The U.S. National Cancer Institute (NCI) has been in the business of granting awards for oncology-related products for two decades, and the agency recently published a report touting some significant success stories. However, NCI managers said on a Feb. 13 conference call that staff at the agency may soon be working informally with the FDA on behalf of grantees in an effort to help grantees navigate the regulatory waters, a development that could foster faster times to market for a variety of therapeutic and diagnostic products in the oncology space.

The National Cancer Advisory Board had met Feb. 12 to present recommendations for strengthening the Small Business Innovation Research (SBIR) program, and the SBIR development center had commissioned an external pilot evaluation of the SBIR Small Business Technology Transfer (STTR) programs in 2018. The surveyed awards were restricted to those granted between 1998 and 2010, a cut-off point necessitated by the lag time needed for a grant to lead to a product that survives to commercialization.

For that time frame, the STTR generated $9.1 billion in total sales of products and services, and had a total economic output of $26.1 billion. These sums were associated with nearly 370 awards with sales, royalties and follow-on R&D funding. Also among the metrics was $2.9 billion in new tax revenues at all levels of government, along with $8.1 billion in labor income. The program is estimated to have generated nearly 108,000 new jobs as well.

The SBIR portfolio runs to about 43 percent for pharmaceutical/biotech therapeutics, while another 16 percent consists of in vitro diagnostics. Following that is 14 percent for oncology imaging, and 11 percent for devices for cancer therapy. The list is rounded out by research into cancer biology (8 percent) and cancer control and epidemiology (also 8 percent).

Mel Billingsly, chair of the ad hoc working group on SBIR, said the advisory board had drafted a set of recommendations for bolstering the program, and noted that the ad hoc group was a good cross section of researchers, venture capitalists and several successful companies from across the life science spectrum. Among the imperatives discussed during the meeting were finding ways to encourage translation of academic research into clinical practice, and how to better integrate the SBIR program within NCI intramural programs. Among the priority goals are to boost the size of phase I awards, and to provide supplemental funding to companies that are close to meeting their development milestones.

Program metrics may include time to award, which is now about seven months in some instances, but Billingsly said there is also an interest in a "phase zero" grant for high-risk/high reward applications that are in very limited stages of development. However, Billingsly said also that the administrators of the SBIR program seek to develop interactions with the FDA to create a regulatory assistance program.

FDA interaction to be informal

Michael Weingarten, director of the SBIR development center, said the FDA interaction proposal "is more about working with the FDA informally on behalf of our small businesses." He said most of the startups involved in SBIR programs have been in existence for only a couple of years, and might have no more than five employees total and no regulatory staff. Weingarten said among the ideas being floated is one to include the use of educational webinars on the premarket review process. However, Weingarten said the NCI staff can also serve as a liaison to the FDA, and provide startups with responses from the FDA on regulatory questions, although those would be informal responses. He said the objective is also to alert SBIR grantees to key FDA guidances, adding, "NCI is going to be a go-between to facilitate a lot these interactions."

Weingarten said one example of a company that benefitted from the SBIR grants is San Diego-based Illumina Inc., which developed some of its early technologies for genotyping via SBIR grants. He said Naviscan PET Systems, also of San Diego, received an award in the mid-1990s and was able to commercialize three devices based on SBIR awards. The first was a high-resolution PET scanner for breast imaging, the first such system to market. Weingarten noted that this system could identify lesions the size of a grain of rice, a novelty at the time. Naviscan has also relied on SBIR programs for a PET mammography-guided biopsy system for lesion removal, and a gamma medical lumagen technology, the combination of which is "really making a difference for women with dense breasts."

Weingarten said the SBIR administrators do not generally prioritize one therapeutic or diagnostic area over another across the board, but said they do try to identify areas with serious needs. He said President Trump had mentioned putting $500 million into NCI for pediatric cancers during the latest State of the Union address, adding, "that would be an area where we are looking to invest."

Billingsly said the SBIR funds are set aside statutorily, but that there is no mandate for a certain ratio of economic return. He said the program had generated nearly $3 billion in tax revenues on an investment of $780 million, adding that $2 billion of those tax receipts ended up in the Federal Treasury. He said that the average salary for SBIR-generated jobs is in the neighborhood of $75,000.

Of the nearly 700 projects financed to a conclusion under the SBIR program, nearly 300 have led to commercial success, and Billingsly said that is a significantly higher rate of success than is seen in the venture community, which he said is closer to 1 in 10. He noted that the recession that followed the housing crash of 2008 put a lot of small companies in a bad pinch and that the SBIR program was a lifeline for many startups.

2009 'a nuclear winter'

Weingarten chimed in, noting that 2009 "was kind of a nuclear winter for a lot of companies trying to raise venture capital." A lot of small companies at that time were running into the dreaded "valley-of-death problem," and he said the SBIR's phase II bridge program kept a number of small entities alive long enough to get into the clinic and generate some private-sector financing. Weingarten said the expectation is that these applicants will raise matching funds from private sources, and that the overall rate of private-sector investment to public-sector investment over the life of the program has been closer to 4:1.