People facing life threatening illness have a right to expect that research into their illness will address the uncertainties that matter most to them. Unfortunately, this is not always the case, as Alessandro Liberati (1954-2012)† describes in this editorial, first published in The Lancet, building on his own experience as a patient.
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 healthcare effectiveness and safety. There are examples where patients have succeeded in influencing what gets studied,[2,3] 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. Almost 10 years later, after a relapse of my disease, I looked at the ‘epidemiology’ of myeloma studies on ClinicalTrials.gov. On 31 July 2011, a search using the term ‘multiple myeloma’ identified 1384 studies. Of these, 107 were phase 2—3 comparative studies. However, only in 58 of these studies was overall survival an endpoint, and only in 10 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, 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. Policies developed in the pre-approval phase of drug development are needed, and this process needs strict collaboration with pharmaceutical companies and 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. There is a need for more initiatives such as the UK-based James Lind Alliance, which has developed models for bringing patients and professionals together to identify shared research priorities. In addition, organisations such as The Cochrane Collaboration have an important role, working with research commissioners, to identify those unaddressed uncertainties that matter most to patients, and to ensure that these are viewed as priorities for future funding. I hope this approach can be further debated for many other areas of clinical research in oncology and beyond.
Alessandro Liberati thanks Mariangela Taricco, Iain Chalmers, Gianni Ciccone, Michele Cavo, Nicola Magrini, and Roberto Satolli for useful comments in preparation of this piece.
The Cochrane Library is grateful to Alessandro Liberati, and Richard Horton (Editor of The Lancet), for granting permission to reproduce, with additions, the original letter.
Università di Modena and Reggio Emilia, Modena, Italy; and Agenzia Sanitaria e Sociale Regionale, Bologna, Italy
How to cite: Liberati A. Need to re-align patient-oriented and commercial and academic research [editorial]. The Cochrane Library 2012 (1 Jan). http://www.thecochranelibrary.com/details/editorial/1431131/Need-to-re-align-patient-oriented-and-commercial-and-academic-research.html (accessed Day Month Year).
1. Liberati A. Need to realign patient-oriented and commercial and academic research. The Lancet; 378(9805):1777–8. doi:10.1016/S0140-6736(11)61772-8.
2. Buckley 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, 3 Aug. 201110.1016/j.jclinepi.2011.03.016. [Epub ahead of print]
3. Stewart RJ, Caird J, Oliver K, Oliver S. Patients’ and clinicians’ research priorities. Health Expect 2011; 14(4):439–48. 201010.1111/j.1369-7625.2010.00648.x.
4. Liberati A. An unfinished trip through the uncertainties. BMJdoi: 10.1136/bmj.328.7438.531. 2004; 328: 531–2.
5. Russel SJ, Rajkumar SV. Multiple myeloma and the road for personalised medicine. Lancet Oncol 2011; 12: 617-619. The Lancet Oncology 2011; 12(7): 617–19. doi:10.1016/S1470-2045(11)70143-7.
6. Liberati 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: 69–86.
Competing interests: The author has declared no conflict of interest.
Image credit: Elisa Liberati
Contact the Editor in Chief, Dr David Tovey (firstname.lastname@example.org): Feedback on this editorial and proposals for future editorials are welcome.
†Professor Alessandro Liberati died on 1 January 2012 in Bologna, Italy, from complications of multiple myeloma. He was the inspiring leader of the Italian Cochrane Centre, and a major figure within the evidence-based healthcare community.