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Perguntas frequentes

Why are some conditions more suitable for gene and cell therapies than others?
There is an approved treatment available for my condition - why can't I receive it?
How to look for approved medicines?
How are medicines developed and approved?

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Glossário

Adeno-associated virus (AAV)
Adenovirus
Adult stem cells
Antibody
Antigen
Base editors
Cas9 nuclease
Chimeric Antigen Receptor (CAR)
Chromosome
Clinical trial

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ATMP PPI Articles

  1. Abelson, J. (2018). "Patient engagement in health technology assessment: what constitutes ‘meaningful’ and how we might get there." Journal of Health Services Research & Policy 23(2): 69-71.
  2. Abelson, J., et al. (2016). "PUBLIC AND PATIENT INVOLVEMENT IN HEALTH TECHNOLOGY ASSESSMENT: A FRAMEWORK FOR ACTION." International Journal of Technology Assessment in Health Care 32(4): 256-264.
  3. Addison, C. (2017). "Bench, bedside, boardroom: negotiating translational gene therapy." New Genetics and Society 36(1): 22-42. 
  4. Bagley, H., et al. (2015). "Developing a toolkit for patient and public involvement in a clinical trials unit." Trials 16(2): O92.    
  5. Bagley, H. J., et al. (2016). "A patient and public involvement (PPI) toolkit for meaningful and flexible involvement in clinical trials – a work in progress." Research Involvement and Engagement 2(1): 15.
  6. Barber, R., et al. (2012). "Can the impact of public involvement on research be evaluated? A mixed methods study." Health Expectations 15(3): 229-241.
  7. Brett, J., et al. (2014). "Mapping the impact of patient and public involvement on health and social care research: a systematic review." Health Expectations 17(5): 637-650.
  8. Brighton, L. J., et al. (2018). "Taking patient and public involvement online: qualitative evaluation of an online forum for palliative care and rehabilitation research." Research Involvement and Engagement 4(1): 14.
  9. Buck, D., et al. (2014). "From plans to actions in patient and public involvement: qualitative study of documented plans and the accounts of researchers and patients sampled from a cohort of clinical trials." BMJ Open 4(12): e006400.
  10. Cavaller-Bellaubi, M., et al. (2021). "Sustaining Meaningful Patient Engagement Across the Lifecycle of Medicines: A Roadmap for Action." Therapeutic Innovation & Regulatory Science 55(5): 936-953.
  11. Chudyk, A. M., et al. (2018). "Models and frameworks of patient engagement in health services research: a scoping review protocol." Research Involvement and Engagement 4(1): 28.
  12. Cordier, J.-F. (2014). "The expert patient: towards a novel definition." European Respiratory Journal 44(4): 853-857.
  13. Coulman, K. D., et al. (2020). "Understanding and optimising patient and public involvement in trial oversight: an ethnographic study of eight clinical trials." Trials 21(1): 543.
  14. Crocker, J. C., et al. (2017). "Is it worth it? Patient and public views on the impact of their involvement in health research and its assessment: a UK-based qualitative interview study." Health Expectations 20(3): 519-528.
  15. Crocker, J. C., et al. (2019). "Patient and public involvement (PPI) in UK surgical trials: a survey and focus groups with stakeholders to identify practices, views, and experiences." Trials 20(1): 119.
  16. Crocker, J. C., et al. (2018). "Impact of patient and public involvement on enrolment and retention in clinical trials: systematic review and meta-analysis." BMJ 363: k4738.
  17. Culme-Seymour, E. J., et al. (2015). "Cell Therapy Regulatory Toolkit: an online regulatory resource." Regenerative Medicine 10(5): 531-534.    
  18. de Bekker-Grob, E. W., et al. (2017). "Giving Patients’ Preferences a Voice in Medical Treatment Life Cycle: The PREFER Public–Private Project." The Patient - Patient-Centered Outcomes Research 10(3): 263-266.    
  19. Dean, S., et al. (2017). "“The patient is speaking”: discovering the patient voice in ophthalmology." British Journal of Ophthalmology 101(6): 700-708.
  20. Devonport, T. J., et al. (2018). "It’s not just ‘What’ you do, it’s also the ‘Way’ that you do it: Patient and Public Involvement in the Development of Health Research." International Journal for Quality in Health Care 30(2): 152-156.
  21. Dudley, L., et al. (2015). "A little more conversation please? Qualitative study of researchers’ and patients’ interview accounts of training for patient and public involvement in clinical trials." Trials 16(1): 190.
