As the explosion in biomedical research increases the potential to cure a wide range of diseases, a growing number of private foundations and other charities now provide money to nonprofits for converting scientific discoveries into patient therapies. The National Institutes of Health is also expanding its support for applied medical research by investing $700-million a year in a new center dedicated to this purpose.
Given the budget pressures faced by nonprofits and government agencies, it is crucial that this money be used effectively. Yet a recent survey of large research organizations found no consensus on how to judge the success of grants for what is known in the scientific world as “translational medical research.”
Because of the way science works, it is not realistic to judge such research on whether it produced a cure for a major disease. Such a breakthrough could be more a result of good luck than effective management. On the other hand, it is not enough for scientists to say: “We are smart and hard-working, so continue to give us large research grants.”
To better assess performance in converting scientific discoveries to human therapies, research organizations need to articulate clear goals and establish specific measures to gauge their progress. Without such a performance-assessment system, grant makers do not have a good way to determine which organizations should get more grants in applied medical research. We could spend literally billions of dollars and make very little headway toward new patient therapies.
Here are the seven key questions grant makers should ask about the performance of organizations trying to apply medical research.
How much money is flowing to the organization? A group that has attracted a lot of money has increased odds of making a breakthrough. An even better sign is its ability to attract second and third grants from the same donor. That typically happens only when an organization is achieving key milestones on time.
Diversity of financing is a helpful measure; groups that get money from a wide array of sources are unlikely to be forced to cancel or delay a project. Also important is how much money goes into administrative overhead versus scientific research: the lower the percentage of overhead, the better.
Does the organization attract top scientific talent? Talent is a virtuous cycle: By recruiting the most talented researchers, a group is more likely to generate new patient therapies. And as it shows fruitful results, it is better able to attract talented researchers.
Check out not just the number and qualifications of a group’s researchers but also the share of people who accept offers to join the research team: A high percentage shows it is attractive to talented scientists. In addition, find out whether it is willing to dismiss researchers who do not produce results in a reasonable amount of time.
What is the institution’s pipeline for innovation? Determine the number of new projects the group generates and its speed of progress toward meeting milestones. Beyond these quantitative measures, try to assess the quality of these new projects. Did they lead to clinical trials that would not be otherwise conducted?
At the same time, look at the tools developed by an institution, such as biological indicators and databases. Although tools do not directly improve patient care, they do so indirectly by aiding other research groups in developing diagnostics or therapies.
What do scientific peers think about the organization? Examine how many of the scientists at the institution are publishing articles in journals that undergo peer review. In particular, see how frequently their articles are cited in other well-regarded publications.
Find out how many patents the researchers have obtained. That reflects a government examiner’s determination that the discovery is truly new and not obvious to other experts in the field. Furthermore, patent protection enhances the value of research by increasing its commercial potential. If patented research leads to a new therapy, the commercial sponsor can keep other companies from copycatting.
Do outsiders make use of the group’s research? If companies, venture philanthropists, or others are investing in the findings of a research group, that is a very positive sign. Such investments move the findings through the many steps needed to obtain regulatory approval.
Most research organizations lack the resources or expertise to secure regulatory approval of new drugs or medical devices. When they can attract partners to take a scientific finding to the next level, such as clinical trials, they are probably high achievers.
Does the organization share with others? Look at the extent to which it shares its methods and findings with other research institutions and the public. A good way to measure a group’s educational efforts is to check its Web site. See how many conferences it holds or newsletters it has created. Also ask if it is willing to license patents to other research institutions on reasonable terms.
Information sharing may be the key objective of a foundation’s grant: for example, letting doctors and the public know the early risk factors for a disease can be critical in saving people’s lives. Information sharing has other goals too: Aside from educating people about a disease, it can prompt donors to give more for further research.
Are scientists at the organization good collaborators? Many characteristics of academe inhibit collaboration between basic research and clinical practice, such as the lack of multidisciplinary training, parochial criteria for promoting scholars, and physical separation of facilities. Translating basic research into useful therapies is inherently multidisciplinary.
If a program is successful, it should break down barriers by promoting interaction between basic researchers and clinicians. Grant makers can measure collaboration by looking at how often scientists share data or equipment or whether they write papers or seek grants together.
In the past, few grant makers have set precise standards for what effectiveness means in applied medical research. As grant makers pour more money into such research, they should ask applicants for aid all seven questions. To improve a research organization’s performance, the grant maker may need to pay for independent reviews—including scientists, clinicians, and industry experts. By providing an outside perspective, such peer reviews help the organization evaluate whether to continue or change its approach to devising new therapies.
No matter how revolutionary the biomedical research under way today may seem, it won’t matter to patients unless it results in new therapies. So grant makers and scientists need to work together to find the most effective ways to develop promising cures. The stakes are too high to squander scarce resources.