Diffusion and Use of Genomic Innovations in Health and Medicine Workshop Summary (2008) / Chapter Skim
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2 Translation of Innovations
2 Translation of Innovations
Pages 3-24
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From page 3... ...
, the process of translating these discoveries almost always begins in the medical products industry, where a basic discovery is followed up with a period of specifically directed preclinical activity intended to test whether the putative therapeutic target is indeed viable. The next step is determined by a decision-making process that comprises multiple steps and includes assessments that link financial support with the probability of success; if the decision is to move forward, then the next stage of development is undertaken by clinical research orga
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Clinical Translation Global Discovery Translation Research and Adoption Health FDA Practice Global Community Preclinical Approval Adoption, Improve Health: Proof of Clinical Assessment Basic Research; PK/PD/ Practice Community Service/ Concept Development •Care delivery Discovery In Viv o To xicology Evidence Guidelines, Health Research, in Man Phase II, III •Outcomes Analysis Based Cost Status Population/ •Economics Medicine Effective Publication Current State Entities AHS/Industry/ Clinical Research HCS/Hospitals/ Government Industry/Biotech Biotech Organizations/AHS Practices/FQHC/AHS NGOs Fragmented/Disorganized Health Service Proof of Guidelines Community Global Health Basic Preclinical PD/PK/ Phase II, III and Concept And Health Research and Discovery Research Toxicology and IV Outcomes in Man Adoption Trends Service Research Current Timeline 25-30 Years FIGURE 2-1 Translation of innovations. SOURCE: Califf, 2007.
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From page 5... ...
and other national and international regulatory bodies to make decisions -- based on criteria that vary depending on which division of the FDA is involved or which country is doing the evaluation -- about whether the therapy is ready to be introduced into clinical use. After a therapy is approved, it is supposed to reach the appropriate people in the approved manner through a competitive system that includes health systems, hospitals, clinical practices, purchasers, and sales representatives for the product or technology.
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SOURCE: Adapted from Harper et2-2.eps al., 2007. instance, although novel targets afford bigger potential returns on investment, investors often shy away from them because of the risks entailed.
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There is a great disincentive, however, to providing low-cost, efficient health care to the people who are experiencing most of the death and disability in the United States. Despite astounding advances in biology, ensuring that innovations reach those members of society who stand to benefit most from them -- and thus that these innovations will have the largest possible effect on the rates of death and disability -- is proving especially difficult.
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The Critical Path Initiative This lack of data about the factors that underlie the success or failure of development efforts is a major motivating factor for the FDA's Critical Path Initiative, which aims to create a "safe haven" for sharing knowledge that can accelerate translation while at the same time doing nothing to impair the drive for competitive advantage that stimulates creativity in our system. The concepts of pre-competitive and pro-competitive spaces are key to understanding the strategy underlying the Critical Path Initiative.
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From page 9... ...
Such exorbitant costs become an inhibiting factor for therapeutic areas that require definitive data as a precondition to marketing. The FDA Critical Path Initiative is seeking to transform the clinical research enterprise through the Clinical Trials Transformation Initiative.
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As is the case in the United States, fi nancial incentives in many other countries emphasize practices that focus on expensive technology that benefits "paying customers," while incentives to provide basic health services receive less emphasis even as the understanding of ways to meet those basic health needs improves. In Durham, North Carolina, with funding from the NIH's Clinical and Translational Science Awards, a study is underway whose goal is to develop a deeper understanding of the issues surrounding the delivery of basic health needs.
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Benjamin and colleagues performed a meta-analysis of clinical trials completed for pediatric exclusivity in order to quantify the dissemination of study results (Benjamin et al., 2006)
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Clay Christensen examined the role of innovation in the computer disk-drive industry and put forth some ideas that have relevance to a discussion on innovation in health care. He describes the process of innovation as resulting from entry of new firms and new business models in the marketplace.
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TRANSLATION OF INNOVATIONS 13 $US $400 Year (billions) BBA 1990 $110.9 $350 Medicare Spending 1997 1995 $184.2 1996 $200.3 $300 1997 $213.6 $250 1998 $213.4 Accelerated 1999 $213.0 $200 compared to 2000 $221.8 pre-BBA $150 2001 $244.8 2002 $265.7 $100 BBA Reform 2003 $280.8 1999/2000 2004 $308.9 $50 2005 $336.4 | | | | | | 1985 1990 1995 2000 2005 2010 FIGURE 2-3 Policy response: A budget freeze.
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From page 14... ...
Since the policy goal is to improve quality and reduce costs, an implicit policy goal should be to encourage disruptive innovation and market entry to achieve this goal. In practice, however, current medical reimbursement strategies reward the sustaining innovators with premiums, making it potentially very difficult for disruptive innovators to enter the health care market.
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From page 15... ...
These are generally firm responses to competitive threats from market entry and this p rocess is how originator firms respond to disruptive innovation. Firms can respond at several levels to new product or service creation, and some can adapt their business model to a new market environment over time.
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As stated earlier, there is an urgent need to better understand the relationship between incentives and market entry in order to foster technology innovation. Lessons for Genomics from Other Technologies Annetine Gelijns, Ph.D. Columbia University Advances in genetics have led to a remarkably improved understanding of the genetic and molecular basis of disease, and these advances are increasingly leading to the development of new interventions in such areas as genetic testing, gene-based therapy, and pharmacogenomics.
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Finally, various economic, sociocultural, and ethical factors powerfully shape the diffusion process as well. In the diffusion literature a technology is generally perceived as being static or constant; however, innovations continue to evolve as they enter clinical practice.
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. The clinical utility of such tests, however, will need to be confirmed in pragmatic clinical trials involving large, well-defined populations.
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Uncertainty about new test interpretations may affect the adoption decisions of health care providers. For example, the AlloMap molecular expression test was developed to detect acute cellular rejection in hearttransplant patients.
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Diffusion depends not only on the benefits that a new intervention provides, but also, importantly, on the institutional environment in which a technology is imbedded. Patients, consumers, and physicians need to understand what to do with new probabilistic risk information; the FDA must decide how to deal with genetic diagnostic tests and how best to regulate diagnostic drug combinations; insurers need to gain comfort with the interpretation of cost-effectiveness analyses of emerging novel genomic technologies; and, finally, the larger policy world will need to deal with privacy and confidentiality issues and the potential for discrimination.
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From page 21... ...
Furthermore, the system prevents open exchange of information and creates many barriers to communication among affected parties -- for example, between FDA and vendors or between those manufacturing or creating new devices or drugs and those who will be using them. This is the sort of situation described by the mathematician John Nash nearly 60 years ago -- that is, that the optimal good is almost never achieved by the individual players optimizing their own individual results without being able to fully discuss how to jointly optimize the system (Nash, 1950)
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From page 22... ...
For anything else, finding investors is a problem because the market is smaller. Gelijns said that one of the important issues is that the premarketing trials often focus on the accuracy of a test and that the ultimate clinical utility of these tests frequently emerges in the post-marketing setting.
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Schulman responded that, intellectually, the use of clinical-practice guidelines for genomic innovations is an exciting area. There is still relatively little clinical information available, however, so how these guidelines will fit into the marketplace is uncertain.
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From page 24... ...
This example illustrates the fact that if there is a competitive situation and market share is at stake, a huge incentive exists to make the investment needed to implement the test, even if there is no reimbursement for the test. It might even be considered unethical to give the drug without testing.
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