  22. Dudley, L., et al. (2015). "What Difference Does Patient and Public Involvement Make and What Are Its Pathways to Impact? Qualitative Study of Patients and Researchers from a Cohort of Randomised Clinical Trials." PLOS ONE 10(6): e0128817.
  23. Exley, A. R., et al. (2020). "Clinical development of cell therapies for cancer: The regulators' perspective." European Journal of Cancer 138: 41-53.
  24. Feldman, D., et al. (2021). "Co-creation of practical “how-to guides” for patient engagement in key phases of medicines development—from theory to implementation." Research Involvement and Engagement 7(1): 57.
  25. Fleming, P. R., et al. (2021). "Patient engagement in fertility research: bench research, ethics, and social justice." Research Involvement and Engagement 7(1): 29.
  26. Gamble, C., et al. (2014). "Patient and public involvement in the early stages of clinical trial development: a systematic cohort investigation." BMJ Open 4(7): e005234.
  27. Gibson, A., et al. (2017). "Evaluating patient and public involvement in health research: from theoretical model to practical workshop." Health Expectations 20(5): 826-835.
  28. Goulao, B., et al. (2021). "Patient and public involvement in numerical aspects of trials (PoINT): exploring patient and public partners experiences and identifying stakeholder priorities." Trials 22(1): 499.
  29. Grine, L., et al. (2020). "Improving Patient Involvement in the Lifecycle of Medicines: Insights From the EUPATI BE Survey." Frontiers in Medicine 7.
  30. Haerry, D., et al. (2018). "EUPATI and Patients in Medicines Research and Development: Guidance for Patient Involvement in Regulatory Processes." Frontiers in Medicine 5.
  31. Harrington, R. L., et al. (2020). "Defining Patient Engagement in Research: Results of a Systematic Review and Analysis: Report of the ISPOR Patient-Centered Special Interest Group." Value in Health 23(6): 677-688.
  32. Hunter, A., et al. (2018). "EUPATI Guidance for Patient Involvement in Medicines Research and Development: Health Technology Assessment." Frontiers in Medicine 5.
  33. Iorio, A., et al. (2018). "Core outcome set for gene therapy in haemophilia: Results of the coreHEM multistakeholder project." Haemophilia 24(4): e167-e172.
  34. Jones, M. and I. Pietilä (2020). "Personal perspectives on patient and public involvement – stories about becoming and being an expert by experience." Sociology of Health & Illness 42(4): 809-824.
  35. Kearney, A., et al. (2017). "Priorities for methodological research on patient and public involvement in clinical trials: A modified Delphi process." Health Expectations 20(6): 1401-1410.
  36. Klingmann, I., et al. (2018). "EUPATI and Patients in Medicines Research and Development: Guidance for Patient Involvement in Ethical Review of Clinical Trials." Frontiers in Medicine 5.
  37. Knowles, S. E., et al. (2021). "More than a method: trusting relationships, productive tensions, and two-way learning as mechanisms of authentic co-production." Research Involvement and Engagement 7(1): 34.
  38. Lander, J., et al. (2019). "Involving patients and the public in medical and health care research studies: An exploratory survey on participant recruiting and representativeness from the perspective of study authors." PLOS ONE 14(1): e0204187.
  39. Laverty, H. and P. Meulien (2019). "The Innovative Medicines Initiative −10 Years of Public-Private Collaboration." Frontiers in Medicine 6.
  40. Levitan, B., et al. (2018). "Assessing the Financial Value of Patient Engagement: A Quantitative Approach from CTTI’s Patient Groups and Clinical Trials Project." Therapeutic Innovation & Regulatory Science 52(2): 220-229.
  41. Lopes, E., et al. (2016). "Involving patients in health technology funding decisions: stakeholder perspectives on processes used in Australia." Health Expectations 19(2): 331-344.
  42. Maguire, K. and N. Britten (2018). "‘You're there because you are unprofessional’: patient and public involvement as liminal knowledge spaces." Sociology of Health & Illness 40(3): 463-477.
  43. Mann, C., et al. (2018). "Reporting and appraising the context, process and impact of PPI on contributors, researchers and the trial during a randomised controlled trial - the 3D study." Research Involvement and Engagement 4(1): 15.
  44. Mathie, E., et al. (2018). "Reciprocal relationships and the importance of feedback in patient and public involvement: A mixed methods study." Health Expectations 21(5): 899-908.
