Open research questions in Biomedical Ethics and Regulation
26 unresolved questions extracted from the limitations and future-work sections of 601 Biomedical Ethics and Regulation papers in our library. Each links back to the study that raised it.
What the literature leaves open
The enactment of China's Regulation on Clinical Research, Translation, and Application of Novel Biomedical Technologies signifies a shift within the healthcare sector from spontaneous exploration toward institutional standardization in the application of frontier technologies. Looking ahead, the deployment of novel biomedical technologies in China will progressively evolve from discrete applications to comprehensive integration. It is anticipated that an increasing number of routine clinicaltertiary hospitals will establish translation capabilities for such technologies, thereby forming a nationwide clinical-research network. In terms of payment models, as outcome-based payment pilots expand, commercial health insurance products mature, and catastrophic illness insurance catalogs undergo dynamic adjustment, a multi-tiered payment system is expected to crystallize; dedicated commercial insurance products for novel biomedical technologies are likely to emerge in the coming years. Concurrently, with the development of real-world data (RWD) integrated platforms and enhanced analytical capacity, hospitals technologies will be adopting novel biomedical to generate high-quality evidence from positioned reported outcomes, data, reimbursement negotiations, and international regulatory harmonization. Ultimately, the successful integration of novel biomedical technologies into China's healthcare landscape will depend on several critical factors: whether their surpasses conventional medical approaches; whether their quality and end-to-end management withstand rigorous scrutiny; whether these administrators technologies as strategic opportunities for transformation rather than merely revenue-generating tools; and whether the value of clinical personalized medicine.
A PEST-SWOT ANALYSIS OF THE IMPACT OF CHINA'S REGULATORY FRAMEWORK ON CLINICAL RESEARCH AND THE TRANSLATION OF NOVEL BIOMEDICAL TECHNOLOGIES INTO HEALTHCARE APPLICATIONS · 2026 · DOIFor India: Continue to refine the SUGAM portal, expand deemed approval provisions to some foreign-developed drugs, increase staffing for the three-tier system, provide clearer guidance on waiver eligibility, and streamline EC registration. For the United States: Develop clearer guidance on compensation for trial-related injuries, strengthen post-trial access guidance, enhance support for smaller sponsors and academic investigators, and continue modernising electronic submission systems. For harmonisation: Establish mutual recognition agreements for certain categories of data, align safety reporting timelines and formats, develop shared guidance on post-trial access and compensation, expand ICH implementation support, and promote joint training programs for EC/IRB members. Figure 7: Summary of INDIA and USA Comparison. www.wjpr.net │ Vol 15, Issue 10, 2026. │ ISO 9001: 2015 Certified Journal │ 538 Prasad et al.
While our study provides valuable insights into trust dynamics among US patients considering unproven SCIs, several limitations should be acknowledged. Our sample, while diverse in medical conditions and geographic distribution, may not fully represent the broader population of patients interested in experimental treatments, including women, Asian participants, and participants living in remote locations. Additional limitations of sampling have been discussed elsewhere (13). Additionally, the cross- sectional nature of interviews captures attitudes at a single point in time rather than tracking how trust relationships evolve over time. Future research should explore the temporal dynamics of (mis)trust, particularly how experiences with conventional healthcare and unproven SCI clinics shape patient trust in sources, the information they provide, and attitudes over time. As with all qualitative research, our analysis and interpretation may have been shaped by the investigative team’s prior assumptions (confirmatory bias). Although we included a reflexivity statement and took steps to minimize this influence during interviews and analysis (see Supplementary material), residual bias may still be present.
Multiple PASS registries for cell therapies in hematologic malignancies (Crohn's disease perianal fistula with darvadstrocel, EBV+ post-transplant lymphoproliferative disease with tabelecleucel) and gene therapies reference observational multi-national longitudinal designs, but do not specify standardized approaches for capturing long-term efficacy durability endpoints or characterizing disease recurrence patterns that may require comparative effectiveness research methodologies.
Regulatory and Case Analysis of Long-Term Follow-Up for Advanced Therapy Medicinal Products Based on the European Medicines Agency Post-Authorisation Safety Study · 2026 · DOIThe paper references healthcare provider knowledge assessment studies (YESCARTA risk minimisation measures quantitative testing) but does not establish standardized metrics for evaluating the effectiveness of risk minimization strategies in real-world ATMP administration settings or identify gaps in provider compliance with long-term safety monitoring protocols across different European jurisdictions.
