Abstract
Aim: The development and introduction of cell and gene therapies presents complex social and economic issues. Fully addressing these challenges requires engagement with patients and the public. Materials & methods: A systematically conducted scoping review was undertaken to gauge current patient and public knowledge and perspectives, and as such inform requirements for future research, education and engagement activities. Results: A heterogeneous collection of 35 studies were identified. Levels of knowledge among patients and the public were extremely variable. Studies indicated general acceptance of therapies. Conclusion: The review identified the need for tailored educational activities, and in particular the importance of addressing misconceptions. There is also a need for robust qualitative research considering perspectives on current and forthcoming licensed therapies.
Papers of special note have been highlighted as: • of interest; •• of considerable interest
References
- 1. 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. European Parliment. Strasbourg, France. (2007).
- 2. Directive 2001/83/EC of the european parliament and of the council of 6 November 2001 on the community code relating to medicinal products for human use. European Parliment. Strasbourg, France (2001).
- 3. . Efficacy and safety of chimeric antigen receptor-T cells in the treatment of B cell lymphoma: a systematic review and meta-analysis. Chin. Med. J. (Engl.) 133(1), 74–85 (2020).
- 4. . Stem cell therapy in heart diseases – cell types, mechanisms and improvement strategies. Cell Physiol. Biochem. 48(6), 2607–2655 (2018).
- 5. . Mesenchymal stromal cell-based therapy: regulatory and translational aspects in gastroenterology. World J. Gastroenterol. 22(41), 9057–9068 (2016).
- 6. . Stem cell therapy for neurological disorders. S. Afr. Med. J. 109(8b), 70–77 (2019).
- 7. Gene therapy in retinal dystrophies. Int. J. Mol. Sci. 20(22), 5722 (2019).
- 8. . Delivering cellular and gene therapies to patients: solutions for realizing the potential of the next generation of medicine. Gene Ther. 27, 537–544 (2019). •• Provides a helpful introduction on the topic of cell and gene therapies and some of them challenges for enabling patients to benefit from these.
- 9. . Systematic review or scoping review? Guidance for authors when choosing between a systematic or scoping review approach. BMC Med. Res. Methodol. 18(1), 143 (2018). • A helpful clarification of the differences between systematic and scoping review approaches and the role of each of these.
- 10. . A systematic review of economic evaluations of advanced therapy medicinal products. Br. J. Clin. Pharmacol. 87(6), 2428–2443 (2020).
- 11. The PRISMA statement for reporting systematic reviews and meta-analyses of studies that evaluate healthcare interventions: explanation and elaboration. BMJ 339, b2700 (2009).
- 12. Fantasies about stem cell therapy in chronic ischemic stroke patients. Stem Cells Dev. 22(1), 31–36 (2013).
- 13. . Attitudes to stem cell therapy among ischemic stroke survivors in the lund stroke recovery study. Stem Cells Dev. 26(8), 566–572 (2017).
- 14. . Perspectives on strategies and challenges in the conversation about stem cells for spinal cord injury. Spinal Cord 53(11), 811–815 (2015).
- 15. . Shared decision-making, control preferences and psychological well-being in patients with RPE65 deficiency awaiting experimental gene therapy. Ophthalmic Res. 54(2), 96–102 (2015).
- 16. Patient perspectives on gene transfer therapy for sickle cell disease. Erratum appears in Adv Ther. 2017 Aug 2; PMID: 28770518. Adv. Ther. 34(8), 2007–2021 (2017).
- 17. Women's views on autologous cell-based therapy for post-obstetric incontinence. Regen. Med. 11(2), 169–180 (2016).
- 18. . A qualitative study of cystic fibrosis (CF) patients’ expectations of gene therapy. J. Cyst. Fibros. 1, S101 (2010).
- 19. . Acceptability of stem cell therapy by pregnant women. Birth (Berkeley, Calif.) 39(2), 91–97 (2012).
- 20. . Analysis of attitudes toward the source of progenitor cells in tissue-engineered products for use in burns compared with other disease states. Wound Repair Regen. 20(3), 311–316 (2012).
- 21. . Pilot assessment of HIV gene therapy-hematopoietic stem cell clinical trial acceptability among minority patients and their advisors. J. Natl Med. Assoc. 102(12), 1123–1128 (2010).