  45. McCarron, T. L., et al. (2021). "Patients as partners in health research: A scoping review." Health Expectations 24(4): 1378-1390.
  46. Mercer, R. E., et al. (2020). "Are We Making a Difference? A Qualitative Study of Patient Engagement at the pan-Canadian Oncology Drug Review: Perspectives of Patient Groups." Value in Health 23(9): 1157-1162. 
  47. Miah, J., et al. (2019). "Patient and public involvement in dementia research in the European Union: a scoping review." BMC Geriatrics 19(1): 220.
  48. Mikami, K. and S. Sturdy (2017). "Patient organization involvement and the challenge of securing access to treatments for rare diseases: report of a policy engagement workshop." Research Involvement and Engagement 3(1): 14.
  49. Mühlbacher, A. C., et al. (2016). "Patient-Focused Benefit-Risk Analysis to Inform Regulatory Decisions: The European Union Perspective." Value in Health 19(6): 734-740.    
  50. O'Shea, A., et al. (2017). "Whose voices? Patient and public involvement in clinical commissioning." Health Expectations 20(3): 484-494.
  51. Pandya-Wood, R., et al. (2017). "A framework for public involvement at the design stage of NHS health and social care research: time to develop ethically conscious standards." Research Involvement and Engagement 3(1): 6.
  52. Parsons, S., et al. (2016). "What do pharmaceutical industry professionals in Europe believe about involving patients and the public in research and development of medicines? A qualitative interview study." BMJ Open 6(1): e008928.
  53. Pellegrini, G., et al. (2016). "From discovery to approval of an advanced therapy medicinal product-containing stem cells, in the EU." Regenerative Medicine 11(4): 407-420.
  54. Pickering, E. C., et al. (2021). "Formalising the induction of patient and public involvement contributors on trial oversight committees." Research Involvement and Engagement 7(1): 42.
  55. Price, A., et al. (2018). "Patient and public involvement in the design of clinical trials: An overview of systematic reviews." Journal of Evaluation in Clinical Practice 24(1): 240-253.
  56. Rayment, J., et al. (2017). "Involving seldom-heard groups in a PPI process to inform the design of a proposed trial on the use of probiotics to prevent preterm birth: a case study." Research Involvement and Engagement 3(1): 11.
  57. Redman, S., et al. (2021). "Co-production of knowledge: the future." BMJ 372: n434.   
  58. Rix, J., et al. (2021). "Stakeholder involvement in the development of trial material for a clinical trial." Health Expectations 24(2): 399-410.
  59. Rix, J., et al. (2021). "A public and patient consultation process as an aid to design a person-centred randomized clinical trial." Health Expectations 24(5): 1639-1648.
  60. Russell, J., et al. (2020). "The impact of public involvement in health research: what are we measuring? Why are we measuring it? Should we stop measuring it?" Research Involvement and Engagement 6(1): 63.
  61. Schilling, I., et al. (2019). "Patient involvement in clinical trials: motivation and expectations differ between patients and researchers involved in a trial on urinary tract infections." Research Involvement and Engagement 5(1): 15.
  62. Selman, L. E., et al. (2021). "Patient and public involvement in randomised clinical trials: a mixed-methods study of a clinical trials unit to identify good practice, barriers and facilitators." Trials 22(1): 735.
  63. Shippee, N. D., et al. (2015). "Patient and service user engagement in research: a systematic review and synthesized framework." Health Expectations 18(5): 1151-1166.
  64. South, A., et al. (2016). "Models and impact of patient and public involvement in studies carried out by the Medical Research Council Clinical Trials Unit at University College London: findings from ten case studies." Trials 17(1): 376.
  65. Spindler, P. and B. S. Lima (2018). "Editorial: The European Patients Academy on Therapeutic Innovation (EUPATI) Guidelines on Patient Involvement in Research and Development." Frontiers in Medicine 5.   
  66. Staley, K., et al. (2017). "The impact of involvement on researchers: a learning experience." Research Involvement and Engagement 3(1): 20.
  67. Staley, K. and D. Barron (2019). "Learning as an outcome of involvement in research: what are the implications for practice, reporting and evaluation?" Research Involvement and Engagement 5(1): 14.
  68. Staley, K., et al. (2014). "‘The missing links’: understanding how context and mechanism influence the impact of public involvement in research." Health Expectations 17(6): 755-764.
  69. Staley, K., et al. (2019). "‘What can I do that will most help researchers?’ A different approach to training the public at the start of their involvement in research." Research Involvement and Engagement 5(1): 10.
