactional databases · Bulletin of the World Health Organization · 2009 · 94 citations
- Pegcetacoplan: First Approval · Drugs · 2021 · 91 citations
- Policy options for pharmaceutical pricing and purchasing: issues for low- and middle-income countries · Health Policy and Planning · 2014 · 86 citations
- There is no market for new antibiotics: this allows an open approach to research and development · Wellcome Open Research · 2021 · 71 citations
- Reimbursement models to tackle market failures for antimicrobials: Approaches taken in France, Germany, Sweden, the United Kingdom, and the United States · Health Policy · 2020 · 66 citations
- Approval of Cancer Drugs With Uncertain Therapeutic Value: A Comparison of Regulatory Decisions in Europe and the United States · Milbank Quarterly · 2020 · 65 citations
Most recent work
- Inventory Management Performance for Lifesaving Maternal and Child Health Medicines in Amhara Regional State, Ethiopia: A Facility‐Based Cross‐Sectional Mixed Method Study · Health Science Reports · 2026
- France versus Germany: differences and similarities in determining the added therapeutic value of medicines · The European Journal of Health Economics · 2026
- Product Life Cycle Management and Strategies for Aging Products in Pharmaceutical Marketing · Zenodo (CERN European Organization for Nuclear Research) · 2026
- Impact of Japanese Subpopulation Data and Overall Survival in Pivotal Trials on Oncology Drug Approvals in Japan · Therapeutic Innovation & Regulatory Science · 2026
- Digital transformation, green finance, and pharmaceutical affordability in China: a health economics perspective · Frontiers in Public Health · 2026
- Drug Development Cost Increase Analysis · Zenodo (CERN European Organization for Nuclear Research) · 2026
- DRUG - DEVICE COMBINATION PRODUCTS: COMPARATIVE REGULATORY PATHWAYS IN THE UNITED STATES AND INDIA · Zenodo (CERN European Organization for Nuclear Research) · 2026
- The relationship between changes in prescription drug sales and changes in mortality and hospital utilization in Denmark, 2005–2019 · Figshare · 2026
- From Weight Loss to Legal Loss: How Wegovy Exposed a Hidden Patent Exception · GRUR International · 2026
- Common Agent or Double Agent? Pharmacy Benefit Managers in the Prescription Drug Market · The Review of Economics and Statistics · 2026
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