Biotechnology and the Health of Canadians (abbreviated version of the December 2004 full report by the same name)

Realizing the promise and meeting the challenges of biotechnology-based health innovation (BHI) in the context of economic, social and political realities will create pressures in Canadian society. Ignoring the pressures for change will not make them disappear — more likely it will simply compound the costs and complexities of dealing with them later. Unless governments effectively address the policy imperatives associated with BHI, Canadians may fail to capture its benefits or may adopt innovations that are ill-suited to Canada's health care system.

With proper planning and by building on the investments already made in new technological development, governments have the opportunity to make biotechnology work for all Canadians.

Over the past century, BHIs (from the development of vaccines and antibiotics, to organ transplantation and gene-based medicine) have contributed significantly to the remarkable improvements in the health of Canadians and may play an even more significant role in the future. Indeed, some predict that BHI may well induce fundamental transformation in how we organize, manage and deliver preventive, diagnostic and therapeutic services.

In Biotechnology and the Health of Canadians, the Canadian Biotechnology Advisory Committee (CBAC) describes the potential role of biotechnology in relation to the various factors that influence health, discusses some of the social and ethical considerations involved in the applications of biotechnology, sets out a policy framework, and recommends actions necessary to equip our systems to meet the challenges that the unfolding era of modern biotechnology will present. In undertaking its analysis, CBAC was mindful that BHI is but one of the ways to improve the health of Canadians. Factors in the social and physical environments have a powerful influence on health and also require concerted attention.

The Promise of Biotechnology

Rapid expansion of knowledge about the molecular basis of health and disease holds the promise of improving our ability to prevent, diagnose and treat disease and disability, and to promote health.

In prevention, biotechnology plays a role in addressing both genetic and environmental influences on health through advances in screening populations for susceptibility to disease; in reducing exposure to noxious agents in the environment and enhancing the body's ability to block or ameliorate the effect of such agents; in the application of genomics and proteomics to the development of vaccines and other preventive strategies against infectious agents long-associated with human disease and those emerging in recent years (e.g., HIV, the SARS virus and West Nile Virus); and in the application of pharmacogenomics to the development of anti-microbials for preventive use in special circumstances.

In diagnosis, advances in biotechnology broaden the array of sensitive and specific tests that speed diagnosis and permit greater individualization of treatment. In the case of emerging infectious diseases, new techniques to detect the presence of infectious agents will be applied both to persons who are symptomatic and to populations at risk.

In treatment, biotechnology will play a significant and, in some cases, pre-eminent role through the application of genomics and proteomics to therapeutic drug development; the development of immunological approaches to the treatment of cancer; the use of stem cells to repopulate diseased organs with tissue-specific cells that function normally; tissue engineering and xenotransplantation to increase the supply of tissues for repairing or replacing defective anatomic structures; and gene therapy to correct primary defects within the genetic makeup of individuals.

Looking ahead, one can reasonably anticipate that many BHIs will result from the application of advances in chemistry, physics, engineering, and computational and information sciences to biological systems. For example, the ability to design devices that operate at the molecular level or new materials through advances in nanotechnology could well provide powerful new tools for clinical application.

The Challenges of BHI

The challenges in applying biotechnology to human health include the need to manage risks and prevent or ameliorate potential harms; to deal with the social and ethical implications of the use of technologies and the personal information resulting from that use; and to enhance access to an expanded array of beneficial BHIs.

There are also challenges related to both the process of policy-making and the implementation of strategies for governing and fostering the development and uses of BHI, including: the limited capacity of our systems and personnel to cope with the rapid pace of new technology development; the multiplicity of jurisdictions and stakeholders involved; the diverse levels of knowledge, interest and engagement within the body politic; the constraining effects of international obligations; and the complexity of scientific and ideological issues in debates about biotechnology.

Policies for BHI

The primary goal of public policy related to BHI is to improve health. This can be achieved by:

• optimizing access to the health and quality of life benefits of BHI in a cost-effective and efficient way;
  
  
• addressing and managing the potential challenges, risks and hazards that may be associated with BHI;
  
  
• ensuring the responsible and ethical development and use of biotechnology in the health care system; and
  
  
• building and sustaining the scientific and management capacity to generate, adapt and assimilate beneficial BHI.
  
  

As with public policy in general, specific policies for BHI should reflect the value that Canadians place on justice and equity, accountability and engagement, autonomy, beneficence, respect for diversity, objectivity, caution and dignity.