  70. Staley, K. and C. Doherty (2016). "It’s not evidence, it’s insight: bringing patients’ perspectives into health technology appraisal at NICE." Research Involvement and Engagement 2(1): 4.
  71. Staley, K. and J. Elliott (2017). "Public involvement could usefully inform ethical review, but rarely does: what are the implications?" Research Involvement and Engagement 3(1): 30.
  72. Staley, K., et al. (2021). "Who should I involve in my research and why? Patients, carers or the public?" Research Involvement and Engagement 7(1): 41.
  73. Staniszewska, S., et al. (2011). "The GRIPP checklist: Strengthening the quality of patient and public involvement reporting in research." International Journal of Technology Assessment in Health Care 27(4): 391-399.
  74. Staniszewska, S., et al. (2017). "GRIPP2 reporting checklists: tools to improve reporting of patient and public involvement in research." BMJ 358: j3453.
  75. Stewart, R. J., et al. (2011). "Patients’ and clinicians’ research priorities." Health Expectations 14(4): 439-448.
  76. Stuhlfauth, S., et al. (2019). "Coming from two different worlds—A qualitative, exploratory study of the collaboration between patient representatives and researchers." Health Expectations 22(3): 496-503.
  77. Telford, R., et al. (2004). "What does it mean to involve consumers successfully in NHS research? A consensus study." Health Expectations 7(3): 209-220.
  78. Thompson, J., et al. (2009). "Health researchers’ attitudes towards public involvement in health research." Health Expectations 12(2): 209-220.
  79. Vale, C. L., et al. (2018). "When participants get involved: reconsidering patient and public involvement in clinical trials at the MRC Clinical Trials Unit at UCL." Trials 19(1): 95.
  80. Vale, C. L., et al. (2012). "Involvement of consumers in studies run by the Medical Research Council Clinical Trials Unit: Results of a survey." Trials 13(1): 9.
  81. van Rensen, A., et al. (2020). "The Launch of the European Patients' Academy on Therapeutic Innovation in the Netherlands: A Qualitative Multi-Stakeholder Analysis." Frontiers in Medicine 7.
  82. Vat, L. E., et al. (2021). "Evaluation of patient engagement in medicine development: A multi-stakeholder framework with metrics." Health Expectations 24(2): 491-506.
  83. Wale, J., et al. (2017). "WHY PATIENTS SHOULD BE INVOLVED IN HEALTH TECHNOLOGY ASSESSMENT." International Journal of Technology Assessment in Health Care 33(1): 1-4.
  84. Warner, K., et al. (2018). "EUPATI Guidance for Patient Involvement in Medicines Research and Development (R&D); Guidance for Pharmaceutical Industry-Led Medicines R&D." Frontiers in Medicine 5.
  85. Weeks, L., et al. (2017). "EVALUATION OF PATIENT AND PUBLIC INVOLVEMENT INITIATIVES IN HEALTH TECHNOLOGY ASSESSMENT: A SURVEY OF INTERNATIONAL AGENCIES." International Journal of Technology Assessment in Health Care 33(6): 715-723.
  86. Whichello, C., et al. (2020). "An overview of critical decision-points in the medical product lifecycle: Where to include patient preference information in the decision-making process?" Health Policy 124(12): 1325-1332.
  87. Wilson, M., et al. (2021). "Stakeholder engagement in economic evaluation: Protocol for using the nominal group technique to elicit patient, healthcare provider, and health system stakeholder input in the development of an early economic evaluation model of chimeric antigen receptor T-cell therapy." BMJ Open 11(8): e046707.
  88. Wilson, P., et al. (2015). "ReseArch with Patient and Public invOlvement: a RealisT evaluation - the RAPPORT study." Health Serv Deliv Res 3(38).
  89. Witham, M. D., et al. (2020). "Developing a roadmap to improve trial delivery for under-served groups: results from a UK multi-stakeholder process." Trials 21(1): 694.
  90. Woollard, L., et al. (2021). "Improving patient informed consent for haemophilia gene therapy: the case for change." Therapeutic Advances in Rare Disease 2: 26330040211047244.
  91. Yu, R., et al. (2021). "Evaluation of a patient and public involvement training programme for researchers at a large biomedical research centre in the UK." BMJ Open 11(8): e047995.
  92. den Oudendammer, W.M., Noordhoek, J., Abma-Schouten, R.Y. et al. (2019). "Patient participation in research funding: an overview of when, why and how amongst Dutch health funds." Res Involv Engagem 5 (33).

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