Equity, Access and the Allocation of Health (and Research ...



Assessment, Allocation and Access: Stem Cells and Health Systems

A) Background and Rationale

Stem cell research holds great promise for the development of novel therapies. It is hoped that it will result in new treatments for a wide range of diseases, including Parkinson’s, diabetes, and heart disease. While there has been a great deal of inappropriate hype about the near future possibility of stem cell therapies, the area of research holds remarkable promise for health. Less clear and uniformly promising are the potential impacts of these developments on health systems. How will developments in regenerative medicine influence service organization and access, or system cost and sustainability? Will utilization be driven by evidence of clinical and cost effectiveness or hype? And what influence should commercial opportunity have on health system uptake? More broadly, what are the equity issues created by this research? As noted by Faden and her colleagues: “The forthcoming transition in the focus of stem cell research from basic science to the development of therapies raises important questions of justice.” (Faden et al., 2003)?

The area of stem cell research provides a unique opportunity to consider health system uptake issues with an emerging technology. The therapeutic fruits of stem cell research will challenge the purchasers of health services in private and public systems alike, creating vexing problems regarding what therapeutics to purchase, under what conditions and with what degree of public subsidy. Regenerative medicine is not unique in this respect, but exemplifies other “system challenging” health technologies of high cost, considerable potential, and limited evidence. At the same time, regenerative medicine poses some unique challenges arising from the charged moral climate associated with embryonic stem cell research, the extraordinarily inflated consumer and provider expectations arising from both commercial opportunity and ‘quackery’ in this domain, and the cost and complexity issues that emerge where therapeutics must sometimes be ‘personalized’ to ensure optimal effectiveness.

This project will explore assessment, allocation and access to regenerative medicines with the ultimate goal of providing guidance for purchasers and policy makers in planning for anticipated developments in regenerative medicine. We explore these issues in three ways, through:

1. Forecasting future regenerative medicines and the commercial conditions of their production

2. Analysis of the current stem cell therapy marketplace

3. A prospective, comparative analysis of resource allocation decision-making for current ‘system challenging’ health technologies

Guidance generated by the project will be relevant both to the specific issues raised by stem cell research and regenerative medicine and to the more general issues raised by other innovative and system challenging health technologies. The project falls under the Stem Cells and Public Policy Strategic Programs, especially in the areas of: stem cell therapeutic applications knowledge, translation, access to stem cell therapies, economic impact assessments, and health policy.

B) Approach and Method:

The project is designed to develop empirical data to guide decision-making for health system uptake of future regenerative medicines. To do so, it considers three interrelated thematic questions, concerning: (i) anticipated regenerative medicines and the commercial contexts of their production; (ii) the current stem cell therapeutic marketplace in which quackery, “media hype”, and private access prevails; and (iii) resource allocation decisions across multiple jurisdictions for current system-challenging health technologies. Analysis of these three themes will tell us: (i) the expected nature and timing of future therapeutic developments in regenerative medicine, and the commercial contexts that will guide the refinement, marketing and uptake of such products; (ii) how current unproven stem cell therapeutics are being interpreted and managed within affected health systems; and (iii) insight into how multiple jurisdictions currently address the challenges of novel therapeutics in making resource allocation decisions, the ways in which decisions are influenced by factors intrinsic and extrinsic to the technologies themselves, and the range of purchasing options available (e.g., degree of public subsidy, degree of and conditions upon access, etc.).

The project addresses three interrelated questions:

1. Forecasting future regenerative medicines and the commercial conditions of their production. Developments in stem cell science have been advanced both for their therapeutic promise and for their potential to create economic benefits. Traditionally, purchasers of health products and services consider therapeutic issues of clinical safety, efficacy and effectiveness and economic issues such as cost and cost effectiveness. Yet there is increasing interest in consideration of economic development questions such as jurisdiction of origin or manufacture (Brimacombe, 2005). Thus, accurate forecasting of the future of regenerative medicine involves understanding both the therapeutic promise, and the commercial conditions under which therapies will be refined, marketed, purchased and used. We will systematically review claims made in the scientific literature and policy documents regarding future therapeutic products/services and work closely with the SCN research community to solicit a variety of perspectives (e.g., skeptics and optimists) regarding the future potential, problems, commercial conditions and timing of stem cell based therapies and interventions.

2. Analysis of the current “stem cell” therapy market. To date, there are no practical therapeutic applications for embryonic stem cell technologies, yet some companies are attempting to sell stem cell services for everything from the treatment of cancer to Parkinson’s disease, wrinkles and impotence (Baker, 2005; Wired News, 2005). These companies seem to be building on the tremendous profile, hype and legitimate promise of this area of research (see, for example, Emcell ()). It has been reported the desperate patients have paid substantial sums to receive unproven “stem cell” therapies (Braude, et al., 2005). This type of “quackery” places patients at physical and financial risk and could undermine public trust in both the research and future therapies. At the same time, it provides an opportunity to understand how patients, providers and health system managers and regulators interpret and respond to unproven and speculative forms of regenerative medicine, and provides insight into systems of private delivery and access to emerging health services. Our analysis will include a comprehensive identification and examination of the existing national and international stem cell therapy market (what is being offered, how is it been offered, etc) and a comparative analysis of health policy and legislation governing the marketing or delivery of these or related health products (e.g., policy concerning direct-to-consumer advertising or marketing, etc.).

