Regenerative medicine and its development and implementation: an analysis of emergent value systems and health service readiness
Lead Research Organisation:
University of York
Department Name: Sociology
Abstract
There has been considerable interest developing over recent years in regard to the therapies that have become and may become available based on what is known as 'regenerative medicine'. This is an important emerging field in clinical research, and one that has attracted considerable investment in the UK and internationally. The main areas of development include gene therapy medicinal products, cell therapy medicinal products and tissue engineered products. Its importance lies in its promise to transform healthcare by offering curative, not palliative, therapeutics through the regeneration of tissue within the body. This in itself may reshape healthcare provision though it is equally important to understand how the existing healthcare system will influence the pathways through which the field develops. In addition, the field poses new challenges for regulators because of the ways in which the 'mode of action' of tissue/cell therapies is quite different from the most common innovation the regulator has to understand and approve (or not), the new pharmaceutical drug. In short, the project seeks to understand the complex relationship between these clinical and wider social, organisational, regulatory and economic issues.
It does so through a series of Workpackages (WP). The first maps the field in the UK (within its wider international - principally EU/US context), especially in regard to current patterns of investment, clinical trials, product development, the intellectual property strategies of companies and the specific regulatory processes shaping these. The second WP examines the role of a range of intermediary agencies, such as the National Institute for Health and Clinical Excellence (NICE), patient groups and health insurance companies, in determining what can be called 'healthcare readiness' for the field, that is, how the field aligns with and can be embedded in existing practice and how far changes need to be made. The third WP involves detailed case studies of firms and hospital sites currently engaged in developing/using cell therapies chosen to reflect a) different regulatory classifications by product area b) the main areas of current activity by disease that are closely linked, and the sites that we would access to track product development. We will use a methodology called Analysis of Life Science Innovation Systems (ALSIS) modelling which captures different types of business models, value-chains and their relationship to the wider innovation environment. This allows us to examine the full range of activities required to bring a product from conception to market or to direct application in a hospital within the wider economic, regulatory, societal and political contexts that play a key role in determining the scale and scope of the innovation.
In the fourth WP the ALSIS method which allows us to map and explain innovation paths will be integrated with and triangulated against our modelling of the potential future of the field using horizon scanning techniques developed by one of the co-applicants based at the National Institute of Health Research Horizon Scanning Centre to interrogate likely diffusion paths within health care, and the expectations surrounding regenerative medicine among public and private actors (including government and private agencies).
Throughout the project we will work with collaborating parties, including the participating companies, hospitals, regulatory and reimbursement agencies (notably the Medicines and Healthcare products Regulatory Agency and NICE), patient groups, Research Councils UK, the Technology Strategy Board's Cell Therapy Catapult, the NIHR's i4i Secretariat, the Science and Technology Select Committee and the All-Party Parliamentary Group for Social Science and Policy.
It does so through a series of Workpackages (WP). The first maps the field in the UK (within its wider international - principally EU/US context), especially in regard to current patterns of investment, clinical trials, product development, the intellectual property strategies of companies and the specific regulatory processes shaping these. The second WP examines the role of a range of intermediary agencies, such as the National Institute for Health and Clinical Excellence (NICE), patient groups and health insurance companies, in determining what can be called 'healthcare readiness' for the field, that is, how the field aligns with and can be embedded in existing practice and how far changes need to be made. The third WP involves detailed case studies of firms and hospital sites currently engaged in developing/using cell therapies chosen to reflect a) different regulatory classifications by product area b) the main areas of current activity by disease that are closely linked, and the sites that we would access to track product development. We will use a methodology called Analysis of Life Science Innovation Systems (ALSIS) modelling which captures different types of business models, value-chains and their relationship to the wider innovation environment. This allows us to examine the full range of activities required to bring a product from conception to market or to direct application in a hospital within the wider economic, regulatory, societal and political contexts that play a key role in determining the scale and scope of the innovation.
In the fourth WP the ALSIS method which allows us to map and explain innovation paths will be integrated with and triangulated against our modelling of the potential future of the field using horizon scanning techniques developed by one of the co-applicants based at the National Institute of Health Research Horizon Scanning Centre to interrogate likely diffusion paths within health care, and the expectations surrounding regenerative medicine among public and private actors (including government and private agencies).
