Special Report
Regenerative medicine, resource and regulation: lessons learned from the remedi project
Published Online:11 Mar 2011https://doi.org/10.2217/rme.10.89
Abstract
The successful commercialization of regenerative medicine products provides a unique challenge to the manufacturer owing to a lack of suitable investment/business models and a constantly evolving regulatory framework. The resultant slow translation of scientific discovery into safe and clinically efficacious therapies is preventing many potential products from reaching the market. This is despite of the need for new therapies that may reduce the burden on the world’s healthcare systems and address the desperate need for replacement tissues and organs. The collaborative Engineering and Physical Sciences Research Council (EPSRC)-funded remedi project was devised to take a holistic but manufacturing-led approach to the challenge of translational regenerative medicine in the UK. Through strategic collaborations and discussions with industry and other academic partners, many of the positive and negative issues surrounding business and regulatory success have been documented to provide a remedi-led perspective on the management of risk in business and the elucidation of the regulatory pathways, and how the two are inherently linked. This article represents the findings from these discussions with key stakeholders and the research into best business and regulatory practices.
Keywords:
Papers of special note have been highlighted as: ▪ of interest ▪▪ of considerable interest
Bibliography
- 1 Prescott C: The promise of stem cells: a venture capital perspective. In: Advances in Tissue Engineering. Polak J, Mantalaris S, Harding S (Eds). World Scientific Publishing Company, London, UK, 491–500 (2008).▪▪ Provides useful information on the mechanisms of venture capital investment as well as a venture capitalists’ perspective on the potential for stem cell therapies.
- 2 Burger SR: Current regulatory issues in cell and tissue therapy. Cytotherapy5(4),289–298 (2003).
- 3 Preti RA: Bringing safe and effective cell therapies to the bedside. Nat. Biotech.23(7),801–804 (2005).
- 4 Halme DG, Kessler DA: FDA regulation of stem-cell-based therapies. N. Engl. J. Med355(16),1730–1735 (2006).
- 5 European Union: Regulation (EC) No 1394/2007 of the European Parliament and of the Council of 13 November 2007 on advanced therapy medicinal products and amending Directive 2001/83/EC and Regulation (EC) No 726/2004. L324/121–L324/137 (2007).
- 6 Christian M, Martinson M: Getting paid for all your hard work: the basics of reimbursement for healthcare products and services. Regulatory Affairs Focus5–10 (2002).
- 7 Sood N, Buntin MB, Escarce JJ: Does how much and how you pay matter? Evidence from the inpatient rehabilitation care prospective payment system. J. Health Econ.27(4),1046–1059 (2008).
- 8 Gajewski JL, Simmons A, Weinstein R et al.: The new apheresis and blood and marrow transplantation-related current procedural terminology codes for payment of apheresis and blood and marrow transplantation services. Biol. Blood Marrow Transpl.11(11),871–880 (2005).
- 9 Pearson S, Rawlins M: Quality, innovation, and value for money: NICE and the British National Health Service. JAMA294(20),2618–2622 (2005).
- 10 Huckman RS, Pisano GP: Turf battles in coronary revascularization. N. Engl. J. Med352(9),857–859 (2005).
- 11 Wilson CB: Adoption of new surgical technology. BMJ332(7533),112–114 (2006).
- 12 Bouchie A: Tissue engineering firms go under. Nat. Biotech.20(12),1178–1179 (2002).
- 13 Lysaght M, Hazlehurst A: Tissue engineering: the end of the beginning. Tissue Eng.10(1/2),309–320 (2004).
- 14 Rayment EA, Williams DJ: Mind the gap: challenges in characterizing and quantifying cell- and tissue-based therapies for clinical translation. Stem Cells28(5),996–1004 (2010).
- 15 Ginty PJ, Singh PB, Smith D, Hourd P, Williams DJ: Achieving reimbursement for regenerative medicine products in the USA. Regen. Med.5(3),463–469 (2010).▪ Seeks to simplify the mechanisms of reimbursement in the USA and is one of very few publications to focus on what is a very poorly understood but very important aspect of the commercialization of regenerative medicine products.
- 16 Kerr DJ, Scott M: British lessons on healthcare reform. N. Engl. J. Med.361(13),e21 (2009).
