SBIR/STTR at the National Institutes of Health (2015) / Chapter Skim
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7 Insights from Case Studies and Survey Responses
7 Insights from Case Studies and Survey Responses
Pages 208-232
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From page 208... ...
Highlighting some of the details of program operation and the various roles that the SBIR/STTR program play in the development of small innovative firms, the case studies and survey comments enable a broader qualitative understanding of the program, particularly from the user's perspective, and are thus an essential part of the information gathered by the committee to assess whether the NIH SBIR/ STTR programs are meeting their legislative goals. ROLE OF CASE STUDIES Case studies are an important part of data collection for this assessment, in conjunction with other sources such as agency data, the survey, meetings with agency staff and other experts, and workshops on selected topics.
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From page 209... ...
This chapter is organized in terms of the broad types of impacts of the NIH SBIR/STTR programs: • Company impacts • Support for agency mission • Program management and company recommendations Together, these sections provide the first wide-ranging publicly available feedback of the NIH SBIR and STTR programs from program recipients. We conclude this chapter with some views on the STTR program from recipients.
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From page 210... ...
Robert Sabbadini, Lpath • "SBIR is the lifeblood of the company. SBIR funding is the only con ceivable way in which the company could have been founded and the technology perfected to the point of successful clinical trials." Dr.
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This difficulty reflects the increasing preference of venture capital firms for supporting more established companies and technologies. The case studies (and survey responses)
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This obstacle is more prevalent in even smaller markets: for example, ArmaGen is developing enzyme replacement therapies that can cross the blood-brain barrier for a pair of lysosomal storage diseases called Hurler syndrome and Hunter syndrome. Fewer than 10,000 people have these diseases in the United States.
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From page 213... ...
The result of the company and innovation has been screening of over 100,000 children for autism thus far." Filling Funding Gaps Many of the case study companies and a considerable number of survey respondents described major difficulties in raising funding before the end of clinical trials. The SBIR/STTR programs were designed to fill some of this gap, by providing funding that could be used for preclinical work, Phase 1 clinical trials, and in some cases work on Phase 2 trials.
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said that SBIR funding had a powerful validating effect for the company, underscoring its efforts to BOX 7-4 2014 Survey Responses -- Validation "The SBIR program is critical to funding early stage technology that has been both scientifically and business-wise vetted by highly skilled individuals. The ben efit to the individual companies and the society at large cannot be overstated." "Essential for our ability to attract private venture capital.
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From page 215... ...
In addition, because non-revenue status means that additional funding must somehow be acquired throughout that period, these small companies are constantly at risk of losing key staff simply through bumps in the funding timeline; a long gap can mean the elimination of key staff who may therefore not be available even if funding resumes. The SBIR/STTR programs improve the certainty of funding over a period of at least 2 years (for Phase II)
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From page 216... ...
This section draws in particular on the case studies undertaken for this report. Other examples of high-impact research can be found in the survey responses and in the success stories highlighted on the NIH SBIR/STTR website.1 The companies listed below are addressing major high-priority diseases or needs.
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Avanti Polar Lipids has a new treatment for cystic fibrosis in clinical trials. Innovative Technologies and Product Development For the majority of SBIR/STTR recipients, the program supports work on the company's core technology.
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. The SBIR program is vital for innovation in this field and should be expanded for its own value without sacrificing other federal research programs." to work on a platform technology that could have applications to several different kinds of cancer, and he observed that this profile did not match the requirements of venture capital firms.
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• ArmaGen is developing enzyme replacement therapies that can cross the blood-brain barrier for a pair of lysosomal storage diseases called Hurler syndrome and Hunter syndrome; fewer than 10,000 people have these diseases in the United States. If successful, ArmaGen plans to apply its technology to other diseases with larger markets.
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From page 220... ...
This is challenging because of the very long period before revenue starts and because the core funders of biomedical research aside from the government (strategic partners, usually from large pharmaceutical companies, and venture capital investors) are increasingly reluctant to fund projects that are not well along the path to market.
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From page 221... ...
allows for obtaining funds for development of innovative technologies with the pace that is not artificially skewed by short-term commercial interests." "This program allowed our core technology to incubate for a period of time that allowed us to advance the technology to the point where strategic partners became interested. Without SBIR funding we could not have gotten the technology to this point and the company would have likely failed." BOX 7-8 2014 Survey -- Comments on Collaboration "We would never have been able to develop this product, conduct the neces sary clinical trials, solidify a strategic partnership, and get it to commercialization without SBIR funding." "The timeliness of the [SBIR]
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• Sanaria is undertaking clinical trials with a wide range of partners in the United States, Africa, Asia, and Europe, including a number of different stakeholders: African governments, Marathon Oil, nonprofit foundations, universities and research labs, and private companies. Sanaria recently signed a path-breaking agreement with Marathon Oil and the government of Equatorial Guinea to completely fund clinical trials through Phase III through $48 million in support.
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From page 223... ...
noted that the alignment between topics and awards has changed significantly over the past 10 years. During her early years with the program, she was confident that a strong project would receive consideration and perhaps funding regardless of its connection to a topic described in the Omnibus Solicitation.
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Another CEO contacted in the course of this study observed that the predominance of academics on study sections is unfortunate, in particular because they tend to take a narrow view when defining innovation. They tend to lower scores of projects that they see as insufficiently innovative, perhaps as compared with viewing them as an NIH Research Project Grant Program (RO1)
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. Review panel members should be educated on the purpose of Phase IIBs before the review panel and proposal reviews take place." "Give proposal reviewer fewer proposals to review so they can do a better job.
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Alongside the general concerns noted above, survey respondents had particular concerns about the gap between Phase I and Phase II. This gap is also addressed in Chapter 5, where survey data reveals it as a persistent problem.
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This has taken more than 12 months in our case. Reduce administrative delays in funding release each year during phase II.
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said that the NCI Bridge program is important given the difficulties in funding clinical trials but that it could be improved. In his experience, neither venture capital firms nor strategic investors believe that the program met their needs.
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suggested that NIH continue to support investigator-initiated proposals, which is a hallmark of the NIH program but is being steadily eroded by a push toward programs defined in advance by the Centers. Support for Working with the Food and Drug Administration Many survey respondents highlighted their difficulties in working with FDA to receive approval for required clinical trials.
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and NIH reviewers are typi cally looking at extremely innovative solutions, which FDA does not look favorably upon. There needs to be a better connection between the two." "FDA process is very uncertain and heavily dependent on the particular re viewers assigned to the application.
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noted that Phase IIB does not fund any commercial or marketing personnel, but these are absolutely necessary for a commercial venture capital, which is what Phase IIB is designed to help fund. He suggested that either NIH or Congress should consider changing these limitations to permit a more realistic approach, in which a limited percentage of Phase IIB funding (perhaps 30 percent)
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said that the STTR program was particularly important for her company. Once Stratatech was established as a functioning company, and the basic research was completed, other sources of funding became more available.
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