CBAC's analysis examines four sectors in which policy initiatives are required to expand capacities and remove barriers to optimum performance: research and development, regulation and commercialization, technology assessment, and health system adoption.

Research and Development

In the past decade, there has been a significant increase in health research investment through the federal granting councils, research laboratories and institutes, and by various provincial agencies. Canada's per capita public funding of research in certain areas of the life sciences (e.g., genomics) compares favorably with that in other western countries. However, Canada's international position in biotechnology research cannot be taken for granted, given the priority that other industrialized nations have placed on increasing investment in this area.

CBAC calls for the development of an integrated and coherent strategy to guide sustained growth in Canada's investment in health research generally, and in biotechnological innovation in particular. The strategy should address the scale and modes of research investment required, and the need for collaboration among biological scientists, social scientists and humanists in advancing knowledge about the interaction of scientific and social dimensions of BHI. It should achieve an appropriate balance of investment between fundamental and applied research, and between investment in research involving the private sector and research on important topics that do not involve participation by the private sector.

The ethical issues involved in the research process per se are of special importance. The privacy concerns surrounding the storage and use of genetic information, the implications of advances in pharmacogenomics with respect to the design and frequency of clinical trials, and the evolving concept of what constitutes truly informed consent, will impose additional pressures on the already strained capacity of research ethics boards in universities, hospitals and research institutes.

The further development of common standards, transparent methods, national and international harmonization, and public involvement are critical for maintaining public trust and confidence in the health research enterprise. Accordingly, CBAC calls for the establishment of a body both to set standards and to accredit organizations and institutions with responsibilities for research ethics boards, and the use of databases and banks of biological specimens for research purposes.

Regulation and Commercialization

Bringing biotechnology products to market is a complex, expensive and time-consuming process that requires efficient and effective mechanisms of regulation and investment in order to facilitate Canada's competitiveness in a growing global marketplace and to provide Canadians with timely access to beneficial products.

Maintaining confidence in the regulatory system on the part of both producers and consumers requires continuing improvement in how the regulatory system functions. The growing number and complexity of biotechnology products requiring regulatory evaluation is placing intense pressure on regulatory bodies. Moreover, the development of therapeutic products targeting specific genetically defined groups may require new regulatory models. We call upon Health Canada to ensure that the regulatory regime is comprehensive (i.e., deals effectively with technologies that cross boundaries between departments or product types), efficient (i.e., meets the highest standards of timeliness in making and communicating regulatory decisions), responsive (incorporates leading edge scientific developments into its operations), and transparent (develops and publicizes standard operating procedures). Barriers to successful commercialization of BHIs in Canada include: inadequate access to early stage capital for emerging enterprises; shortage of bio-manufacturing capacity; underutilization of public institutions for product testing; inadequate linkages among institutions and businesses and among disciplines to support development of shared platforms for commercialization; and the lack of alignment of Canada's patent system with its major trading partners.

Several initiatives to strengthen the commercialization of health-related biotechnology have been proposed but relatively little progress has been made in implementing them. CBAC shares the view that in order to purposefully pursue such initiatives, and others that emerge from future technological advances, Canada requires a clearly articulated, comprehensive, coherent and integrated national health innovation and commercialization strategy that includes a specific focus on BHI.

Technology Assessment and Appraisal

Canada's current health technology assessment (HTA) systems are more fragmented, receive significantly less funding on a per capita basis than HTA systems in certain other jurisdictions, focus on a relatively narrow range of products and processes, and are insufficiently developed to cope with an increased load of BHIs requiring evaluation, and to apply the expertise needed for evaluating complex new classes of products and processes.

Sound decision-making about the adoption of new technologies by the health system requires more than traditional HTA (evaluation of the objective evidence about the efficacy of health technology and its use); it also requires what, in the U.K., has been called technology appraisal (judgment about wider issues of priority, equity, fiscal capacity, ethical acceptability, etc.), and which may be better termed “analysis of potential health system impacts and implications.” By comparison with traditional HTA, this broader approach to technology assessment is relatively poorly developed in Canada.

The Federal, Provincial and Territorial Advisory Committee on Information and Emerging Technologies has been tasked with developing a comprehensive strategy for HTA in Canada. CBAC recommends that such a strategy include extending the reach of HTA by:

• broadening the array of products and processes subjected to assessment;
  
  
• establishing a national mechanism for complementing traditional HTA with evaluation of the social, ethical, economic and health system impacts of BHIs;
  
  
• developing rapid response mechanisms to deal with emergent demands;
  
  
• establishing mechanisms for conditional approval, in special circumstances, of BHIs where complete assessments and appraisal cannot be conducted in a timely fashion, and for assessment of BHIs after they have been introduced and used in the community; and
  
  
• incorporating a communication strategy that makes the processes and results of assessments readily available to health professionals, system managers and the public.
  