- 101 Martin P, Hawksley R, Turner A: The commercial development of cell therapy – lessons for the future? Survey of the cell therapy industry and the main products in use and development Link▪ Provides an interesting insight into the thoughts and opinions of those within the regenerative medicine industry.
- 102 Department of Human Health and Services: A new vision, a future for regenerative medicine www.hhs.gov/reference/newfuture.shtml (Accessed May 2009)
- 103 Tengion Inc. Press Releases: Tengion Inc. completes $50 million series “B” round financing led by Bain Capital and Quaker BioVentures will support neo-bladder clinical trials, manufacturing capacity and pipeline development www.tengion.com/news/press/20060623.cfm (Accessed June 2009)
- 104 Feurstein A: Geron’s stem-cell research hype soaks investors. Genetics and Societywww.geneticsandsociety.org/article.php?id=4533 (Accessed May 2010)
- 105 Perrin NMR: The global commercialisation of stem cell research in the UK: how can the UK’s strengths in stem cell research be translated into commercial opportunities in a global market? www.chrismason.com/industry_library/assets/UKTI%20Stem%20Cell%20Res.pdf (Accessed April 2010)
- 106 remedi: aims and objectives www.lboro.ac.uk/departments/mm/research/healthcare/remedigc/aims-objectives.html (Accessed January 2010)
- 107 Intercytex Press Releases: Proposed placing of 23,076,924 shares at 52 pence per share raising £12 million www.intercytex.com/icx/news/releases/2007/2007–2005–03/index.html (Accessed May 2009)
- 108 US FDA: Guidance, compliance and regulatory information. Draft guidance for industry: current good tissue practice (CGTP) and additional requirements for manufacturers of human cells, tissues, and cellular and tissue-based products (HCT/Ps) www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/Tissue/ucm062693.htm (Accessed January 2009)
- 109 US FDA: Guidance for industry; CGMP for Phase I investigational drugs www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm070273.pdf (Accessed August 2008)
- 110 European Agency for the Evaluation of Medicinal Products: Questions and answers on the regulation of advanced therapy medicinal products www.cbg-meb.nl/NR/rdonlyres/553119CE-917D-4956-AA16-EBC410AC7426/0/CATQA.pdf
- 111 EU Heads of Medicines Agencies CTFG Activity Report, 2008–2009 www.hma.eu/uploads/media/CTFG_activity_report_2008–2009.pdf (Accessed July 2010)
- 112 Centers for Medicare and Medicaid Services www.cms.hhs.gov (Accessed July 2010)
- 113 National Institute for Clinical Excellence: Guide to the methods of technology appraisal www.nice.org.uk/niceMedia/pdf/TAP_Methods.pdf
- 114 House of Commons Science and Technology Committee: Bioengineering–Seventh Report of Session 2009–2010 www.publications.parliament.uk/pa/cm200910/cmselect/cmsctech/220/22002.htm
- 115 Geron Inc. News Releases: Geron’s IND for spinal cord injury placed on hold www.geron.com/media/pressview.aspx?id=1187 (Accessed May 2010)
- 116 Geron Inc. News Releases: Geron receives FDA clearance to begin world’s first human clinical trial of embryonic stem cell-based therapy www.geron.com/media/pressview.aspx?id=1148 (Accessed February 2009)
- 117 San Jose Mercury News: Human tests of Geron’s spinal-cord medication put on hold www.stemcellclinic.com/en/pr/item/700.html (Accessed May 2010)
- 118 Proinvestors UK: ReNeuron Group says clinical trial on hold www.proactiveinvestors.co.uk/companies/news/754/reneuron-group-says-clinical-tril-on-hold-0754.html (Accessed May 2010)
- 119 European Medicines Agency Press Release: The EU and the FDA working together to create a Common Application for Orphan Designation for Medicines www.ema.europa.eu/pdfs/general/direct/pr/55739107en.pdf (Accessed May 2010)
- 120 UK National Stem Cell Network Bulletin: Technology Strategy Board Regenerative Medicine Programme www.uknscn.org/events/interim_ebulletin_2_sept2009.pdf (Accessed May 2010)
- 121 Tigenix. Press Releases: Tigenix receives positive CHMP opinion on European MAA for ChondroCelect Link (Accessed March 2010)
