8 NEW APPROACHES TO PROMOTING INNOVATION 85 PATIENT

13 HENRY BACON SYNTHESIZING APPROACHES IN FILM THEORY FILM
1INTERNATIONAL LAW RELATED TO PRECAUTIONARY APPROACHES TO NATIONAL REGULATION
20 ABORIGINAL PEOPLE WITH DISABILIY UNIQUE APPROACHES TO UNIQUE

3 NEW APPROACHES FOR LOGISTICBENCHMARKING IN THE CLOTHING INDUSTRY
33 ORGANIZATIONAL COMMITMENTS AND TEACHING APPROACHES DO ORGANIZATIONAL COMMITMENTS
381828DOC PAGE 14 OF 14 TITLE COMPARING DIFFERENT APPROACHES

8 . New approaches to promoting innovation

8.5 Patient and citizen involvement

See Background Paper Chapter 8.5

At the time of the 2004 Report, patient and citizen participation in priority setting was uncommon and knowledge about and experience of the impact of such participation was limited. Today, the involvement of patients and citizens in health research and policy is supported by legal and regulatory requirements. Moreover, there is a substantial body of literature on the topic and much work has been done to realize patient and citizen involvement. This progress indicates that the need for patient and citizen involvement is widely acknowledged by stakeholders. A variety of underlying motivations drive the efforts to involve patients and citizens in priority setting for pharmaceutical innovation.

First there are political reasons, based on the desire to promote democratic ideals of legitimacy, transparency and accountability. In the year 2000, The Council of Europe declared that the right of the public to be involved in the decision-making processes affecting health care is a basic and essential part of any democratic society.^¹ This democratic right is echoed in government reports, legislation and in statements from patient and citizen groups. Setting (research) priorities affects the use of limited public resources, and research demonstrates that values and ethical considerations play a role in recommendations on, for example, guideline development.Therefore, societal values should be considered and decisions should be informed by input from patients and citizens since they are affected by the decisions.^²

The promotion of patient and citizen involvement can be driven by arguments of transparency and trust. For example, an analysis of the benefits of patient involvement by the EMA led the agency to conclude that: “participation of patients in the scientific committees leads to increased transparency and trust in regulatory processes and develops mutual respect between regulators and the community of patients.”^³

A second category is health-related motivation that stems from the need to better align pharmaceutical innovation with the real, unmet needs of patients. Pharmaceutical innovations do not always meet the needs of patients effectively.^⁴ Biases within the health research system may tend to favour certain research and topics over others. This could result, for example, in a lack of interdisciplinary and integral approaches and little attention paid to recovery of patient function.^⁵ In addition, important questions may be overlooked because of an emphasis on chronic but not acute conditions, severe but not common health problems, and disease-specific but not cross-cutting issues, such as social care, improved surgery, and anaesthesia.^⁶ Evidence shows that health professionals’ values of different health states and research priorities differ from those of patients.^⁷

Another health-related motivation focuses on the actual contribution which especially patients can make to the decision-making process, and thus to the rationality of the process and the quality of its direct or long-term outcome. Patients not only have a right to engage in discussions on decision making about priorities (the political stance), their input is also needed because they have a specific, relevant type of knowledge: their ‘experiential knowledge’.^⁸^,^⁹

These motivations provide a strong justification for efforts to further develop patient and citizen involvement in priority setting. A next step is to create an evidence base for meaningful models of involvement. At present, there is a lack of an overview of various initiatives undertaken and several knowledge gaps exist; together these are hampering efforts to evaluate and further develop patient and citizen involvement in priority setting.

Perspectives of patients and citizens

One of the first questions to be asked is: how should we distinguish between patients and citizens? While there is widespread belief that values for health states differ between patients and the general public, there is a long-standing debate among health economists about the evidence to support this belief. Research findings suggest that patient and population preferences can both be used to set priorities for pharmaceutical innovation,^¹⁰ although there is evidence that patients may give higher valuations to health states compared to members of the general public.^¹¹ In general, it seems that patient and citizen involvement can be captured by the term ‘public involvement’ in many but not all cases. For a start, patients and citizens may have competing or contrasting interests in priority setting for pharmaceutical innovation. Second, there are circumstances which call for a more specific use of experience. This is the case when involvement is sought with the explicit aim of seeking to use the experiential knowledge of a patient, or a well-described group of patients or carers.

