Title: Need to realign patient-oriented and commercial and academic research
Abstract: Clinical research is motivated by several factors. Some are more defensible than others, but most clinical researchers would state that their research is intended to improve health-care effectiveness and safety. There are examples where patients have succeeded in influencing what gets studied,1Buckley BS Grant A Glazener CMA Case study: a patient-clinician collaboration that identified and prioritized evidence gaps and stimulated research involvement.J Clin Epidemiol. 2011; (published online Aug 3.)https://doi.org/10.1016/j.jclinepi.2011.03.016Summary Full Text Full Text PDF Scopus (24) Google Scholar, 2Stewart RJ Caird J Oliver K Oliver S Patients' and clinicians' research priorities.Health Expect. 2010; (published online Dec 22.)https://doi.org/10.1111/j.1369-7625.2010.00648.xCrossref PubMed Scopus (77) Google Scholar but these are exceptions.I have had the opportunity to consider from more than one perspective the mismatch between what clinical researchers do and what patients need. I am a researcher; I have responsibility for allocating funding for research; and I have had multiple myeloma for the past decade. A few years ago I stated publicly that several uncertainties I faced at the beginning of my disease were avoidable.3Liberati A An unfinished trip through the uncertainties.BMJ. 2004; 328: 531-532Crossref Scopus (27) Google Scholar Almost 10 years later—after a relapse of my disease—I looked at the "epidemiology" of myeloma studies on ClinicalTrials.gov. On July 31, 2011, a search using the term "multiple myeloma" identified 1384 studies. Of these, 107 were phase 2/3 comparative studies. However, in only 58 of these studies was overall survival an endpoint, and in only ten of these was it the primary endpoint. No trial was a head-to-head comparison of different drugs or strategies. Meanwhile, experts feel that cytogenetic studies and gene-expression profiling will lead to personalised treatment in myeloma,4Russel SJ Rajkumar SV Multiple myeloma and the road for personalised medicine.Lancet Oncol. 2011; 12: 617-619Summary Full Text Full Text PDF PubMed Scopus (42) Google Scholar and pharmaceutical companies avoid research that might show that new and expensive drugs are no better than another comparator already on the market.If we want more relevant information to become available, a new research governance strategy is needed. Left to themselves, researchers cannot be expected to address the current mismatch. Researchers are trapped by their own internal competing interests—professional and academic—which lead them to compete for pharmaceutical industry funding for early-phase trials instead of becoming champions of strategic, head-to-head, phase 3 studies.Nor are patients' groups alone likely to change the prevailing pattern of research: given the lack of explicit mechanisms for research prioritisation, they are often dominated by experts with vested interests. Neither would public funding alone solve the problem.5Liberati A Moja PL Trotta F Traversa G Feasibility and challenge of independent research on drugs: the Italian Medicines Agency (AIFA) experience.Eur J Clin Invest. 2010; 40: 69-86Crossref PubMed Scopus (41) Google Scholar Policies developed in the preapproval phase of drug development are needed, and this process needs strict collaboration with pharmaceutical companies and with input from regulatory bodies.An essential component of any new governance strategy would be to bring together all the stakeholders, starting from an analysis of existing and ongoing research, produced independently of vested interests. Patient advocacy groups in myeloma spend millions to support research, hoping to promote better care. With public support they should be in a strong position to call for a redefinition of the research agenda, in the interests of patients. I hope this approach can be further debated in The Lancet for many other areas of clinical research in oncology and beyond.I thank Mariangela Taricco, Iain Chalmers, Gianni Ciccone, Michele Cavo, Nicola Magrini, and Roberto Satolli for useful comments in preparation of this letter. I declare that I have no conflicts of interest. Clinical research is motivated by several factors. Some are more defensible than others, but most clinical researchers would state that their research is intended to improve health-care effectiveness and safety. There are examples where patients have succeeded in influencing what gets studied,1Buckley BS Grant A Glazener CMA Case study: a patient-clinician collaboration that identified and prioritized evidence gaps and stimulated research involvement.J Clin Epidemiol. 2011; (published online Aug 3.)https://doi.org/10.1016/j.jclinepi.2011.03.016Summary Full Text Full Text PDF Scopus (24) Google Scholar, 2Stewart RJ Caird J Oliver K Oliver S Patients' and clinicians' research priorities.Health Expect. 2010; (published online Dec 22.)https://doi.org/10.1111/j.1369-7625.2010.00648.xCrossref PubMed Scopus (77) Google Scholar but these are exceptions. I have had the opportunity to consider from more than one perspective the mismatch between what clinical researchers do and what patients need. I am a researcher; I have responsibility for allocating funding for research; and I have had multiple myeloma for the past decade. A few years ago I stated publicly that several uncertainties I faced at the beginning of my disease were avoidable.3Liberati A An unfinished trip through the uncertainties.BMJ. 2004; 328: 531-532Crossref Scopus (27) Google Scholar Almost 10 years later—after a relapse of my disease—I looked at the "epidemiology" of myeloma studies on ClinicalTrials.gov. On July 31, 2011, a search using the term "multiple myeloma" identified 1384 studies. Of these, 107 were phase 2/3 comparative studies. However, in only 58 of these studies was overall survival an endpoint, and in only ten of these was it the primary endpoint. No trial was a head-to-head comparison of different drugs or strategies. Meanwhile, experts feel that cytogenetic studies and gene-expression profiling will lead to personalised treatment in myeloma,4Russel SJ Rajkumar SV Multiple myeloma and the road for personalised medicine.Lancet Oncol. 2011; 12: 617-619Summary Full Text Full Text PDF PubMed Scopus (42) Google Scholar and pharmaceutical companies avoid research that might show that new and expensive drugs are no better than another comparator already on the market. If we want more relevant information to become available, a new research governance strategy is needed. Left to themselves, researchers cannot be expected to address the current mismatch. Researchers are trapped by their own internal competing interests—professional and academic—which lead them to compete for pharmaceutical industry funding for early-phase trials instead of becoming champions of strategic, head-to-head, phase 3 studies. Nor are patients' groups alone likely to change the prevailing pattern of research: given the lack of explicit mechanisms for research prioritisation, they are often dominated by experts with vested interests. Neither would public funding alone solve the problem.5Liberati A Moja PL Trotta F Traversa G Feasibility and challenge of independent research on drugs: the Italian Medicines Agency (AIFA) experience.Eur J Clin Invest. 2010; 40: 69-86Crossref PubMed Scopus (41) Google Scholar Policies developed in the preapproval phase of drug development are needed, and this process needs strict collaboration with pharmaceutical companies and with input from regulatory bodies. An essential component of any new governance strategy would be to bring together all the stakeholders, starting from an analysis of existing and ongoing research, produced independently of vested interests. Patient advocacy groups in myeloma spend millions to support research, hoping to promote better care. With public support they should be in a strong position to call for a redefinition of the research agenda, in the interests of patients. I hope this approach can be further debated in The Lancet for many other areas of clinical research in oncology and beyond. I thank Mariangela Taricco, Iain Chalmers, Gianni Ciccone, Michele Cavo, Nicola Magrini, and Roberto Satolli for useful comments in preparation of this letter. I declare that I have no conflicts of interest.
Publication Year: 2011
Publication Date: 2011-11-01
Language: en
Type: letter
Indexed In: ['crossref', 'pubmed']
Access and Citation
Cited By Count: 96
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