Evidence Gathering
by Adam Istas
October 2007
With the cooperation of pharma and academia, investigator-initiated sponsored research facilitates innovation.
A mainstay of drug research in academic institutions, investigator-initiated trials (IITs)—or investigator-sponsored research (ISR)—can provide valuable information regarding a new or existing therapeutic. From further indications to product line extensions, pharmaceutical manufacturers with formal IIT programs have the potential to reap the rewards of this nominal investment for years to come. IITs are not without significant risk, however, and companies choosing to provide this support must exercise due diligence when selecting research programs.
The pharmaceutical industry supports IITs to enhance the understanding of a specific drug or therapeutic area of interest, says Daniel Pardi, manager of medical affairs, investigator-initiated trials and medical communications at Jazz Pharmaceuticals, in Palo Alto, Calif., USA. Additionally, supported IITs may also serve as proof-of-concept projects in new therapeutic areas. After the research is conducted and analyzed, if a positive therapeutic signal is identified, some companies will then evaluate the feasibility and potential of a full clinical program designed for regulatory registration, Pardi says.
Although IITs can include programs in basic science and non-clinical work, most large pharmaceutical companies choose to support post-marketing research, says Steve Shinall, Ph.D., manager of external grants at Takeda Pharmaceuticals, in Deerfield, Ill., USA. Shinall is quick to suggest that investigator-initiated sponsored research should not be considered a substitute for an actual post-marketing clinical development plan, but a well-run IIT can certainly augment the work that a company is already doing. “ISRs primarily serve to improve our understanding and awareness of a product,” he says. “And that can be a win-win for everybody.” But well before the research begins and any new knowledge is gained, a considerable amount of front-end work must occur, including contract negotiations with the institution and setting the investigator’s expectations.
Quid Pro Quo
Companies are more likely to support proposals from academic investigators at prestigious universities who have a proven track record for high-quality research. Working with universities can be challenging as contract negotiations can often significantly delay the approved project from initiating. If an investigator has been part of company-sponsored, multi-center trials for a Phase III program only, for example, he may be accustomed to getting trained on a protocol then getting started soon afterwards. As the chairman of a recent IIT conference, Pardi surveyed the attendees and found that, on average, the time from concept proposal to research initiation is seven to eight months. “Sharing average process timelines with new investigators has been very helpful,” he says.
“A hot topic for the industry right now is overhead costs,” Pardi says. Academia and industry often disagree about who is responsible for these charges. “The academics will say that these are necessary costs that support the facilities where the research is conducted, and industry will say that they want to support the research, but they don’t necessarily want to pay the gardening bill,” he says. A growing trend, Pardi notes, is to establish limits as to the percent of overhead that companies will support.
Another hot-button issue with regard to investigator-initiated sponsored research is intellectual property. “I think institutions are becoming much more sophisticated in their need to include intellectual property protection as part of the negotiation process,” Shinall says. “And companies have a vested interest in maintaining their intellectual property protection as well.” The goal of an ISR program is to represent an area where both sides agree to work together and to come to a compromise, he says. “I believe that if the research results in a patentable discovery, then it should belong to the investigator. However, the manufacturer should have a favorable negotiating position to license and otherwise develop whatever results,” he says. For the protection and comfort of all parties, all of these types of agreements must be worked out well in advance, Shinall adds.
Process Communication
If an investigator has done many industry-sponsored trials, he may be accustomed to getting started rather quickly and his expectations may be skewed, Pardi says. “So the first thing we try to do is set investigator expectations.” The process at Jazz Pharmaceuticals for communicating with investigators is well mapped out, he says, and the company provides investigators with realistic timelines for each step, as well as a start-to-finish road map of the research to be done and the roles and responsibilities of both the company and the investigator. “We want [investigators] to have a solid understanding of what they need to do to be successful,” Pardi says.
Once the research is under way, a typical agreement calls for regular updates from the investigator on the mutually agreed upon milestones of the study. In almost all cases, payments to the institution are tied to these milestones, Shinall says. Delivery of data also should be spelled out in the contract and agreed upon by both parties. “A fundamental expectation is that the research will lead the investigator to present or publish the data, so in addition to a standard final study report, a copy of any manuscript or abstract resulting from the work should be submitted by the sponsor-investigator, as a matter of courtesy.”
Shinall suggests the future of investigator-initiated sponsored research will depend on the extent to which pharmaceutical companies and academic centers can cooperate with respect to intellectual property concerns. “If this cooperation begins to unravel, then ISRs will be seen as less advantageous and more of a headache to companies, and the number of ISR programs will start to decline,” he says. “But if that mutually beneficial relationship can be emphasized, then ISRs represent a great way for companies—with a modest amount of financing—to facilitate some really innovative research that can have the potential to identify new indications and broaden the use of a compound.” Shinall says this type of innovative research can provide a justification for companies to draft a true clinical development program for the studies compound. “That’s the hope. And if that continues to happen, then I think ISRs will continue to play a large role.”
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