Models for involvement and their impact

The rich variety of structures for involvement that have been employed in the field of health policy and research is a sign of a developing field of expertise and experience. In general, the literature on roles of patients and citizens in health policy and research appears to reflect a joint search for models for involvement that not only yield a patient or citizen perspective, but also allow real influence on the decisions made. However, one aspect of patient and citizen involvement seems to lag behind in this process: developing good understanding of the expertise and the contribution of patients and citizens at different levels of involvement (i.e. consultation, collaboration or control) and in a variety of models.

Assessment of the impact of patient and citizen involvement is complicated by the way experiences are reported in the literature. In general, these descriptions are brief and provide limited evidence of impact. Concepts like consultation, representation, and expertise have been used interchangeably, with patient and citizen involvement variably defined and often poorly described. Longer qualitative descriptions often provide a better insight into impact. However, while such descriptions can be very valuable, they provide no indication of the extent of impact, its magnitude or how it compares across different areas of impact.^¹²

Validity and representativeness

One of the main arguments for patient involvement concerns the contribution that patients could make to the relevance and quality of biomedical research based on their 'experiential knowledge'. However, the validity of patients' experiential knowledge in the context of biomedical research processes raises questions: To what extent is the experienced perception of a patient representative credible? And how can this specific experiential knowledge be absorbed into the scientific process? The same questions may also occur in citizen involvement: (How) can one representative account for the perspectives of citizens with a variety of social and cultural backgrounds? And how can a lay perspective be preserved when citizens (or patients) are educated to participate in the scientific process?^¹³ What methods can be used to enhance the credibility of the contribution of patients and citizens to the decision making?

A second problem for validity and representativeness is the potential for conflict of interest. Many patient and consumer groups accept pharmaceutical industry funding to support their activities. Some of them see this as a necessity to achieve their aims and argue that patient groups are able to defend their independence from the influence of any sponsor.^¹⁴ Other patient organizations refuse drug industry funding in order to maintain their autonomy.^¹⁵ With the rise of patient and citizen involvement, the focus of attention on conflicts of interest has also grown. Accepting funding from the pharmaceutical industry clearly puts patient organizations in a position of potential conflict of interest. As with conflict of interest among professionals, the response of most regulatory authorities is to request transparency. The EMA, for example, formulated criteria to be met by patients’ and consumers’ organizations involved in EMA activities.^¹⁶ Problems may arise when publication of this information about funding sources leads to diminished trust in patient organizations and additional problems of validity and representativeness. Therefore, relationships with sponsors and common policies to maintain independence should be discussed in order to avoid these problems.

Questions of validity need to be addressed since they limit acceptance of non-expert involvement.^¹⁷ While patient and citizen organizations struggle to demonstrate credibility, their position may be further undermined by ambiguity in their roles and the goals of their involvement in priority-setting and decision making.

Recommendations for research

Although the wide variety of approaches for patient and citizen involvement has helped in the accumulation of experience, this has not yet resulted in a model or a framework for meaningful involvement. Such a framework is needed to ground patient and citizen involvement in an evidence base and to optimize its practice. To help facilitate this, further research is needed on models for patient and citizen involvement, based on a combination of five variables. These variables are: what is the goal of the involvement of patient and citizen organizations in priority setting and decision-making; who should be involved; what is the role or expertise of the patient or representative involved; what level of involvement is pursued; and what is the most suitable structure for involvement.