Throughout the project we will work with collaborating parties, including the participating companies, hospitals, regulatory and reimbursement agencies (notably the Medicines and Healthcare products Regulatory Agency and NICE), patient groups, Research Councils UK, the Technology Strategy Board's Cell Therapy Catapult, the NIHR's i4i Secretariat, the Science and Technology Select Committee and the All-Party Parliamentary Group for Social Science and Policy.
Planned Impact
The project will make an important contribution towards understanding the likely pathways that regenerative medicine takes within the UK and other health care systems, by identifying distinct commercial and non-commercial (e.g. hospital) routes to its application, including in particular cell therapies but in other domains too. It will also benefit regulatory and HTA agencies for their own formal regulatory models as well as more informal deliberations with those in the field seeking advice about regulation, product development and uptake within the NHS. Bio-clinical researchers participating in the regional stem cell and tissue engineering networks and patient groups keen to understand the likely availability of therapies will have a much clearer understanding of the trends ahead through the horizon-scanning element of the project. The research will clarify likely scenarios of adoption and diffusion for NHS policymakers and commissioners in different clinical sectors. Our case studies' descriptions and analysis will provide examples of RM development models and their challenges that will be of direct value to the Technology Strategy Board's Catapult Centre, to companies and public researchers developing their own innovation strategies. Patient groups likely to be most interested in the outcome of the research are those associated with the principal areas of inquiry where trialling and product development is underway, such as in musculoskeletal, stroke, and cardiovascular areas of research.
These outcomes of the research address the needs identified in the UK government's very recent review of its current investment in the life-sciences, most importantly to understand investment trends and the reimbursement landscape and to use social science to identify the value and likely market penetration of regenerative medical products. Moreover, our work on comparing hospital-based innovation via the 'exemption' or 'specials' route with the product-driven trials process addresses the government's desire to show how the NHS and commercial activities might be more effectively integrated. NICE is charged with the assessment of new products, requiring information about comparators, cost-consequence analysis, impact on patient pathways, and workflows in the NHS. The results of our work feed directly into these concerns and we will be able to help NICE, the new NHS Commissioning Board and the recently established Emerging Science and Bioethics Advisory Committee (ESBAC - Tait is leading ESBACs Regulatory Issues Working Group) develop new policy for the field, including informing the possible establishment of a national commissioning agency for RM.
The research also addresses a major issue identified by the Ministerial Industry Strategy Group (MISG) in a report that the applicants helped to prepare on the regulation and development of regenerative medicine, which has been passed to the House of Lords Inquiry due to report in 2013. There it is argued that social science research is needed to understand the 'institutional readiness' of the healthcare system, to complement the 'technological readiness' which the major science funding agencies are working to secure.
Finally, the research will contribute to debates at a European level about core problems of the RM market that have been raised in the European Medicines Agency and the European Parliament and which need to be addressed over the next few years as products come to regulators for market approval: these include the form that clinical trials and postmarket surveillance will take, clarification of the use of the hospital exemption, orphan medicine and other routes to therapy to ensure an appropriate balance is struck between commercial incentivisation and non-commercial development, and the pattern of reimbursement regimes and whether new models are needed to foster the development of the field.
These outcomes of the research address the needs identified in the UK government's very recent review of its current investment in the life-sciences, most importantly to understand investment trends and the reimbursement landscape and to use social science to identify the value and likely market penetration of regenerative medical products. Moreover, our work on comparing hospital-based innovation via the 'exemption' or 'specials' route with the product-driven trials process addresses the government's desire to show how the NHS and commercial activities might be more effectively integrated. NICE is charged with the assessment of new products, requiring information about comparators, cost-consequence analysis, impact on patient pathways, and workflows in the NHS. The results of our work feed directly into these concerns and we will be able to help NICE, the new NHS Commissioning Board and the recently established Emerging Science and Bioethics Advisory Committee (ESBAC - Tait is leading ESBACs Regulatory Issues Working Group) develop new policy for the field, including informing the possible establishment of a national commissioning agency for RM.
The research also addresses a major issue identified by the Ministerial Industry Strategy Group (MISG) in a report that the applicants helped to prepare on the regulation and development of regenerative medicine, which has been passed to the House of Lords Inquiry due to report in 2013. There it is argued that social science research is needed to understand the 'institutional readiness' of the healthcare system, to complement the 'technological readiness' which the major science funding agencies are working to secure.
