Are there Specific Translational Challenges in Regenerative Medicine? Lessons from Other Fields

Gardner, John; Faulkner, Alex; Mahalatchimy, Aurélie; Webster, Andrew

doi:10.2217/rme.15.50

Cited by 39 publications

(32 citation statements)

References 35 publications

Supporting

Mentioning

Contrasting

Unclassified

Order By: Relevance

“…Many of the reimbursement challenges are not specific to RMPs, because other fields such as orphan drugs can also have high up-front costs [35,36]. However, we maintain that some kinds of RMPs raise specific challenges, such as gene therapies when they are curative [37].…”

Section: ) Discussionmentioning

confidence: 99%

The Emerging Landscape of Reimbursement of Regenerative Medicine Products in the UK: Publications, Policies and Politics

Mahalatchimy

Faulkner

2017

Regen. Med.

Self Cite

View full text Add to dashboard Cite

Aim: This paper aims to map the trends and analyze key institutional dynamics that constitute the policies for reimbursement of regenerative medicine (RM), especially in the UK. Materials & methods: Two quantitative publications studies using Google Scholar and a qualitative study based on a larger study of 43 semi-structured interviews. Results: Reimbursement has been a growing topic of publications specific to RM and independent from orphan drugs. Risk-sharing schemes receive attention among others for dealing with RM reimbursement. Trade organizations have been especially involved on RM reimbursement issues and have proposed solutions. Conclusion: The policy and institutional landscape of reimbursement studies in RM is a highly variegated and conflictual one and in its infancy.

show abstract

Section: ) Discussionmentioning

confidence: 99%

The Emerging Landscape of Reimbursement of Regenerative Medicine Products in the UK: Publications, Policies and Politics

Mahalatchimy

Faulkner

2017

Regen. Med.

Self Cite

View full text Add to dashboard Cite

show abstract

“…Although ATMPs lend themselves naturally to a greater level of coordination, not all of these challenges are uncommon to other breakthrough areas (14). Despite the high investment in R&D during the past decades, or perhaps as a direct consequence, a striking gap remains in innovation reaching patients, as breakthrough science is outpacing the current assessment system in a number of ways (15).…”

Section: Changing Course: Reinventing the Way We Develop Treatmentsmentioning

confidence: 99%

Adaptation through Collaboration: Developing Novel Platforms to Advance the Delivery of Advanced Therapies to Patients

Papadaki

2017

Front. Med.

View full text Add to dashboard Cite

For the nascent field of advanced therapies, collaboration will be a game-changer, turning scientific progress that was once unimaginable into transformative medical practice. Despite promise for lifelong management and even cure of disease, skepticism remains about the feasibility of their delivery to patients, fueling investment risks. With the potential for long-term effectiveness in need of frequent reassessment, current approaches to predict real-life drug performance bear little relevance, necessitating novel and iterative schemes to monitoring the benefit–risk profiles throughout the life span of advanced therapies. This work explains that reinventing an adoption route for Advanced Therapy Medicinal Products is as much about the scientific and clinical components, as it is about the organizational structures, requiring an unprecedented level of interactions between stakeholders not traditionally connected; from developers and regulators, to payers, patients, and funders. By reflecting on the successes and lessons learned from the growing space of global precompetitive consortia and public–private partnerships, as well as a number of emerging accelerated development pathways, this work aims to inform the foundations for a future roadmap that can smooth the path to approval, reimbursement, and access, while delivering value to all stakeholders. Echoing the growing demands to bring these transformative products to patients, it provides critical insights to enhance our capacity in three fundamental domains: deploying the operational flexibilities offered by the growing space of collaborations, utilizing emerging flexible and accelerated pathways to tackle challenges in quantifying long-term effectiveness, and building the necessary digital and clinical infrastructure for knowledge development.

show abstract

“…One of the main challenges identified in the House of Commons and House of Lords reports was the design and conduct of clinical trials, an issue that has also been highlighted in the academic literature (for instance Gardner et al 2015;Webster 2013). Clinical trials form a bridge between the lab and the clinic (Webster 2013, 81), and are thus a key element of so-called translational medicine and an important locus of inquiry for understanding the dynamics of biomedical innovation.…”

Section: Introductionmentioning

confidence: 99%

Imagining the future of cell therapies: clinical trials, innovation and the intersection of clinical-academic and commercial visions

Higham

2019

New Genetics and Society

View full text Add to dashboard Cite

This paper examines the role of clinical trials in regenerative medicine innovation, exploring how trials have contributed to translational challenges in the field. Using data from an ethnographic study of UK cell therapy trials I interrogate the institutional framework for clinical trials and the identitymaking of trialists. This analysis uncovers a disconnect between a commercially-aligned regulatory framework and a clinical-academic identity apparent in the majority of current trialling activity. These different pathways appear to represent two distinct sociotechnical imaginaries for cell therapies; one which reflects the assumptions of commercial innovation and prioritizes economic success, and another which embodies the cultural expectations of academia and emphasizes the importance of clinical care. These two imaginaries operate in synergy to some extent but there are significant tensions between them. How and to what extent these tensions are reconciled is likely to determine both the long-term success and the future shape of the field.

show abstract

Are there Specific Translational Challenges in Regenerative Medicine? Lessons from Other Fields

Cited by 39 publications

References 35 publications

The Emerging Landscape of Reimbursement of Regenerative Medicine Products in the UK: Publications, Policies and Politics

The Emerging Landscape of Reimbursement of Regenerative Medicine Products in the UK: Publications, Policies and Politics

Adaptation through Collaboration: Developing Novel Platforms to Advance the Delivery of Advanced Therapies to Patients

Imagining the future of cell therapies: clinical trials, innovation and the intersection of clinical-academic and commercial visions

Contact Info

Product

Resources

About