Panel: On the horizon: R&D renaissance or reboot?
On the horizon: R&D renaissance or reboot?
Rising costs, data deficiencies hinder research and development
Records were set last year for the total number of new drug approvals and for new drugs to treat rare diseases. The output provided additional options to patients and prompted optimistic forecasts for the biopharmaceutical industry. Yet rising development costs, stricter post-marketing requirements, and tightening access to approved medicines reflect a new reality, too.
With that backdrop, moderator Robert Califf, deputy commissioner for medical products and tobacco at the U.S. Food and Drug Administration (FDA), asked a panel of researchers and strategists at Partnering for Cures to share their perspectives on whether the increase in approvals means development is, indeed, becoming cheaper and faster. While the overwhelming response was a tepid "no," a tone of cautious optimism flavored the discussion. Teri Lawver, global commercial strategy leader and global vice president, cardiovascular and metabolism at Janssen Pharmaceutical Companies of Johnson & Johnson, identified three main factors that drive a drug development landscape that, in her view, has become more complicated: macroeconomics that require products that meet regulatory as well as payer needs; diseases that require large, lengthy clinical trials that further complicate development while driving up costs; and the variability of requirements imposed by regulatory bodies.
Max Hodak, co-founder of Transcriptic, said the increased complexity and cost of research and development suggest that "something is lacking on the discovery side." He explained that there is an overall poor understanding of the underlying biology for many diseases, a situation that will inevitably lead to clinical trial failures.
Although there is much to be done in basic science, major advancements have been made. As Kenneth Buetow, director of the computational science and informatics core program in the Complex Adaptive Systems Initiative at Arizona State University, pointed out, the biomedical research enterprise is able to take on whole new classes of issues that were inconceivable in the past. To build on that point, Clifford Hudis, chief of the breast medicine service, vice president for government relations and chief advocacy officer at Memorial Sloan Kettering Cancer Center, mentioned that doctors and researchers are better able to segregate disease. For example, the view that cancer is not just one disease but a collection of diseases unified by common molecular pathways is now commonplace. However, it is unclear whether this knowledge actually improves a patient's quality of life. While we have made strides and are heading in the right direction, we are not quite there yet, Hudis said.
Michael Rosenblatt, executive vice president and chief medical officer at Merck, expressed unqualified optimism with the progress indicated by the record number of drugs approvals by the FDA last year. However, Rosenblatt asserted that "we won't go forward unless we can reproduce data." Data reproducibility is a major issue in R&D and is absolutely necessary for successful drug development. Further, data reproducibility has ramifications across the whole spectrum of science, from basic to clinical research, as Hudis pointed out, and the associated opportunity cost is high. Because only 20 percent to 30 percent of the molecules that undergo Phase II clinical trials yield a treatment, it is imperative to focus on studies that have the highest potential, and this hinges on reproducible data.
So the question remains, what would be needed to transform the current R&D system? Califf posed this question to the panel, and the responses coalesced around harnessing big data to inform and enrich R&D. Hudis suggested that it may be time to consider blurring the line between "on study" and "off study" to gather as much data as possible. Only 3 percent of the population participates in clinical trials, meaning that we essentially "do not learn from 97 percent of the population," which Hudis said is unacceptable. Consumer companies such as Amazon and Netflix passively collect data from a much larger swath of the population -- an enviable goal for biomedicine. Buetow said there is a need to achieve "the full engagement of all the data that needs to be considered," including the clinical context of the data and the patient's perspective.
As the panel discussion came to a close, the positive frustration with the R&D ecosystem highlighted the fact that we are near a tipping point: On the preclinical side, there is a need for more data sharing and a culture change around sharing and reproducibility; and on the clinical side, more data sharing and a culture change around patient centricity are required to move the needle. The incredible promise of mobile technology platforms is helping to reshape the possibility of bold, modern clinical trial designs. However, if we can do one thing differently, Lawver stated, it is to "put the patients at the center."