Finally, the research will contribute to debates at a European level about core problems of the RM market that have been raised in the European Medicines Agency and the European Parliament and which need to be addressed over the next few years as products come to regulators for market approval: these include the form that clinical trials and postmarket surveillance will take, clarification of the use of the hospital exemption, orphan medicine and other routes to therapy to ensure an appropriate balance is struck between commercial incentivisation and non-commercial development, and the pattern of reimbursement regimes and whether new models are needed to foster the development of the field.
Publications
Ali RR
(2014)
Regulating cell-based regenerative medicine: the challenges ahead.
in Regenerative medicine
Andrew Webster
(2017)
Regenerative medicine and responsible research and innovation: proposals for a responsible acceleration to the clinic
in Regenerative Medicine
Aurelie Mahalatchimy
(2018)
Innovative Drugs
Banda G
(2018)
Evolution of Business Models in Regenerative Medicine: Effects of a Disruptive Innovation on the Innovation Ecosystem.
in Clinical therapeutics
Banda G
(2019)
Understanding the emergence and evolution of new business models in the UK regenerative medicine sector
in Technology Analysis & Strategic Management
Banda G
(2019)
Regenerative medicine as a disruptive technology: implications for manufacturing & clinical adoption
in Cell and Gene Therapy Insights
Corbett MS
(2017)
Innovative regenerative medicines in the EU: a better future in evidence?
in BMC medicine
Duggal S
(2020)
Promissory and protective imaginaries of regenerative medicine: Expectations work and scenario maintenance of disease research charities in the United Kingdom.
in Public understanding of science (Bristol, England)
Faulkner A
(2018)
The politics of valuation and payment for regenerative medicine products in the UK
in New Genetics and Society
Faulkner A
(2018)
The politics of valuation and payment for regenerative medicine products in the UK
in New Genetics and Society
Description | • there are specific innovation challenges of a technical, regulatory and manufacturing nature with regard to deriving, stabilising, classifying and scaling-up live (cell) tissue for clinical purposes, not found in other areas of biomedical innovation: this has led to the creation of specific innovation niches to foster RM development • through the detailed case studies and wider fieldwork we have identified five paradigmatic routes to the clinic being pursued by clinical and commercial actors and mapped these onto the leading firms in the field within the UK and elsewhere: the leading activity relates to what is known as CAR T cell therapy • clinical therapies are and will be shaped by the interplay of 'technology identities, adoption and development spaces' that generate diverse contexts within which implementation is more, or less, likely: these can help model the actual and prospective take-up of therapies in the future • RM will require a range of novel or redistributed manufacturing micro-factories close to clinics to be viable • as a result, scenarios need to be developed for the likely size and profiles of clinical populations treatable through different manufacturing modalities and scales to support national planning in the NHS Further details on the findings and additional results when the project is fully complete later in 2017 will be uploaded to Research Fish |
Exploitation Route | In terms of being directly involved in steering decision-making in regard to the assessment and strategic path best suited to the field of regenerative medicine, Webster was appointed (as the only social scientist) to serve on the DoH/MRC Regenerative Medicine Expert Group's sub-committee on the governance and regulation of RM and its subsequent Advisory Group in regard to the National Institute for Health and Care Excellence's (NICE) assessment and recommendations relating to the technology appraisal methodology for RM. The project has helped to shape the NHSE's approach to innovation 'spread'. This is especially so for members of the NHSE's Specialised Commissioning Service in helping them to understand the pathways to adoption of new biomedical technologies and envisaged routes for RM that go beyond the currently limited framework of 'specialised' treatment to how more extensive therapy might be possible. More widely, Webster was asked to establish a new 'specialist interest group' in the RM field for the NHS's CHAIN (Contact, Help, Advice and Information Network) network (established some 20 years ago), which has over 15000 members working in the NHS across the UK. This has allowed members to secure specific advice and information from the research to help shape their local needs. For example, CHAIN members from the Community Health Exchange in Scotland have been advised on the possible RM developments relating to long-term conditions and the likely clinical routes to therapy ahead. The project has also informed government thinking through its contribution to the House of Commons Select Committee (data drawn on) and collaborated with the MHRA and its Patient Forum (note: see documents that will be available mid Jan 2016 for both of these) to run the first MHRA/social science event (described by the host - Mike Dykes - as 'pioneering') that examined the implications of RM for patient groups and regulatory affairs. The key issues raised and which will form the basis for the Patient Forum's activity in the