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Open research questions in Pharmaceutical Economics and Policy

257 gap statements mined from Pharmaceutical Economics and Policy papers in our 7.9M-paper local library, which holds 2,648 papers on the topic — drawn mostly from each paper's own stated research gap, future-work, challenge and limitation notes, and its abstract. The ones listed below are a selection still marked open; each names the study that raised it, with a DOI link where the paper has one.

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What the literature leaves open

  • It may also enable the early identification of potential gaps in drug supply across specific therapeutic areas and research support for the development of needed therapies. With regard to EMA MAPs, evaluating these procedures can help identify potential areas for optimization at an early stage.

    Analysis of marketing authorizations, withdrawals, and refusals by the European Medicines Agency (EMA): trends in approval procedures 1995–2025 · 2026 · DOI

  • The extent of added benefit and the target population size can only partially explain the large heterogeneity of results. There is a need to understand the factors that influence prices and costs of orphan drugs.

    Price development of orphan drugs in Germany after market access and subsequent assessments: a descriptive analysis · 2026 · DOI

  • The need to account for the price cross-referencing concern associated with the wide reliance on ERP. The challenge of constructing a measure of the number of domestic competitors at the therapeutic level.

    The Impact of Health Technology Assessment on Pharmaceutical Prices: Evidence from Germany · 2026 · DOI

  • The gap is that previous work does not account for the possibility that including future price falls may result in higher prices during the patent period. The gap is also that most reimbursement agencies do not consider future price falls in their economic evaluations. The paper aims to fill this gap by exploring the consequences of including off-patent prices in cost-effectiveness analyses.

    Exploring the consequences of including off-patent prices in reimbursement decisions for patented drugs · 2026 · DOI

  • One challenge is the complexity of pharmaceutical innovation and the need for structured frameworks for innovativeness appraisal. Another challenge is the variability in regulatory timelines and the need for a comprehensive overview of the Italian framework.

    Closing the first wave of drug innovativeness in italy: final evidence from 2017 to 2025 · 2026 · DOI

  • The gap in previous analyses is the lack of a comprehensive overview of the Italian framework for drug innovativeness appraisal. The study extends previous analyses to cover the complete set of appraisals publicly available as of June 2025.

    Closing the first wave of drug innovativeness in italy: final evidence from 2017 to 2025 · 2026 · DOI

  • The lack of transparency and heterogeneous methodologies in pricing policies for Innovative Medicines and Advanced Therapies. The need to better understand and refine pricing approaches for these products. The importance of establishing fair and transparent pricing frameworks that balance access, incentives for innovation, and the sustainability of health systems.

    Pricing of innovative medicines and advanced therapies worldwide: an scoping review · 2026 · DOI

  • There is a lack of evidence on the effect of higher out-of-pocket payments on drug prices and demand.

    The effect of higher out-of-pocket payments on drug prices and demand · 2026 · DOI

  • The risk of approving reformulations that may lead to harm or waste will grow as 505(b)(2) use expands. Pharmaceutical marketing can influence prescribing behavior and lead to the increased use of branded and higher-cost drugs. The lack of formal safety trials for benzgalantamine raises concerns about its safety and effectiveness.

    FDA approval of benzgalantamine exposes flaws in the 505(b)(2) pathway for innovations on existing drugs · 2026 · DOI

  • To fulfill the growing demand for next-generation vaccines, including those against emerging pathogens and new indications, a multipronged de-risking approach is needed that can shift vaccine development towards a more streamlined and lower-risk process.

    De-risking vaccine development: lessons, challenges, and prospects · 2025 · DOI

  • One challenge is the lack of mature OS data for many cancer indications at the time of NRDL listing. Another challenge is the use of surrogate endpoints instead of OS gains in the approval of new cancer drugs. The study also faces the challenge of assessing drugs with insufficient evidence of clinical benefits.

    Evolution of evidence on overall survival benefits of cancer drugs included on the national reimbursement drug list of China, 2005–2022: an observational study · 2025 · DOI

  • The gap in the current research is the lack of understanding of how the evidence of overall survival benefits of cancer drugs included on China's National Reimbursement Drug List has evolved over time. The study aims to address the challenge of assessing drugs with insufficient evidence of clinical benefits.

    Evolution of evidence on overall survival benefits of cancer drugs included on the national reimbursement drug list of China, 2005–2022: an observational study · 2025 · DOI

  • Generic drugs in China used to lack of standards and regulation, and GCE is regarded as the solution.

    Dynamic effects of China’s national volume-based procurement on generic drug consistency evaluation: An interrupted time series analysis · 2026 · DOI

  • Future research should examine discordance in approval rates while taking into account therapeutic value, pricing and reimbursement, preferably while encompassing both orphan and non-orphan drugs.

