A recent study finds a significant logjam in the development of new drugs at the discovery and early preclinical phases. Could the creation of an open-source translational research database help solve the problem?
The traditional view of how new drugs are developed goes as follows: A university lab invents or discovers a biopharmaceutical with therapeutic potential. The academic inventors then patent their discovery and license it to a small biotech company that performs preclinical studies. The small biotech company then sublicenses the drug to a larger company that can afford to take the drug through clinical trials. What this study shows, however, is that the path from bench to bedside is rarely so linear.
The study—conducted by professors Matthew Higgins, Jerry Thursby, and Marie Thursby at Georgia Tech's Scheller College of Business—tracked how drug patents are transferred between biotech companies at the preclinical stage and how long it takes for the drugs themselves to reach the clinical-trial stage.
The researchers created a database of 835 biopharmaceutical patents. Each originated from university research and had been licensed to a biotech firm. Out of these patents, 27 percent were later licensed to a second biotech firm, suggesting they had strong commercialization potential. However, a large proportion (44 percent) of the second licenses were for testing the drug on a different category of disease instead of bringing the drug into clinical trials for the original indicated disease. While not inherently problematic, this lateral 'bench to bench' move essentially restarts the drug development timeline.
The study highlights just how long it takes for a biopharmaceutical to travel from lab to clinic. The authors found that it took an average of five and a half years for a drug to go from discovery to first license and then another three and a half years to be licensed a second time. Considering that it takes an average of 13 years for a drug to go from discovery to final approval by the U.S. Food and Drug Administration, speeding up these vital, early steps could dramatically expedite the process.
To speed the process along, the authors suggest the creation of an open-source translational research database that would store "patents and licenses for fundamental biomedical research believed to be destined for eventual therapeutic use" and which would complement the already-existing clinicaltrials.gov database. The researchers suggest that reporting to this database should be mandatory for biopharmaceutical research performed with federal funding and for any biopharmaceutical that a company hopes will be eventually approved by the FDA. The exact nature of the database is unclear, but the researchers say that this "initiative would provide clarity on which areas of research and disease indications are pursued by specific scientists and institutions and help to diminish the information asymmetries that exist in early-phase translational research."
For this idea to work, it is critically important that the database contain so-called translational research 'failures'—drugs that companies have decided not to pursue developing. Making this information available could not only prevent the unnecessary and costly duplication of experiments but also spur the testing of drugs for multiple applications earlier in the process. In the end, requiring open access to this information could save time (and federal tax dollars) and decrease the likelihood that potentially life-saving drugs stall out completely early in the development process.
This last point is key. The study found that only six percent of the first-licensed and 22 percent of the second-licensed patents had proceeded to the clinical trial stage. One need only look at the tale of Pfizer's cancer drug palbociclib to see how the current system impedes speedy drug development. This drug was discovered in 2001 but was shelved by the company for almost a decade. Thanks to a push by an academic scientist who persuaded Pfizer that the drug did have therapeutic potential, clinical trials are now underway to determine whether palbociclib prolongs the lives of women with metastatic breast cancer. If an open-access database had existed five or ten years ago, we might already know the answer.
Opinions expressed in this blog are those of the author and are not necessarily the opinions of AAAS, its officers, general members, and/or AAAS MemberCentral department or staff.