area were: understanding pathways to the clinic; the impact of geography and regional differences in regard to the availability and access of RM therapies; and the need to consider carefully the range and level of risk that patients are prepared to take (especially in treatments of the last resort). Webster also organised an All Party Parliamentary Group on Health (House of Commons, November 8, 2016) meeting to discuss RM which led to a Report for Group members and a series of questions for its MPs to follow-up. Moreover, the House of Commons Science and Technology Select Committee invited questions from the project that were then put during an oral evidence session on December 7th with senior staff from the NHS, and other parties. The Chair of the Select Committee - Stephen Metcalfe - invited Webster to a personal meeting to discuss the issues raised. A number of UK Patient Groups have worked with the project in late 2016 order to help shape their forward plans and possibilities in relation to the role of RM for their patient groups: these include ScarFree and Parkinson's UK. The latter, for example, have developed a joint-proposal with SATSU to explore the role of patient-centred/led innovation; the implications this has for pathways to the clinic; and the production, marshalling and integration of diverse forms of patient data. The Association of the British Pharmaceutical Industry's (ABPI) invited Webster (in 2017) to contribute towards its work with industry on manufacturing innovation, especially around MMIP (the Medicines Manufacturing Industry Partnership) and the specific place of RM within this.Finally, the conceptual model that we developed during the project - the notion of 'institutional readiness' for innovative therapies - has been adopted and used by major research groups in the regenerative medicine field. It helped to underpin, for example, a successful application by Newcastle University and other academic and business partners for a major Innovate UK funded new 'Advanced Therapy Treatment Centre' to be developed during 2018 |
Sectors | Healthcare |
URL | http://www.york.ac.uk/satsu/current-projects/regenablemed/ |
Description | Through engagement with UK and international policy agencies the REGenableMED project has been directly involved in steering decision-making in regard to the assessment and strategic path best suited to the field of regenerative medicine (RM). The project has contributed to NHS England's, NICE's and the HoC Select Committee on the adoption of advanced therapies, established a new learning/information resource on RM to diverse users in the UK health research/care system, and contributed towards the ABPI's Medicines Manufacturing Industry Partnership . At the EU level, the project has contributed to policy within the EC and EMA through formal consultation, and its results have been adopted by the OECD informing its future programme on 'responsible research and innovation'. The research led to being commissioned by the Cell and Gene Therapy Catapult to undertake a review of risk-sharing agreements for novel medicines, and to act as a member of the government's 2017 institutional review of the Catapult itself. In more detail, In terms of being directly involved in steering decision-making in regard to the assessment and strategic path best suited to the field of regenerative medicine, Webster was appointed (as the only social scientist) to serve on the DoH/MRC Regenerative Medicine Expert Group's sub-committee on the governance and regulation of RM and its subsequent Advisory Group in regard to the National Institute for Health and Care Excellence's (NICE) assessment and recommendations relating to the technology appraisal methodology for RM. The project has helped to shape the NHSE's approach to innovation 'spread'. This is especially so for members of the NHSE's Specialised Commissioning Service in helping them to understand the pathways to adoption of new biomedical technologies and envisaged routes for RM that go beyond the currently limited framework of 'specialised' treatment to how more extensive therapy might be possible. More widely, Webster was asked to establish a new 'specialist interest group' in the RM field for the NHS's CHAIN (Contact, Help, Advice and Information Network) network (established some 20 years ago), which has over 15000 members working in the NHS across the UK. This has allowed members to secure specific advice and information from the research to help shape their local needs. For example, CHAIN members from the Community Health Exchange in Scotland have been advised on the possible RM developments relating to long-term conditions and the likely clinical routes to therapy ahead. The project has also informed government thinking and recommendations made by the House of Commons Science and Technology Select Committee of Inquiry on RM (May 2017) and collaborated with the MHRA and its Patient Forum (note: see documents that will be available mid Jan 2016 for both of these) to run the first MHRA/social science event (described by the host - Mike Dykes - as 'pioneering') that examined the implications of RM for patient groups and regulatory affairs. The key issues raised and which will form the basis for the Patient Forum's activity in the area were: understanding pathways to the clinic; the impact of geography and regional differences in regard to the availability and access of RM therapies; and the need to consider carefully the range and level of risk that patients are prepared to take (especially in