Open research questions in Biomedical Ethics and Regulation
73 unresolved questions extracted from the limitations and future-work sections of 740 Biomedical Ethics and Regulation papers in our library. Each links back to the study that raised it.
What the literature leaves open
How post-approval requirements are utilized to complement these specific evidence gaps remains unclear.
Addressing Efficacy Uncertainties in Expedited Programs for Gene Therapy Products: A Comparative Assessment of Post-Approval Requirements by the FDA and EMA · 2026 · DOIBackground: Early feasibility studies (EFSs) are small-scale clinical investigations conducted during the early development of medical devices to assess initial safety and performance, especially when bench or in-silico testing is insufficient.
Stakeholder Perspectives on Early Feasibility Studies for Digital Health Technologies in the European Union: Qualitative Interview Study · 2025 · DOIDespite the recognized potential of EFSs to support iterative, user-centered innovation, little is known about how European DHT companies and contract research organizations (CROs) perceive and implement EFSs, or what barriers and opportunities exist for broader adoption.
Stakeholder Perspectives on Early Feasibility Studies for Digital Health Technologies in the European Union: Qualitative Interview Study · 2025 · DOIThe implementation of LTFU requirements across different ATMP types has not been systematically analyzed.
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 · DOIMultiple 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 · DOIQuestions remain regarding scalability and manufacturing models for bioengineered skin products. 3D bioprinting and bioinks still face major challenges before they can be clinically implemented. Further progress in academic settings is needed for 3D bioprinting and bioinks.
There is a need to anticipate system-level changes to implement regenerative medicine across Canada. There is a lack of effective treatment options for many rare diseases. There are challenges in scalability and manufacturing models for bioengineered skin products.
There is a lack of understanding about how patients describe interpersonal and institutional trust in relation to their interest in and decisions about unproven stem cell interventions. There is a need to examine the factors that influence patients' trust in the unproven stem cell intervention industry.
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.
Further development of genetic tests and screening protocols is needed. Improved support for patients and caregivers in navigating the healthcare system is necessary.
Principles and practices for successful gene therapy innovative contracting: insights from a multistakeholder convening · 2026 · DOIMany patients face delays in diagnosis and treatment, resulting in significant financial burdens. Gene therapy innovative contracting is a complex process.
Principles and practices for successful gene therapy innovative contracting: insights from a multistakeholder convening · 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.
The rise of advanced therapy medicinal products demands a radically different regulatory approach. The need for a more strategic, data-driven approach to regulatory affairs. The importance of upskilling regulatory professionals to remain relevant in a rapidly changing environment.
The next horizon for regulatory harmonization is no longer about merely aligning technical requirements but about operationalizing collaborative assessments at scale. The future of regulatory affairs will be defined by a fundamental reimagining of how global regulators collaborate and how technology transforms compliance.
There is a need to understand the association between Expedited Programs and Time to Approval. The current study addresses this gap by examining the impact of Expedited Programs on the development and approval of new molecular entities.
The implementation of the proposed model will require careful harmonization of consent and preference categories. The model may not be suitable for all jurisdictions and repositories of PSCs and their derivatives. The paper acknowledges the need for empirical evaluation of donor experience and administrative and resource burden.
Reconceptualizing the right to withdraw in stem cell research: Striking a balance between donor autonomy and scientific utility · 2026 · DOIEmpirical evaluation of donor experience and administrative and resource burden. Development of tiered preference taxonomies and participant recontact thresholds. Pilot implementations and comparative assessments of different governance models.
Reconceptualizing the right to withdraw in stem cell research: Striking a balance between donor autonomy and scientific utility · 2026 · DOILack of GMP-compliant cell-manufacturing facilities and interdisciplinary clinical talent in Chinese hospitals. Uneven ethical-review capacity and unbalanced regional development.
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 · DOIFurther study on the implementation of the proposed strategies. Investigation of the effects of the regulatory framework on the healthcare sector over time.
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 · DOIFurther exploration of the use of TIL as a foundation for other types of CAR therapies. Investigation of the potential for improved treatment of solid tumors using CAR-enhanced TIL.
Letter to the Editor for submission to journal of translational medicine contextual programming of CAR‑enhanced TIL · 2026 · DOIThe limited efficacy of engineered T-cells against solid tumors due to antigen heterogeneity and immunosuppressive microenvironments. The need for a novel approach to enhance effector function against autologous tumor tissue.
Letter to the Editor for submission to journal of translational medicine contextual programming of CAR‑enhanced TIL · 2026 · DOIThe lack of real-world evidence to support or refute concerns about product drift in biosimilars. The need for a better understanding of the stability of critical quality attributes in biosimilars.
Why Product Drift Is Not an Issue for Biosimilars/Biologics: Start the Same, Stay the Same · 2026 · DOIThe current labelling information for medical devices and in-vitro diagnostics is not scalable and accessible. There is a need for a comprehensive and standardized digital labelling solution.
The lack of a single route to navigate the regulatory process poses a challenge for drug repurposing. Off-label use of a drug repurposing candidate is considered problematic. Fair pricing principles should be implemented to ensure equitable access to repurposed drugs.
Navigating regulatory scenarios for accelerating drug repurposing in rare diseases: a multi-stakeholder perspective on the challenges and opportunities · 2026 · DOIThe paper does not provide a comprehensive analysis of the regulatory challenges and opportunities for basket trials in drug repurposing. The multi-stakeholder dialogue session was limited to 28 experts from eight European countries.
Navigating regulatory scenarios for accelerating drug repurposing in rare diseases: a multi-stakeholder perspective on the challenges and opportunities · 2026 · DOI
Most-cited papers in Biomedical Ethics and Regulation
- Ethics and Clinical Research · New England Journal of Medicine · 1966 · 1,297 citations
- Credibility of analogue therapy rationales · Journal of Behavior Therapy and Experimental Psychiatry · 1972 · 1,186 citations
- World Medical Association Declaration of Helsinki · JAMA · 2024 · 1,078 citations
- Herbal medicine research and global health: an ethical analysis · Bulletin of the World Health Organization · 2008 · 292 citations
- A Critique of Clinical Equipoise: Therapeutic Misconception in the Ethics of Clinical Trials · The Hastings Center Report · 2003 · 282 citations
- The Drugs Don't Work · Social Studies of Science · 2003 · 246 citations
- Remdesivir: First Approval · Drugs · 2020 · 234 citations
- Human embryonic stem cells: research, ethics and policy · Human Reproduction · 2003 · 215 citations
- Clinical Trials in Rare Disease · Journal of Child Neurology · 2013 · 209 citations
- Regulatory objectivity and the generation and management of evidence in medicine · Social Science & Medicine · 2006 · 200 citations
Most recent work
- Clinical translational research on stem cell products: prospects and challenges · Signal Transduction and Targeted Therapy · 2026
- Integration of stem cell therapy and artificial intelligence: From promise to clinical reality · World Journal of Stem Cells · 2026
- Defining core competencies for advanced therapy medicinal products translation · Frontiers in Digital Health · 2026
- 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
- South America and Mexico’s cell and gene therapy landscape: accelerating growth and persistent challenges · Cytotherapy · 2026
- Shared decision-making and integrative medicine in the allergist’s office · Annals of Allergy, Asthma & Immunology · 2026
- A Love Letter to Clinical Trials · JAMA · 2026
- Politicisation of the US FDA: eroding integrity and trust · The Lancet · 2026
- “Beyond the state: First-person ethics and the governance of targeted therapies in China” · Social Science & Medicine · 2026
- The value of patient-focused drug development · Nature Medicine · 2026
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