3. Prospective, comparative analysis of resource allocation decision-making for current ‘system challenging’ health technologies. Because of the absence of proven stem cell therapies, purchasers do not currently make decisions about whether and under what conditions to make these novel technologies available to their ‘members’ (whether citizens or consumers). Yet much can be learned about how these decisions might be made, and the possible terms and conditions of health system access, through analysis of purchasing decisions for other system challenging biotechnologies (Giacomini, Miller, & Browman, 2003)(Giacomini 2005). We will conduct a comparative analysis of a set of 6 ‘cases’ of purchasing decisions identified prospectively for system challenging health biotechnologies across Canada and a set of three relevant comparator countries, specifically the US, UK and Australia to develop an understanding of the range of purchasing options available (e.g., degree of public subsidy, degree of and conditions upon access), and of the ways in which the features of specific technologies, and of the commercial and health system contexts in which they are deployed, influence decision-making.

.

The study design is broadly ethnographic, in the tradition of science and technology studies (Law & Hassard, 1999; MacKenzie & Wajcman, 1999). Ethnography is concerned with the observation of everyday behaviour in naturalistic settings, in order to understand the meanings underlying these behaviours (Atkinson, Coffey, Delamont, Lofland, & Lofland, 2001). We combine the ethnographic approach with case study methods that use multiple sources of overlapping data to document complex phenomena when the contexts in which they occur have specific influences on their dynamics (Yin, 1994). In accordance with this orientation, we use several data collection and analysis techniques (legal analysis, semi-structured interviews, document analysis), and an expert workshop. We detail each method in turn:

1. Semi-structured qualitative interviews. We will conduct interviews with members of the stem cell research community to assist our efforts to forecast future regenerative medicines and the commercial conditions of their production. We will also conduct interviews with representatives of purchasing bodies, and policy makers in the several jurisdictions under review to understand: (i) how future regenerative medicines and the commercial conditions of their production might be received, (ii) how the current stem cell therapy market is interpreted and managed, and (iii) how resource allocation decisions are made for system challenging health biotechnologies.

Interviews will be in-depth and semi-structured interviews to gain insight into the values, attitudes and experiences of respondents. Interviews will be open-ended, encouraging participants to address issues of particular interest and concern in the sequence and style that they prefer (Kvale, 1996). We will impose a question structure through the use of an interview guide that identifies the questions we are seeking input on, but encourage open-ended responses and a conversational style that may depart from the question sequence or structure (Foddy, 1994; Gilchrist & Williams, 1999).

2. Document analysis. We will collect and analyze print media and policy documents from the relevant jurisdictions in order to: (i) understand the claims made regarding future therapeutic stem cell products/services and the commercial conditions and timing of their development; (ii) understand the contours of the current national and international stem cell therapy market (what is being offered, how is it been offered, etc) and relevant health policy and legislation governing the marketing or delivery of these or related health products: (iii) identify and analyze a set of 6 ‘cases’ of purchasing decisions for system challenging health biotechnologies across relevant jurisdictions.

3. Legal analysis. We will collect and analyze relevant statutes, jurisprudence and regulation in the jurisdictions under review to better understand how the current stem cell therapy market is being and might be governed.

4. Expert workshop. The project will conclude with an expert workshop of academics, policy makers and other key stakeholders (e.g., basic researchers, industry representatives, etc.) to foster the development of guidelines for policy makers and purchasers in planning for anticipated developments in regenerative medicine. Such guidance will be relevant both to the specific issues created by regenerative medicine and to the more general issues associated with innovative and ‘system challenging’ health technologies and will include a consideration of public perception issues.

C) Outcomes

Many policy reports have noted the need for more robust technology assessment and coordinated resource allocation decision-making (Romanow, 2002; Lee et al., 2003). This project, which we believe is the first of its kind in relation to stem cell research, will help to inform current and future policy development. We have and will continue to establish links with key provincial health system policymakers (see attached letters) and will ensure that knowledge generated from the project is presented in an utilizable format for relevant knowledge consumers. The project will also inform the current stem cell research agenda in the context of health care systems. In other words, we hope this project will not only inform future purchasing policy, but also provide the SCN with help in discerning what research is needed now and what potential stem cell therapies are most likely to meet the prospective needs of health purchasers.