treatments of the last resort). Webster also organised an All Party Parliamentary Group on Health (House of Commons, November 8, 2016) meeting to discuss RM which led to a Report for Group members and a series of questions for its MPs to follow-up. Moreover, the House of Commons Science and Technology Select Committee invited questions from the project that were then put during an oral evidence session on December 7th with senior staff from the NHS, and other parties. The Chair of the Select Committee - Stephen Metcalfe - invited Webster to a personal meeting to discuss the issues raised. A number of UK Patient Groups have worked with the project in late 2016 order to help shape their forward plans and possibilities in relation to the role of RM for their patient groups: these include ScarFree and Parkinson's UK. The latter, for example, have developed a joint-proposal with SATSU to explore the role of patient-centred/led innovation; the implications this has for pathways to the clinic; and the production, marshalling and integration of diverse forms of patient data. Webster attended an invitation-only meeting with Innovate UK to identify the priorities for RM within the government's 2017 'Industrial Strategy', and was the only social scientists member of an expert panel to evaluate the Cell and Gene Therapy Catapult in 2017. The Association of the British Pharmaceutical Industry's (ABPI) invited Webster (in 2017) to contribute towards its work with industry on manufacturing innovation, especially around MMIP (the Medicines Manufacturing Industry Partnership) and the specific place of RM within this. The model of 'institutional readiness' (IR) which was developed through the project analysis of clinical adoption was deployed by two science-led proposals for funding in December 2018 to Innovate-UK's Call to establish three new Advanced Therapy Treatment Centres for cell and gene therapy. One of the successful bids, from a Northeast consortium deployed the IR concept in shaping their proposal. The IR model will therefore be deployed in the Newcastle-led centre over the coming three years. The Director of the new Centre, Professor Michael Whitaker, has said that the IR model will be 'used as a benchmark' to assess the centres progress towards developing clinical adoption strategies. Webster has been invited to assist the new consortium in operationalising the model there. Internationally At the EU level, we have made ongoing contributions to draft documentation prepared by the EMA/EC. These relate to regulatory proposals on regen medicine. The OECD in Paris has liaised directly with Webster and drawn extensively on the research in order to incorporate RM into its future activities across its members/delegates. This has led to an agreement to build work around RM via an OECD Workshop to be held in Berlin in July 2017 that will examine the societal implications and regulatory issues of RM, and how open science and open innovation help the realisation of regenerative medicine. A series of tightly-written Policy Briefings have been produced throughout the duration of the research and distributed to government, NHS, Patient and Industrial stakeholders in the UK and Europe. These have been published on diverse websites including EuroStemCell, the primary resource site on RM for over 3000 EU-based scientists, patient groups, clinicians and policymakers. |
First Year Of Impact | 2017 |
Sector | Healthcare,Pharmaceuticals and Medical Biotechnology |
Impact Types | Policy & public services |
Description | Adoption of the 'institutional readiness' model in the NHS |
Geographic Reach | National |
Policy Influence Type | Influenced training of practitioners or researchers |
Impact | The ESRC REGenableMED project 2014-17 has been directly involved in steering decision-making and practice in the field of regenerative medicine (RM). The project developed a science and technology studies model of 'institutional readiness' to provide a new framework through which complex advanced therapies in this field might be more readily adopted in clinical settings. A new Advanced Therapy Treatment Centre based in the north east (the NAATTC) funded by Innovate-UK in 2018 adopted the model. It is now used to 'benchmark' the success of the Centre, whose partners are drawn from clinical, academic, business support and product development firms. |
URL | https://www.theattcnetwork.co.uk/centres/northern-alliance |
Description | Broadening the use of the Institutional Readiness model across bioclincal research centres in three UK regions |
Geographic Reach | Local/Municipal/Regional |
Policy Influence Type | Influenced training of practitioners or researchers |
Impact | The model of 'institutional readiness' (IR) was deployed by bioscientists and partners when submitting their bid for £10m in 2018 to fund a new research and clinical centre headquartered in Newcastle, the 'Northern Alliance Advanced Therapy Treatment Centre' (NAATTC). Since the end of January 20189, it has been decided to roll out the model across the two other Advanced Therapy Treatment Centres based in the Northwest (iMatch) and the Midlands/Wales. Senior staff at Innovate-UK who coordinate the centres have noted the importance of the concept to them and to industry involved in developing new products. The IR model helps to identify the institutional capacities needed to enable effective adoption of complex advanced (cell-based) therapies. The model helps improvements therefore in clinical service delivery. |
