May 18 - 19, 2015
Making sense of clinical translation: ethical, regulatory and policy challenges for Europe and the US.
- Aarden Erik, University of Vienna, PhD, EU Marie Curie Fellow
- Holloway Dustin, Harvard Medical School, PhD; Research Associate in Systems Biology; Program Manager for the Harvard Program in Therapeutic Science
- Blasimme Alessandro, INSERM (UMR1027 INSERM - Université Paul Sabatier, Faculty of Medicine - Toulouse, France), Harvard (Fulbright Fellow at Harvard Kennedy School, STS program), MA; PhD; Fulbright Fellow; Postdoctoral Researcher
- Marelli Luca, European School of Molecular Medicine (SEMM), European Institute of Oncology (IEO) and University of Milan; John F. Kennedy School of Government, Harvard University, PhD student in Foundations & Ethics of the Life Sciences (European School of Molecular Medicine and University of Milan), Visiting Research Fellow, Program in Science, Technology, and Society, Harvard Kennedy School
Ever more frequently, innovation policies in biomedicine are driven by the idea that a large and growing volume of biological knowledge is being used to poor effect, as evidenced by the stagnating number of new therapeutic products being introduced on the market (1–4). We label this representation of biomedical innovation the “translational lag narrative”. This narrative presents markedly normative overtones, as it depicts the acceleration of clinical translation as a moral and societal imperative, and consequently, as a political priority (5–9).
We will convene an interdisciplinary panel of scholars animated by a common recognition that, as the translational turn in biomedicine carries forward the re-negotiation of values such as fairness, equality, welfare, vulnerability and risk, these issues have to be brought to the surface and thoroughly analyzed.
Background and Significance
Ever more frequently, innovation policies in biomedicine are driven by the idea that a large and growing volume of biological knowledge is being used to poor effect, as evidenced by the stagnating number of new therapeutic products being introduced on the market (1–4). We label this representation of biomedical innovation the “translational lag narrative”.
This narrative presents markedly normative overtones, as it depicts the acceleration of clinical translation as a moral and societal imperative, and consequently, as a political priority (5–9).
The beneficial consequences of fostering bench-to-bedside research are taken to be self-evident. However, important issues loom large on the horizon of clinical translation in terms of fair allocation of public resources, equality in access to health care, and the ethical assessment of new risks for participants in innovative clinical research. So far, those questions and their causal relation to the idea of the translational lag have largely been evaded.
To address this gap, instead of taking the translational lag for granted, we propose to make sense of it as a potent discursive device that is able to align the interests and the practices of decision-makers, regulators, industry, scientists, patients and wider publics.
For instance, little is known as to the impact of innovative therapies on public health systems and on citizen’s access to cures. Evidence suggests, however, that new drugs will likely be exceedingly expensive and thus arguably remain accessible to few, economically privileged patients – as in the case of the recently approved anticancer drug Imbruvica (23). Moreover, the threshold of risk for medical research is being lowered to permit patients’ enrollment in clinical trials (24). So, while major regulatory changes and huge investments are underway to harness the translational potential of science (for instance in domains such as personalized medicine, genomics, regenerative medicine) more should be done to anticipate the ethical and political stakes of the translational enterprise. A first fundamental move in this direction is to abandon a self-limiting, linear account of biomedical innovation and map the terrain by investigating the ethical and political assumptions that sustain the translational lag narrative. To do so we will convene an interdisciplinary panel of scholars animated by a common recognition that, as the translational turn in biomedicine carries forward the re-negotiation of values such as fairness, equality, welfare, vulnerability and risk, these issues have to be brought to the surface and thoroughly analyzed.
Specific themes of the Workshop:
The rhetorical dominance of the translational lag narrative in biomedical science policy in most industrially advanced countries is widely accepted and generally unquestioned. As a consequence, the idea of such a lag or the necessity of repairing it is not further interrogated, nor made the object of in-depth critical appraisal.