    Discordance in orphan drug approvals between the U.S. Food and Drug Administration and the European Medicines Agency: A retrospective observational analysis · 2026 · DOI

  • To enhance cost-effectiveness within the Brazilian National Health System, mechanisms for centralized price negotiation or procurement should be further explored.

    Variability in public procurement prices for Group 1A drugs in the specialized pharmaceutical component: an observational study, 2013-2022 · 2025 · DOI

  • While the overall impact of pooled drug procurement on drug procurement has been evaluated, little is known about how healthcare institutions’ pre-policy procurement composition is associated with heterogeneous post-policy procurement responses.

    Understanding Heterogeneous Drug Procurement Behaviour of Healthcare Institutions Under Pooled Procurement: Evidence From China · 2026 · DOI

  • Decisions about what drugs should be publicly covered and how long it took to make those decisions were not investigated.

    Orphan Drug Approval in Canada, 1999-2022: A Cross-sectional Study · 2025 · DOI

  • The long-term impacts of the National Essential Medicines Policy (NEMP) in remote and rural areas of China remain underexplored.

    Longitudinal Trends in Medicine Supply, Price and Utilisation in Primary Care Facilities in Rural Southwestern China Under National Essential Medicines Policy (2012-2017): Disparities Across Facilities and Medicines · 2025 · DOI

  • While there was some knowledge of generic medications' cost advantages, still significant gaps in knowledge and trust might hinder their widespread adoption.

    Knowledge and Perception of Consumers Towards Generic Medicines in Saudi Arabia: A Prospective Cross-Sectional Survey · 2025 · DOI

  • While this legislative initiative marks an important step toward incentivizing further research into known compounds, the paper argues that IP-based solutions alone cannot overcome systemic obstacles.

    Between Patents and Patients: Why Intellectual Property Alone Cannot Sustain Drug Repurposing · 2025 · DOI

  • Limited information is available regarding the distribution of increasing pharmaceutical expenditures within large representative samples of national populations globally.

    Characteristics of High-Cost Beneficiaries of Prescription Drugs in Kazakhstan: A Cross-Sectional Study of Outpatient Data from 2022 · 2024 · DOI

  • However, it remains to be seen to what extent national differences, particularly in appraisal, will persist despite joint evidence processing within the framework of a common EU HTA dossier.

    France versus Germany: differences and similarities in determining the added therapeutic value of medicines · 2026 · DOI

  • Incomplete and non-standardized reporting reduces the ability to assess financial performance and limits cross-country comparability.

    A comparative analysis of managed entry agreements in Europe: trends, reporting practices, and financial outcomes · 2026 · DOI

  • The review did not assess the impact of pricing policies on access to IMs and ATs in detail, - The review did not evaluate the effectiveness of different pricing policies, - The review was limited to publications describing pricing policies for IMs…

    Pricing of innovative medicines and advanced therapies worldwide: an scoping review · 2026 · DOI

  • Further research could investigate the effects of out-of-pocket price differentiation on other healthcare services, - Further research could examine the impact of the policy on healthcare…

    The effect of higher out-of-pocket payments on drug prices and demand · 2026 · DOI

Most-cited papers in Pharmaceutical Economics and Policy

Most recent work

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Open questions raised by papers listed on this page include: (1) There is a need to understand the factors that influence prices and costs of orphan drugs (“Price development of orphan drugs in Germany after market access and subsequent…”, 2026); (2) The gap in previous analyses is the lack of a comprehensive overview of the Italian framework for drug innovativeness appraisal (“Closing the first wave of drug innovativeness in italy: final evidence from…”, 2026); (3) The lack of transparency and heterogeneous methodologies in pricing policies for Innovative Medicines and Advanced Therapies (“Pricing of innovative medicines and advanced therapies worldwide: an scoping…”, 2026); (4) There is a lack of evidence on the effect of higher out-of-pocket payments on drug prices and demand (“The effect of higher out-of-pocket payments on drug prices and demand”, 2026); (5) The lack of formal safety trials for benzgalantamine raises concerns about its safety and effectiveness (“FDA approval of benzgalantamine exposes flaws in the 505(b)(2) pathway for…”, 2026). 257 gap statements have been mined from Pharmaceutical Economics and Policy papers in our 7.9M-paper local library, which holds 2,648 papers on the topic; the gaps come from whichever of those papers state one. They are mostly the research gaps the authors state and the papers' abstracts, plus future-work, limitations and challenges passages. The ones listed below are a selection still marked open; each names the study that raised it, with a DOI link where the paper has one, so you can read the original claim in context.

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