Description | Data and results used directly by House of Commons Inquiry into Regenerative Medicine |
Geographic Reach | National |
Policy Influence Type | Citation in other policy documents |
URL | https://publications.parliament.uk/pa/cm201617/cmselect/cmsctech/275/27502.htm |
Description | Institutional Readiness |
Geographic Reach | National |
Policy Influence Type | Membership of a guideline committee |
Impact | The model of Institutional Readiness which was developed during the REGenableMED project has continued to make headway being adopted by the four hospital/commercial partnerships that make up the Northern Alliance Advanced Therapy Treatment Centre. The current organisation of clinics is not conducive to the therapeutic delivery of the new advanced cell and gene technologies of regenerative medicine (RM) developed in the laboratory setting. This poses a risk to the momentum in developing pathways to commercial and clinical success. From his research, Webster has developed a model of 'institutional readiness' (IR model) to address this problem. The model has been operationalised as a tool now being used to identify the resources and capacities needed to deliver advanced therapies. It was initially adopted in 2018 by the Northern Alliance Advanced Therapies National Treatment Centre (NA-ATTC), one of three Centres funded by Innovate-UK, with a remit to develop RM therapies and to act as a potential model for roll-out to other NHS sites. In June 2020, the model was adopted by the pan-Advanced Therapy Treatment Centres (pan- ATTCs)(Manchester, Midlands, and Wales), and it will be rolled-out in 2021 to the other NHS sites where RM is being introduced. |
Description | Policy Briefings |
Geographic Reach | National |
Policy Influence Type | Contribution to a national consultation/review |
Impact | Through engagement with UK and international policy agencies the REGenableMED project has been directly involved in steering decision-making in regard to the assessment and strategic path best suited to the field of regenerative medicine (RM). The project has contributed to NHS England's and NICE strategy in regard to steering policy on the adoption of innovative biomedical technologies, established a new learning/information resource on RM to diverse users in the UK health research/care system, and contributed towards the ABPI's Medicines Manufacturing Industry Partnership . At the EU level, the project has contributed to policy within the EC and EMA through formal consultation, and its results have been adopted by the OECD informing its future programme on 'responsible research and innovation'. |
Description | ESRC Responsive Mode |
Amount | £750,000 (GBP) |
Funding ID | ES/P002943/1 |
Organisation | Economic and Social Research Council |
Sector | Public |
Country | United Kingdom |
Start | 03/2017 |
End | 03/2020 |
Title | Regenerative medicine: a database of 700 public and private organisations working in the field |
Description | This is an ACCESS Database held securely at the University of York's Science and Technology Studies Unit, leading on the project. It is a major and unique database of firms, university sites, hospitals and related agencies and the products and clinical therapies available or under development. It will be made public in the form of responding to all requests made by external bodies for PDF-formatted reports on specific aspects of the data, when the project is fully completed later in 2017. |
Type Of Material | Database/Collection of data |
Provided To Others? | No |
Impact | The material will be of especial value to companies and policy-makers keen to understand the trends in the field. |
Description | Collaboration with the NAATTC Advanced Therapy Treatment Centr |
Organisation | Newcastle upon Tyne Hospitals NHS Foundation Trust |
Country | United Kingdom |
Sector | Academic/University |
PI Contribution | The NAATTC made of four partners from NHS Trusts in Edinburgh, Glasgow, Leeds and Newcastle adopted the model of institutional readiness from the REGenableMED project to serve as a tool in their four hospitals to help develop appropriate pathways, resources and skills from the lab to the clinic. The Centre is funded by I-UK to the tune of c£10m over three years. |
Collaborator Contribution | The members of the NAATTC have, in collaboration with Professor Andrew Webster developed an operational model for the tool and are deploying it throughout the centres during the years 2018-2020. |
Impact | The tool has been deployed and results for 2018/9 are being compared with 2019/20. There will be at least three papers published. It has been proposed that the model could be rolled out across other NHS centres dealing with advanced cell therapies. |
Start Year | 2018 |
Description | Digital Version of the full research findings for REGenableMED - published online as 'REGenableMED Special Report'. |
Form Of Engagement Activity | Engagement focused website, blog or social media channel |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Public/other audiences |
Results and Impact | The project developed and designed, in collaboration with EuroStemCell, a bespoke digital version of the project results and resources (including presentations and briefings from the final conference) for a wide, general audience with a potential readership of many thousands via the EuroStemCell international readership. |
Year(s) Of Engagement Activity | 2017 |
URL | http://www.eurostemcell.org/regenerative-medicine-special-report |