Quite to the contrary, we believe that, given their possible impact on society and public health, new policy and regulatory approaches to the perceived translational lag in biomedicine call for dedicated scholarly analysis, as do the assumptions underlying the idea of ‘translation’ and its associated socio-legal infrastructures. In this workshop, we seek to develop resources for a more profound and situated analysis of translation and its policies. To do so, the workshop will focus on three distinct but deeply interconnected aspects of the translational turn:
1. Accelerating translation
Current emphasis on translation can be seen in the recent consolidation of innovative funding structures (10–13) aimed at maximizing the synergic impact of public and private resources, and also in the proliferation of innovative forms of organization (14) (e.g., supply−side policies such as translational institutes, and an emphasis of contract as opposed to grant-based science). Moreover new regulatory instruments are being designed to overcome the perceived slowness of the current clinical trial pipeline so as to accelerate and to expand individual patients’ access to new therapies, diagnostics and drugs (15–22) (demand−side policies). Regrettably, little has been done to systematically analyze how innovation policies aimed at accelerating bench-to-bedside translation remodel health priorities and rescale the nature of public oversight on clinical development.
2. Re-imagining national priorities
Despite the abundance of documentary sources, the reorganization of national priorities, vis-à-vis new promising areas of biomedicine (e.g. personalized medicine, genomics, regenerative medicine), has not been sufficiently described. Moreover, the assumptions of translational science policy as to fairness and equality in health care have so far remained regrettably under-theorized (25,26). In particular, no specific scholarly attention has been devoted to a comparative analysis aimed at uncovering the ways by which change in regulatory cultures and organizational arrangements plays out differently in distinct national contexts with respect to collectively held representations of national futures. Analyzing these phenomena will unravel tacit ethical and political implications and provide insights into policy decisions that are better equipped to serve society’s needs (27).
3. Expanding access / remaking risk
Even though the demarcation between “patient” and “research subject” is embedded in contemporary medicine and medical ethics, the reconfiguration of clinical translation is blurring this distinction (31). As state agencies embrace the translational lag narrative, they set up new standards of clinical evidence and risk assessment. In particular, regulatory instruments such as adaptive clinical trials for new drugs (16), compulsory licensing (28) for expensive therapies, exemplify how considerations about risk and equality are called into play in the redefinition of access policies to experimental or new drugs. Relevant examples include genomics, where risks flow through patients to family and community members, and orphan drugs, where the evidentiary standards of clinical trials are purposely weakened to accelerate translation. Such examples, and many others, testify to the mutually productive relationship between science and social order and should be analyzed accordingly (29,30).
Our interdisciplinary approach
Science and Technology Studies (STS) represents a particularly promising analytical platform to tackle the above phenomena and stands to advance our critical understanding of the sources and consequences of the translational turn in the life sciences. The cultural and institutional conditions, as well as the ethical and political assumptions that sustain the translational turn are precisely the subject matter most suited to an STS methodology. The applicant, the co-applicant and the majority of the participants – despite coming form a range of different disciplines – received advanced training in STS and will thus be able to structure the workshop around the most fruitful analytical resources developed in this domain. In particular the framework of bioconstitutionalism (24) will provide avenues for understanding the mutual relationships between scientific advances and their socio-legal preconditions and implications. Analogously, the notion of socio-technical imaginaries (26) offers a methodologically sound way to connect changes in science policy to wider national and international political transformations. These two core analytic tools will guide our investigation of the assumptions and implications embedded in the translational turn in biomedicine.
At the same time, the interdisciplinary inclinations of STS provide a fruitful point of departure for having wider conversations across various relevant disciplines. For two and a half days, our workshop will bring together a mid-sized group of international scholars of different disciplinary backgrounds, sharing a common interest and specific expertise in the ethical, regulatory and political study of biomedicine. We plan to invite scholars from disciplines such as molecular medicine, bioinformatics, regulatory science, law, policy and medical ethics to join an STS-informed conversation that will generate a new critical appraisal of the impact of innovation politics in the ever-expanding domain of the life sciences. Our ideal mix of attendees includes representatives from academia, industry, and government (e.g., the US Food and Drug Administration and the European Medicines Agency). We will take particular advantage of Fondation Brocher’s proximity to the World Health Organization, the UN High Commissioner for Human Rights, and the World Intellectual Property Organization to include and engage individuals from those institutions.
In this way we will be pulling together the necessary critical resources to make sense of current ethical, regulatory and political changes at the translational frontier of biomedicine, while at the same time assuring the cohesive interpretive thread that is needed for advancing our understanding of this crucial phenomenon in today’s life sciences.