Regulatory and Case Analysis of Long-Term Follow-Up for Advanced Therapy Medicinal Products Based on the European Medicines Agency Post-Authorisation Safety Study · 2026 · DOIRisk management plans (RMPs) for ATMPs require long-term follow-up, but the paper does not specify quantitative criteria for determining when PASS studies should be upgraded to prospective study designs versus remaining observational, particularly for rare ATMP populations where patient enrollment in registries (e.g., SMA registry RESTORE) may be insufficient to detect low-frequency safety signals.
Regulatory and Case Analysis of Long-Term Follow-Up for Advanced Therapy Medicinal Products Based on the European Medicines Agency Post-Authorisation Safety Study · 2026 · DOIThe paper identifies CAR-T cell therapy registries (tisagenlecleucel, axicabtagene ciloleucel, idecabtagene vicleucel) and gene therapy registries (valoctocogene roxaparvovec, etranacogene dezaparvovec, voretigene neparvovec) but does not characterize specific long-term adverse event signal detection thresholds or statistical methods for identifying delayed-onset safety signals unique to integrating vector-based therapies, which may have extended latency periods for malignancy or insertional mutagenesis events.
Regulatory and Case Analysis of Long-Term Follow-Up for Advanced Therapy Medicinal Products Based on the European Medicines Agency Post-Authorisation Safety Study · 2026 · DOIThe referenced PASS registries (INSPIRE, RESTORE, GENEr8-GTR, GENEr8-COAS, CSL222_4001) employ observational designs with heterogeneous data collection methodologies across European multi-database linkage studies, but the paper does not address standardized data harmonization approaches or interoperability standards necessary for combining real-world data (RWD) from disparate healthcare systems monitoring ATMP safety outcomes.
Regulatory and Case Analysis of Long-Term Follow-Up for Advanced Therapy Medicinal Products Based on the European Medicines Agency Post-Authorisation Safety Study · 2026 · DOIThe paper catalogs multiple Post-Authorisation Safety Studies (PASS) for advanced therapy medicinal products (ATMPs) including gene therapies and CAR-T cell therapies, but does not specify standardized protocols for long-term follow-up duration across different ATMP modalities. The optimal follow-up periods for gene therapy products (e.g., AAV vectors, retroviral vectors) versus cell therapy products remain inconsistently defined in current regulatory frameworks.
Regulatory and Case Analysis of Long-Term Follow-Up for Advanced Therapy Medicinal Products Based on the European Medicines Agency Post-Authorisation Safety Study · 2026 · DOIThe practice of medicine is quickly evolving with ongoing development and access to technologies that drive interest and implementation of personalized (or precision) medicine. These technological advances tend to focus on the impact of genomics in the delivery of the right med icine to the right patient, but this only partially addresses the critical problem facing physicians and patients today. The more difficult, yet most critical, compo nent for improving patient management and outcome involves understanding the disease process and accurately diag nosing and staging a patient within it.
Comparative effectiveness in CVD SMI, in collaboration with the CNR, led the creation of this consortium involv ing eight countries and 12 institutions to explore the comparative effectiveness in CVD in four areas: CVD risk assessment in asymptomatic patients; Evaluation of acute coronary syndrome patients in the emergency room setting; Evaluation of genotyping in the deter mination of cardiovascular risk; Assessing risk and optimization of patient management in valvular disease in the elderly. This consortium is focused on evaluat ing and improving clinical decision sup port in the management of patients at risk for or with active CVD to improve quality of life for the patient and improve the return on investment for use of critical clinical resources.
Using the case of BiDil, a historical precursor to pharmacogenetic technology, I offer a framework for further studies of high‐technology medicine in which policy analysis is part of a social review based on the justice standard of ex ante mutual advantage.
It is emphasized that liberal principles of making appropriate decisions by doctors should dominate, which in no way can be limited by the current national “Standards of care for…” devoted not to the treatment issues, but, above all, to the principles of organizing care, patient routes, etc.