  

The effectiveness of HTA depends on its results being recognized and used by decision-makers in the health system. So far, HTA has had limited impact on resource allocations in part because of inadequate linkages between HTA agencies and decision-makers.

A comprehensive strategy for HTA in Canada must also include measures to enhance its impact by:

• developing mechanisms that promote linkage and interaction between experts in HTA to experts and decision-makers in the organization, management and delivery of health services;
  
  
• establishing incentives and removing or ameliorating impediments to applying HTA in decision-making;
  
  
• establishing the tools required by decisionmakers to model the impacts of introducing BHIs on institutions or the health care system generally; and
  
  
• developing and disseminating educational materials on HTA that are suitably customized for professionals, system managers and the public respectively.
  
  

Health System Adoption of BHI

The adoption of BHIs by the health system is a complex process. Health care system managers face difficult choices in regard to the adoption and funding of BHIs because of their technical complexity and "disruptive" effects (on costs, organizational structure, professional roles) and, in some instances, because of their ethical and social implications.

Practices vary among provinces, regional health care systems, hospitals, and health care practitioners, in part, because there has been relatively little systematic study of how health technology is introduced, which would lead to identifying best practices. The challenge of making room for new technologies is compounded by impediments to discontinuing older, less effective technologies and by difficulties in estimating longer-term costs and benefits. Systematic engagement of consumers in the process of health system adoption of BHI is generally regarded as essential, but has not been pursued effectively in Canada.

There is clearly a need to learn a lot more about the adoption of BHIs, and CBAC therefore calls for a body, sponsored by or involving the federal, provincial and territorial governments and other stakeholders (such as the newly formed Health Council of Canada), to develop the foundation for improving Canada's ability to adopt beneficial BHIs by:

• identifying barriers to health system adoption of BHIs and making recommendations to remove or ameliorate them;
  
  
• promulgating goals, strategies and guidelines for implementing best practices for citizen engagement in the process of adopting BHIs; and
  
  
• giving consideration to including such practices among the attributes evaluated in institutional performance reviews and/or in accrediting health care institutions.
  
  

Foundations of Success

The development and application of BHIs must be a central element in the articulation and implementation of Canada's overall innovation strategy. Specific initiatives related to research and development, regulation and commercialization, health technology assessment and health system adoption, must be enabled by general strategies that:

• foster the collaboration among jurisdictions, sectors and disciplines necessary to realize the potential of biotechnology to contribute to improving the health of Canadians;
  
  
• respond to the urgent need to build and sustain the range and depth of scientific and managerial expertise required to generate, adopt and assimilate beneficial BHIs;
  
  
• facilitate public participation at appropriate points in the development and adoption of BHIs including fostering public education — in part through the development and communication of reliable, clear information about BHIs;
  
  
• facilitate special education of patients and providers about the clinical application of BHIs; and
  
  
• support evidence-based decision-making by ensuring that the information tools required are developed and maintained.
  
  

Successful implementation of the recommendations is likely to involve significant transformations in important institutions in Canadian society — transformations that will be influenced by the need to strike a sustainable balance among competing objectives and social values.

This publication is available upon request in alternative formats. Contact the Canadian Biotechnology Advisory Committee (CBAC) at the numbers listed below.

For additional copies of this publication, please contact:

Canadian Biotechnology Advisory Committee (CBAC)
235 Queen Street
Ottawa, Ontario K1Z 0H5

Toll free: 1 866 748-CBAC (2222)
TTY: 1 866 835-5380
Fax: (613) 946-2847
Web: www.cbac-cccb.ca
E-mail: info@cbac-cccb.ca

This publication is also available electronically on the World Wide Web at the following address:
www.cbac-cccb.ca

Permission to Reproduce
Except as otherwise specifically noted, the information in this publication may be reproduced, in part or in whole and by any means, without charge or further permission from CBAC, provided that due diligence is exercised in ensuring the accuracy of the information reproduced; that CBAC is identified as the source institution; and that the reproduction is not represented as an official version of the information reproduced, nor as having been made in affiliation with, or with the endorsement of, CBAC.

© 2005, Government of Canada (Canadian Biotechnology Advisory Committee). Biotechnology and the Health of Canadians. All rights reserved.

Cat. No.: Iu199-1/2005
ISBN: 0-662-69110-5