Description | Exploring new frontiers of regenerative medicine, Edinburgh, November 2014 |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | Yes |
Geographic Reach | Regional |
Primary Audience | Schools |
Results and Impact | Joyce Tait (Co-I), Innogen and colleagues worked with the Law School in Edinburgh to produce a film on the socio-legal aspects of stem cells research as part of the Festival of Social Science The film led to a wide ranging debate among participants especially schoolchildren |
Year(s) Of Engagement Activity | 2014 |
Description | Joint REGenableMED/MHRA (regulatory authority) Workshop for Patient Groups |
Form Of Engagement Activity | Participation in an activity, workshop or similar |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Patients, carers and/or patient groups |
Results and Impact | This joint presentation provided a brief summary of the current state of play in the Regenerative Medicine field and shows where there have been early developments and why; what the potential might be. Aims for the day: - to explore what treatments are available or likely to figure in the future in the field of regenerative medicine - to learn from the patient group delegates about their engagement with, interests in and possible concerns and questions about regenerative medicine - to see both of the above in tandem with the MHRA's role in regulating new therapies. The main issues raised were as follows: there was an important theme relating to how we can understand pathways to the clinic. The discussion identified three discrete pathways that were of especial importance: the first which emphasises innovation (cf 'the 'innovation cycle') and complementary business models; a second which focuses on the 'rare-disease' model for getting products through to patients (via specialised commissioning); and a third, much less developed approach that is emerging among the larger, more highly resourced charities (such as Parkinsons UK), which was described as 'critical path analysis', where the charities go beyond simply supporting researchers elsewhere and undertake strategic review of clinical trials design, how medicines might be repurposed and so on. It would be important to see how these three relate to each other, and how they map onto discrete regulatory oversight - the impact of geography and regional differences in regard to the availability and access of RM therapies was a second theme that emerged during the discussion. It was argued that it is extremely important to avoid the contingencies of 'postcode' medicine, which might be unintentionally exacerbated by the move towards devolved government and so differing priorities and resourcing for new therapies. In regard to the regional dimension, there is considerable interest at present in preparing the ground for new RM Centres of Excellence and Delivery across the UK. These could be supported through national commissioning but would need to have available an appropriate skills base and infrastructure for handling the movement of cells from localised micro-factories to/in local hospitals. - a third issue related to the need to consider carefully the range and level of risk that patients are prepared to take (especially in treatments of the last resort). More broadly, the role of PPI in clinical trials should help to address this as well as help reflect on the appropriate ethical and consent framework for new therapies, such as RM. It is important to ensure that the clinical trials model is fit for purpose for RM therapies, and to consider whether there is a need to develop a new authorised trials model for RM at the national level. Issues related to the terms of inclusion criteria, and the burden of participation for patients was raised, as was the need to simplify trial protocols where possible. |
Year(s) Of Engagement Activity | 2016 |
URL | http://www.eurostemcell.org/regenerative-medicine-special-report |
Description | Membership of the Committee on Advanced Therapies, European Medicines Agency |
Form Of Engagement Activity | Participation in an activity, workshop or similar |
Part Of Official Scheme? | Yes |
Geographic Reach | International |
Primary Audience | Policymakers/politicians |
Results and Impact | The engagement is yet to be fully realised and this entry on ResearchFish will be updated over the coming months See above |
Year(s) Of Engagement Activity | 2014 |
Description | Membership of the DoH Regenerative Medicine Expert Group |
Form Of Engagement Activity | A formal working group, expert panel or dialogue |
Part Of Official Scheme? | Yes |
Geographic Reach | National |
Primary Audience | Policymakers/politicians |
Results and Impact | The RMEG has recommended the establishment of a senior strategy group which the Minister is likely to support. It is envsiaged that Webster and Faulkner will play a continuing role here. Helped to shape the debate surrounding proposals to develop policy in the field |
Year(s) Of Engagement Activity | 2014 |
Description | REGenableMED closing conference |
Form Of Engagement Activity | Participation in an activity, workshop or similar |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Policymakers/politicians |