In the absence of people who are willing and able to realize the full potential of this kind of stakeholder involvement, it will remain a paper tiger –weak and indecisive. What is needed now is capacity building to help ensure the meaningful involvement of patients and citizens in priority setting for pharmaceutical innovation. In addition to the framework development discussed above, other research efforts are needed to establish and capture best practices, mainly in education and training. All stakeholders need to be prepared for decision making on priorities that involve patients and citizens. This requires the empowerment of patients and citizens and education and training for all the parties involved

A third research recommendation is to assure structural outcome assessment of initiatives to involve patients and citizens. This will not only strengthen the evidence for patient and citizen involvement, it is also needed to justify policy making and the expenditures required to facilitate this involvement. Critical scrutiny of initiatives would not only involve description and effect measurement, but also a cost-benefit assessment.

References

1 The development of structures for citizen and patient participation in the decision making process affecting health care. In: Recommendation adopted by the Committee of Ministers of the Council of Europe, Strasbourg, 24 February 2000.

2 Schünemann HJ, Fretheim A, Oxman AD. Improving the use of research evidence in guideline development. 10. Integrating values and consumer involvement. Health Research Policy and Systems 2006;4(22).

3 The role of patients as members of the EMA Human Scientific Committees. European Medicines Agency, 2011.

4 Rosenberg-Yunger ZR, Thorsteinsdóttir H, Daar AS, Martin DK. Stakeholder involvement in expensive drug recommendation decisions: an international perspective. Health Policy, 2012, 105(2-3):226-35.

5 Broerse JEW, Elberse JE et al. Enhancing a transition towards a needs-oriented health research system through patient participation. In: Broerse JEW and Bunders JFG (eds.). Transitions in Health Systems: Dealing with Persistent Problems. Amsterdam, VU University Press, 2010.

6 Partridge N, Scadding J. The James Lind Alliance: patients and clinicians should jointly identify their priorities for clinical trials. Lancet, 2004, 364 (9449), 1923-1924.

7 Tallon D, Chard J, Dieppe P. Relation between agendas of the research community and the research consumer. Lancet, 2000, 355: 2037–2040. PMID: 10885355.

8 Nordin, I. Expert and non-expert knowledge in medical practice. Med Health Care Philos,2000, 3(3): 297-304.

9 Telford, R., C. A. Beverley, et al. Consumer involvement in health research: fact or fiction? British Journal of Clinical Governance, 2002, 7(2): 92-103.

10 Dolders MGT, Zeegers MPA, Groot W, Ament A. A meta-analysis demonstrates no significant differences between patient and population preferences. Journal of Clinical Epidemiology, 2006, Volume 59, Issue 7, pp 653-664.

11 Peeters Y, Stiggelbout AM. Health State Valuations of Patients and the General Public Analytically Compared: A Meta-Analytical comparison of Patient and Population Health State Utilities. Value in Health, 2010, 13 (2):306-309.

12 Staniszewska S et al. Developing the evidence base of patient and public involvement in health and social care research: the case for measuring impact. International Journal of Consumer Studies,2011, 35: 628–632.

13 Ives J, Damery S, Redwod S. PPI, paradoxes and Plato: who’s sailing the ship? J Med Ethics, 2012. doi:10.1136/medethics-2011-100150.

14Kent A. Should patient groups accept money from drug companies? Yes. BMJ, 2007, 334: 934–935.

15 Colombo C, Mosconi P, Villani W, Garattini S. Patient Organizations’ Funding from Pharmaceutical Companies: Is Disclosure Clear, Complete and Accessible to the Public? An Italian Survey. PLoS One, 2012, 7(5): e34974.

16 European Medicines Agency. Criteria to be fulfilled by patients’ and consumers’ organisations involved in European Medicines Agency (EMA) activities. EMA/MB/24913/2005; revised 2011.

17 Harding E, Brown D, Hayward M, Pettinari CJ, Service user perceptions of involvement in developing NICE mental health guidelines: A grounded theory study. Journal of Mental Health, 2010, 19, (3) 249-257

7 OSNOVNA TAKSONOMIJA PRISTUPA USVAJANJU JEZIKA (APPROACHES TO LANGUAGE
8 NEW APPROACHES TO PROMOTING INNOVATION 85 PATIENT
AE201 APPROACHES TO ARCHAEOLOGICAL SURVEY IN THE OLD WORLD

Tags: approaches to, of approaches, innovation, promoting, patient, approaches