In the span of nearly a year, Mayo Clinic’s use of IBM’s Watson computing system for matching patients to clinical trials helped increase enrollment by an average of 80 percent in studies of systemic therapies for breast cancer. Over 11 months, the time needed to screen an individual patient for clinical trial matches also fell when compared to traditional manual methods, they said in a joint press release. In July 2016, Mayo began using the system with a team of screening clinical research coordinators in its ambulatory practice for patients with breast cancer. “This has enabled all patients to be screened for all available clinical trial opportunities,” said Mayo oncologist Tufia Haddad, physician leader for the Watson matching project. The two organizations also plan to expand training and use of the system, including using Watson in additional cancer types, as well as other aspects of cancer therapy, such as surgery, radiation and supportive care. Currently, the system is trained to support clinical trial matching for breast, lung and gastrointestinal cancers.
The NIH is launching a two-year pilot project to help genomic researchers connect with individuals with genotypes of interest who have consented to further study of their phenotypes and health consequences.The new database of over 10,000 patient genomes and exomes — dubbed The Genomic Ascertainment Cohort, launched by the National Institute of Arthritis and Musculoskeletal and Skin Diseases in cooperation with the Inova Health System — will allow researchers to predict the effects of specific genes or variants, and test their hypotheses by re-examining the individual donors that consented to the project. “It’s basically taking clinical research and turning it on its head,” described Leslie Biesecker, chief of the Medical Genomics and Metabolic Genetics Branch at the National Human Genome Research Institute, which will maintain the database. For example, a researcher may have a functional lab assay demonstrating a link between the loss of a particular gene and cholesterol metabolism. The database would be able to identify patients with variants in that gene who have already consented to being re-contacted by investigators, for measurements of their cholesterol levels, Biesecker said. One thousand new volunteers will be recruited to have their genomes sequenced for the pilot project, while NIH institutes will contribute data from existing programs. In addition, Inova will contribute sequences from 8,000 parent-child trios from its Longitudinal Childhood Genome Study. While the project will initially only be available to NIH intramural researchers and Inova staff, TGAC plans to open the database to outside researchers in the future if the pilot is successful.
The FDA plans to encourage small groups or individual physicians to collaborate in developing stem-cell and regenerative medicine products and share their clinical data, according to CBER Director Peter Marks and Commissioner Scott Gottlieb in the New England Journal of Medicine. The collaborations will ultimately lead to the receipt of a biologics license for each party, they wrote. If the pooled clinical data — submitted in conjunction with manufacturing information — demonstrates a favorable risk-benefit profile, the FDA could rely on that data in product review, Marks and Gottlieb wrote. “The investigators who manufacture the product will need to agree on and follow a common manufacturing protocol, and develop a common clinical trial protocol,” they said. Each site will then manufacture its own product to treat patients enrolled at the clinical trial site. This system could be convenient for groups or small firms that do not have access to the necessary infrastructure or patient populations to go through the clinical development process alone, but can follow common clinical and manufacturing standards. “Our aim is to refashion our traditional tools for regulation to meet the challenges and opportunities presented by such highly innovative products as cell-based regenerative medicine,” they said. The NEJM article is available here: www.nejm.org/doi/full/10.1056/NEJMsr1715626.
The House of Representatives failed to pass its version of federal right-to-try legislation last week. An update of the Senate’s bill passed last summer; the House bill would have added requirements to comply with informed consent and institutional review board regulation. Sponsors would also be required to notify the government within seven days of granting a right-to-try request — and physicians would have to immediately inform sponsors of any serious adverse events. It would also restrict the use of clinical outcomes in regulatory reviews and approvals, unless the FDA determines the data is critical to understanding the safety of an investigational drug. Last week, Republicans in the House appeared set to bring the bill back to the floor.