Reflections on the AAN Position Statement, 2025: Principles for Novel Neurologic Therapeutics: is there a place for liberalism in real clinical practice under the dominance of the principles of evidence-based medicine? · 2025 · DOIExisting Food and Drug Administration efforts are insufficient because of a lack of statutory authority, whereas international examples offer lessons for improving and harmonizing domestic medical device regulatory policy.
However, the use of DTx as a general medical component is still ambiguous, and this ambiguity may be owing to a lack of consensus on a definition, in addition to insufficiencies in research and development, clinical trials, standardization of regulatory frameworks, and technological maturity.
The report recommends some oversight mechanisms-such as intellectual property licensing-previously undiscussed or underexplored in sister reports, and it recognizes that others-like international law-may be impractical.
Although remarkable progress has been made in the research of stem cells, many aspects of their use, especially of embryonic cells, have not been fully clarified and made comprehensible.
The tangled web of competing interests, copyright barriers, inappropriate metrics, misaligned business models, and desire for reward and recognition in traditional publishing could continue to obstruct the imperative that medical research be openly accessible.
The current business models of high-impact journals with copyright barriers need to change to permit full access and reuse of the world's most important medical research and underlying data.
There is a need to reform the misuse of journal-level metrics and move away from using editorial decisions as proxies for the significance of individual research articles and the value of individual authors.
Journal-level metrics, particularly the journal impact factor, are overdue for reassessment and evolution to fulfill the imperative of ensuring research validity and practical relevance.
We suggest that the FDA's arguments defending their practice are insufficient to justify medical research that violates the Declaration of Helsinki.
Most-cited papers in Biomedical Ethics and Regulation
- World Medical Association Declaration of Helsinki · JAMA · 2024 · 1,078 citations
- Remdesivir: First Approval · Drugs · 2020 · 234 citations
- Human embryonic stem cells: research, ethics and policy · Human Reproduction · 2003 · 215 citations
- Casirivimab/Imdevimab: First Approval · Drugs · 2021 · 97 citations
- Daprodustat: First Approval · Drugs · 2020 · 76 citations
- Update on unethical use of placebos in randomised trials · Bioethics · 2003 · 63 citations
- Olverembatinib: First Approval · Drugs · 2022 · 59 citations
- Hydroxychloroquine Controversies: Clinical Trials, Epistemology, and the Democratization of Science · Medical Anthropology Quarterly · 2020 · 56 citations
- The Structure of Clinical Translation: <i>Efficiency, Information, and Ethics</i> · The Hastings Center Report · 2015 · 55 citations
- Regenerative medicine regulatory policies: A systematic review and international comparison · Health Policy · 2020 · 45 citations
Most recent work
- The role of real-world data and real-world evidence in advancing regulatory science and targeted therapeutics: a narrative review from the United States perspective · Personalized Medicine · 2026
- Regulatory and Case Analysis of Long-Term Follow-Up for Advanced Therapy Medicinal Products Based on the European Medicines Agency Post-Authorisation Safety Study · Journal of Pharmacoepidemiology and Risk Management · 2026
- Invited Commentary: Stem Cell Therapies for Spinal Cord Injury · Current Physical Medicine and Rehabilitation Reports · 2026
- 2026 Watch List: Regenerative Medicine · Canadian Journal of Health Technologies · 2026
- Industry updates in advanced therapy medicinal products and regenerative medicine – March 2026 · Regenerative Medicine · 2026
- (Mis)trust among patients seeking unproven stem cell therapies: a qualitative analysis · Frontiers in Medicine · 2026
- Recommendations on post-trial responsibility in implantable neural device research: a multidisciplinary consensus study · BMC Medical Ethics · 2026
- Principles and practices for successful gene therapy innovative contracting: insights from a multistakeholder convening · Journal of Managed Care & Specialty Pharmacy · 2026
- COMPARATIVE STUDY OF CLINICAL TRIAL APPROVAL TIMELINE IN INDIA AND USA · Zenodo (CERN European Organization for Nuclear Research) · 2026
- Why promising ATMPs fail to reach patients: a qualitative stakeholder-informed analysis of cell therapy access in Europe · Journal of Translational Medicine · 2026
Find a gap in your own Biomedical Ethics and Regulation sub-topic
This page shows what the Biomedical Ethics and Regulation literature already flags as unresolved. To narrow it to your specific question, run the guided finder — it searches the gap library on demand and checks candidates against 250M+ OpenAlex works.
Open the Research Gap Finder →