Bringing Regenerative Medicines to the Clinic: The Future for Regulation and Reimbursement

Bubela, Tania; McCabe, Christopher; Archibald, Peter; Atkins, Harold L.; Bradshaw, S.; Kefalas, Panos; Mujoomdar, Michelle; Packer, Claire; Piret, James M.; Raxworthy, Mike; Soares, Marta; Viswanathan, Sowmya

doi:10.2217/rme.15.51

Cited by 43 publications

(33 citation statements)

References 29 publications

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“…Expedited‐approval pathways and programs, priority review, or programs alternative to the standard review processes for medical products have been developed, and legislation has been enacted by the U.S. Food and Drug Administration and the European Medicines Agency 66, 67. Recently, the Japanese government reformed its pharmaceutical affairs legislation and created a new regulation called the Pharmaceuticals, Medical Devices, and Other Therapeutic Products Act in November 2014 68.…”

Section: Discussionmentioning

confidence: 99%

Liver tissue engineering: From implantable tissue to whole organ engineering

Mazza

Al‐Akkad

Rombouts

et al. 2017

Hepatology Communications

101

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The term “liver tissue engineering” summarizes one of the ultimate goals of modern biotechnology: the possibility of reproducing in total or in part the functions of the liver in order to treat acute or chronic liver disorders and, ultimately, create a fully functional organ to be transplanted or used as an extracorporeal device. All the technical approaches in the area of liver tissue engineering are based on allocating adult hepatocytes or stem cell‐derived hepatocyte‐like cells within a three‐dimensional structure able to ensure their survival and to maintain their functional phenotype. The hosting structure can be a construct in which hepatocytes are embedded in alginate and/or gelatin or are seeded in a pre‐arranged scaffold made with different types of biomaterials. According to a more advanced methodology termed three‐dimensional bioprinting, hepatocytes are mixed with a bio‐ink and the mixture is printed in different forms, such as tissue‐like layers or spheroids. In the last decade, efforts to engineer a cell microenvironment recapitulating the dynamic native extracellular matrix have become increasingly successful, leading to the hope of satisfying the clinical demand for tissue (or organ) repair and replacement within a reasonable timeframe. Indeed, the preclinical work performed in recent years has shown promising results, and the advancement in the biotechnology of bioreactors, ex vivo perfusion machines, and cell expansion systems associated with a better understanding of liver development and the extracellular matrix microenvironment will facilitate and expedite the translation to technical applications. (Hepatology Communications 2018;2:131–141)

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Section: Discussionmentioning

confidence: 99%

Liver tissue engineering: From implantable tissue to whole organ engineering

Mazza

Al‐Akkad

Rombouts

et al. 2017

Hepatology Communications

101

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“… 7 The importance of aligning the regulatory processes and reimbursement strategies to suit all stakeholders is vital to this process and has been identified by national bodies such as the National Institute for Health and Care Excellence (NICE, UK) and the Centre for Commercialization of Regenerative Medicine (CCRM, Canada). 12 This will, hopefully, enable the increased adoption of these products into the clinical setting.…”

Section: Discussionmentioning

confidence: 99%

A quantitative, multi-national and multi-stakeholder assessment of barriers to the adoption of cell therapies

et al. 2017

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Cellular therapies, such as stem cell–based treatments, have been widely researched and numerous products and treatments have been developed. Despite this, there has been relatively limited use of these technologies in the healthcare sector. This study sought to investigate the perceived barriers to this more widespread adoption. An anonymous online questionnaire was developed, based on the findings of a pilot study. This was distributed to an audience of clinicians, researchers and commercial experts in 13 countries. The results were analysed for all respondents, and also sub-grouped by geographical region, and by profession of respondents. The results of the study showed that the most significant barrier was manufacturing, with other factors such as efficacy, regulation and cost-effectiveness being identified by the different groups. This study further demonstrates the need for these important issues to be addressed during the development of cellular therapies to enable more widespread adoption of these treatments.

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“…More globally, among the eight ATMPs authorised on the EU market to date, four are orphan drugs. As those cases show, orphan drugs and RM based products often share the two main features of high cost and uncertainties around evidence and value [39,40]. However, these same uncertainties are also seen in the weak long-term evidence for RMPs that are not orphan drugs.…”

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.

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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.

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Bringing Regenerative Medicines to the Clinic: The Future for Regulation and Reimbursement

Cited by 43 publications

References 29 publications

Liver tissue engineering: From implantable tissue to whole organ engineering

Liver tissue engineering: From implantable tissue to whole organ engineering

A quantitative, multi-national and multi-stakeholder assessment of barriers to the adoption of cell therapies

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

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