Regenerative Medicine: From Bench to Bedside to Market

Dear Colleagues,

We are pleased to invite submissions for a Special Issue of the Journal of Market Access and Health Policy (JMAHP) dedicated to the multifaceted challenges and opportunities surrounding regenerative medicine, including gene therapies, cell therapies, tissue-engineered products, and related advanced therapy medicinal products (ATMPs).

Regenerative medicine stands at a pivotal juncture. While the pipeline of transformative therapies continues to expand, offering potentially curative treatments for previously intractable conditions, their translation into sustained and equitable patient access remains fraught with unprecedented challenges. These challenges span the entire value chain, from the complexities of clinical development and regulatory evaluation to the fundamental uncertainties surrounding long-term efficacy, durability of effect, and the management of potential waning therapeutic benefit over time. Simultaneously, health systems worldwide grapple with the economic implications of high-cost, potentially one-time treatments, raising critical questions about pricing, funding models, health technology assessment (HTA), Joint Clinical Assessment (JCA), and return on investment.

This Special Issue aims to bring together original research, systematic reviews, commentaries, and policy analyses addressing, but not limited to, the following topics:

Clinical development and trial design for regenerative therapies; regulatory pathways and conditional approvals; long-term efficacy, durability of effect, and management of potential waning therapeutic benefit; safety monitoring and risk management; market access strategies and HTA/JCA methodologies for ATMPs; health economic evaluation and cost-effectiveness modelling; innovative pricing, payment, and funding mechanisms (including amortisation, outcomes-based contracts, and managed entry agreements); real-world evidence generation and post-market surveillance; value assessment frameworks, return on investment, and commercialisation challenges; manufacturing scalability, supply chain logistics, and production upscaling; academic development of ATMPs; uncertainty quantification and decision-making under uncertainty; equity of access, global disparities, and the challenges facing low- and middle-income countries; public perception, hesitancy, patient preferences and ethical considerations related to genetic modification and DNA-based interventions; and the role of artificial intelligence and digital tools in supporting regenerative medicine development and access.

This call is addressed to researchers, clinicians, health economists, regulatory scientists, HTA practitioners, policy experts, and industry professionals working at the intersection of regenerative medicine and healthcare decision-making. We welcome contributions from diverse disciplinary perspectives and geographical contexts.

Submission Guidelines: Manuscripts should be prepared in accordance with the JMAHP author guidelines, available on the journal’s website. All submissions will undergo rigorous peer review.

We look forward to receiving your contributions and to advancing the scientific discourse on this critically important and timely topic.

Sincerely,

Prof. Dr. Mondher Toumi
Prof. Dr. Steven Simoens
Guest Editors

Manuscript Submission Information

Manuscripts should be submitted online at www.mdpi.com by registering and logging in to this website. Once you are registered, click here to go to the submission form. Manuscripts can be submitted until the deadline. All submissions that pass pre-check are peer-reviewed. Accepted papers will be published continuously in the journal (as soon as accepted) and will be listed together on the special issue website. Research articles, review articles as well as short communications are invited. For planned papers, a title and short abstract (about 250 words) can be sent to the Editorial Office for assessment.

Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). All manuscripts are thoroughly refereed through a double-anonymized peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. Journal of Market Access & Health Policy is an international peer-reviewed open access quarterly journal published by MDPI.

Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 1500 CHF (Swiss Francs). Submitted papers should be well formatted and use good English. Authors may use MDPI's English editing service prior to publication or during author revisions.

• Advanced Therapy Medicinal Products (ATMPs)
• regenerative medicine
• clinical development
• regulatory affairs
• Health Technology Assessment (HTA)
• market access
• innovative payment models
• real-world evidence
• pricing and reimbursement