- 22. . Stem cell research: the role of information seeking and scanning. Health Info. Libr. J. 33(4), 269–282 (2016).
- 23. . Regenerative medicine: stroke survivor and carer views and motivations towards a proposed stem cell clinical trial using placebo neurosurgery. Health Expect. 21(1), 367–378 (2018).
- 24. Parent and adult patient attitudes about gene therapy as a therapeutic option for duchenne muscular dystrophy. Value Health 21(Suppl. 1), S256 (2018).
- 25. . Decision-making in stem cell trials for spinal cord injury: the role of networks and peers. Regen. Med. 7(4), 513–522 (2012).
- 26. . Nationwide survey of patient knowledge and attitudes towards human experimentation using stem cells or bee venom acupuncture for Parkinson's disease. JMD 7(2), 84–91 (2014).
- 27. . Diversity in public views toward stem cell sources and policies. Stem Cell Rev. 5(2), 102–107 (2009).
- 28. . “Is a cure in my sight?” Multi-stakeholder perspectives on Phase I choroideremia gene transfer clinical trials. Genet. Med. 16(5), 379–385 (2014).
- 29. . ‘No one here's helping me, what do you do?’: addressing patient need for support and advice about stem cell treatments. Regen. Med. 12(7), 791–801 (2017).
- 30. . Gordie Howe's stem cell ‘Miracle’: a qualitative analysis of news coverage and readers' comments in newspapers and sports websites. Stem Cell Rev. Rep. 11(5), 667–675 (2015).
- 31. Religion and the public ethics of stem-cell research: attitudes in Europe, Canada and the United States. PLoS ONE 12(4), e0176274 (2017).
- 32. . Patients' attitudes toward the donation of biological materials for the derivation of induced pluripotent stem cells. Cell Stem Cell 14(1), 9–12 (2014).
- 33. . What's in a name: are cultured red blood cells ‘natural’? Sociol. Health Illn. 40(4), 687–701 (2018).
- 34. . Public opinion about stem cell research, 2002 to 2010. Public Opin. Q. 78(4), 1003–1022 (2014).
- 35. . Prevailing public perceptions of the ethics of gene therapy. Hum. Gene Ther. 25(8), 740–746 (2014).
- 36. . Utilizing social media to study information-seeking and ethical issues in gene therapy. J. Med. Internet Res. 15(3), e44 (2013).
- 37. . Identity and ownership issues in the regulation of autologous cells. Regen. Med. 12(7), 827–838 (2017).
- 38. Patient's attitude towards the donation and use of adipose tissue and adipose derived stem cells for research. J. Plast. Reconstr. Aesthet. Surg. 68(4), 588–589 (2015).
- 39. . European attitudes to gene therapy and pharmacogenetics. Drug Discov. Today 16(19–20), 843–847 (2011).
- 40. . US attitudes toward human embryonic stem cell research. Nat. Biotechnol. 29(6), 484–488 (2011).
- 41. . The public and the gene-editing revolution. N. Engl. J. Med. 374(15), 1406–1411 (2016). • A good introductory discussion on public views around the use of gene therapy.
- 42. Public attitudes toward gene therapy in China. Mol. Ther. Methods Clin. Dev. 6, 40–42 (2017).
- 43. . A comparative analysis of attitudes on communication toward stem cell research and regenerative medicine between the public and the scientific community. Stem Cells Transl. Med. 7(2), 251–257 (2018).
- 44. . Firefighter willingness to participate in a stem cell clinical trial for burns: a mixed methods study. Burns 42(8), 1740–1750 (2016).
- 45. . Is belief larger than fact: expectations, optimism and reality for translational stem cell research. BMC Med. 10, 133 (2012).
- 46. . Media presentation and public understanding of stem cells and stem cell research in Hungary. New Genet. Soc. 30(1), 1–26 (2011).
- 47. . The public, political parties, and stem-cell research. N. Engl. J. Med. 365(20), 1853–1856 (2011).
- 48. Eliciting public preferences for healthcare: a systematic review of techniques. Health Technol. Assess. 5(5), 1–186 (2001).
- 49. . Societal preferences for funding orphan drugs in the United Kingdom: an application of person trade-off and discrete choice experiment methods. Value Health 21(5), 538–546 (2018).