In addition to traditional modes of knowledge translation (KT) using explicit or codified knowledge channels e.g. articles, books, reports etc., this project will explore KT strategies identified in recent literature aimed at improving the knowledge-value chain, especially by producing ‘know-how’ targeted towards specific needs of policymakers and public health officials (Landry et al., 2006). The project design will also promote increased networking between the scientific, ELSI, and policymaking community (forecasting interviews), and provide opportunities for HQP and trainee development at every stage.

Specific outcomes include:

1. Resource allocation planning guidelines;

2. Forecasting data to inform both public dialogue/communication strategies and resource allocation policies;

3. Guidance to SCN on current and future stem cell market and emerging health system issues;

4. Development of an informal stem cell research resource allocation network (including relevant policy makers) to monitor ongoing policy developments in this area beyond the life of the project;

5. First international database of stem cell research-specific resource allocation knowledge This will include some of first conceptual work on health systems issues associated with stem cell research; and

6. International collaboration, HQP and trainee development.

D) Milestones

1. Literature collection, review and analysis: The project will commence with an extensive literature collection, review and analysis covering all identified areas (claims and commercial conditions, current stem cell market and the regulatory environment, identification of 6 ‘cases’ of purchasing decisions, international comparative analysis). This milestone will lead to the production of review articles, background papers, papers suitable for publication in peer-reviewed journals, and a comprehensive bibliography and database of resource allocation materials relevant to stem cell research.

2. Planning workshop: This workshop will bring together core project members to formulate research questions, firm up data collection approach and methodology, and develop possible approaches for data analysis.

3. Data collection and analysis: Data collection will be done through semi-structured interviews (see page 3). This milestone will lead to the production of scholarly papers and editorials for the popular press.

4. Forecasting publications, including a comparative analysis with media representations.

5. Production and dissemination of a workshop report, planning guidelines, and scholarly papers.

[pic]

RA: Ogbogu

Steve Morgan, Jeremiah Hurley, Fiona Miller, Mita Giacomini , “Predictive genetic tests and health system costs” (2003) 168 CMAJ 989-991

BBC News, “Stem cell therapy disease warning” (19 May 2005) ()

Stephen Barrett, “The Shady Side of Embryonic Stem Cell Therapy” Quackwatch ()

EmCell ()

WiredNews, “Stem Cell Craze Spreads in Russia” (14 March 2005) ()

Monya Baker, “Stem Cell Therapy or Snake Oil?” (2005) 23 Nature Biotechnology 1467-1469.

Pater Braude, Stephen Minger, Ruth Warwick, “Stem cell therapy: hope or hype?” (2005) 330 BMJ 1159.

R. Lee et al., “Health Technology Assessment, Research and Implementation Within A Health Region in Alberta” (2003) 19 International Journal of Technology Assessment in Health Care 513-520.

Ruth Faden, et al., “Public Stem Cell Banks;” (Nov/Dec 2003) Hastings Center Report 13-27.

Atkinson, P., Coffey, A., Delamont, S., Lofland, J., & Lofland, L. (2001). Editorial introduction. In P. Atkinson,A. Coffey,S. Delamont,J. Lofland, & L. Lofland (Eds.), Handbook of Ethnography (pp. 1-7). London, Thousand Oaks, New Delhi: Sage Publications.

Brimacombe, G. (2005). Health, healthcare and nation-building: a three-dimensional approach to innovation in Canada. Healthcare Quarterly, 8(3), 65-68.

Foddy, W. (1994). Constructing Questions for Interviews and Questionnaires: Theory and Practice in Social Research. Cambridge, UK: Cambridge University Press

Giacomini, M., Miller, F.A., & Browman, G. (2003). Confronting the 'gray zones' of technology assessment: evaluating genetic testing services for public insurance coverage in Canada. International Journal of Technology Assessment in Health Care, 19(2), 301-315.

Giacomini, M. (2005). One of these things is not like the others: the idea of precedence in health technology assessment and coverage decisions. Milbank Quarterly. 83(2): 193-223.

Gilchrist, V.J., & Williams, R.L. (1999). Key informant interviews. In B.F. Crabtree, & W.L. Miller (Eds.), Doing Qualitative Research (pp. 71-88). Thousand Oaks, London, New Delhi: Sage Publications.

Kvale, S. (1996). Interviews: an introduction to qualitative research interviewing. Thousand Oaks, California: Sage

Law, J., & Hassard, J. (1999). Actor Network Theory and After. Oxford: Blackwell Publishers.

Landry, R. et al. (2006). The knowledge-value chain: a conceptual framework for knowledge translation in health. Bulletin of the World Health Organization. 84(8), 597-602.

MacKenzie, D., & Wajcman, J. (1999). The social shaping of technology. Buckingham, Philadelphia: Open University Press.

Yin, R.K. (1994). Case study research. design and methods Newbury Park, London, New Delhi: Sage Publications

................
................

In order to avoid copyright disputes, this page is only a partial summary.

Google Online Preview   Download