Results and Impact | The Conference reported the results and insights of the ESRC-funded REGenableMED project. Complemented by two panels - on international developments and on future strategy - a series of strategic issues were identified. The contribution from the delegates was especially important in helping to define these issues more precisely. The summary below is of direct relevance to current government policy and the recent Select Committee report. The principal ideas that should shape future planning are: A novel innovation infrastructure for RM: Providing the right sort of organisational and scientific infrastructure for RM is vital. There needs to be a paradigm-shift away from a traditional approach to product development to one that focuses on a more co-ordinated value-chain, on targeted (rather than broad) population groups, patient access and on a broader range of evidentiary sources to track and assess emergent therapies. 'Transformative' products are in fact transformative processes in the RM field, which then need a transformative framework to support them. As part of this process, further thought should be given to establishing a formal RM network at the national level to help coordinate infrastructural development building on recent policy. Moreover, this should foster better professional networking/communities of practice in the wider NHS, especially in regard to encouraging developmental learning within clinical practice. The envisaged Advanced Therapy Treatment Centres (see below) would operate much more effectively in such an environment. Advanced Therapy Treatment Centres - building effective institutional and business models: The proposed ATTCs to be supported by Innovate UK are in effect 'pilots' of central importance to the development of the field (in the language of the 'trial', they are organisationally at 'Phase 1' in the process). The centres need to reflect an institutional readiness for RM therapies, specific clinical competences, a role for relevant patient associations in providing patient input, and an alignment between the new ATTCs and existing rare-disease treatment centres. There is a role for the RM Clinical Reference Group informed by the social science from the project in regard to defining the scope and specification for possible centres. Centres need to ensure that they create additional knowledge and skills and leverage existing infrastructure (such as the NHSBT/ SNBTS and the NIHR Biomedical Research Centres), while being geographically accessible within the UK. Five paradigmatic pathways to innovation have been identified in the project, which in turn have distinctive clinical adoption parameters. These need to be linked to the six key and distinctive business models that have also been identified through the project's detailed analysis of SMEs in the field. Integration of clinical databases: Relevant biobanks, clinical registers and databases need to be more strongly integrated, especially in embracing real world data systems (i.e. 'off-trial' data); standardising data across these different sources should become a priority, and are key to moves towards proposed risk-sharing schemes (or more formally outcomes-based managed entry agreements). Reimbursement and regulatory models: There are a number of reimbursement and regulatory models that do not necessarily map on to the specific market niche for RM. Risk-sharing commissioning schemes will also need coordinated data-collection infrastructures to support their iterative approach, and to resolve the tension between a 'values' and a 'needs' driven reimbursement and regulatory perspective. An annuity cap model is especially worth exploring within this context to help manage problems of affordability in the health care system, and foster innovation, within the recently adopted net budget impact test (that innovative therapies do not exceed an overall budgetary impact of £20m over three years). Understanding international developments: There are diverse regulatory models internationally. The conference discussed presentations related to recent developments in Europe, China and Japan. Developments relating to iPSCs in Europe have had a significant impact in the entry of major pharmaceutical companies to the area while in China it is clear that there are tensions between national regulation and regional regulatory 'bionetworks'. In Japan, the relatively recent (2014) regulatory changes to foster early access for patients reflects Japan's desire to play a leading role internationally in the RM field. Further information can be found at the project website, especially in regard to the 4 Policy Briefings that are available: https://www.york.ac.uk/satsu/current-projects/regenablemed/newsandbriefings/#tab-5 |
Year(s) Of Engagement Activity | 2017 |
URL | http://www.eurostemcell.org/regenerative-medicine-special-report |
Description | Regenerating the clinic |
Form Of Engagement Activity | A magazine, newsletter or online publication |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Policymakers/politicians |
Results and Impact | This was a piece for the ESRC's Society Now Magazine which reported some key aspects of the REGenableMED project |
Year(s) Of Engagement Activity | 2017 |
URL | http://www.esrc.ac.uk/files/news-events-and-publications/publications/magazines/society-now/society-... |
Description | Web based summary of all findings from the project, in collaboration with EUROSTEMCELL |
Form Of Engagement Activity | Engagement focused website, blog or social media channel |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Professional Practitioners |
Results and Impact | Working with the EU-funded engagement project - EuroStemCell - we prepared a detailed and interactive summary of all findings, papers and related resources from the REGenableMED project |
Year(s) Of Engagement Activity | 2017,2018 |
URL | https://www.eurostemcell.org/regenerative-medicine-special-report |