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FDA to Launch Pilot Program on Model-Informed Drug Development

April 23, 2018

The FDA announced a new pilot program in which drug sponsors can meet with agency officials to discuss strategies for model-informed drug development (MIDD) to make their clinical trials more efficient and increase the chances of regulatory approval. Under the pilot, each applicant whose proposal is approved will receive two meetings with the relevant agency center to discuss approaches to applying MIDD, which uses quantitative analysis to assess the risk-benefit profile of in-development drug products. “The goal of the early meeting discussions granted under this pilot program is to provide advice on how specific, proposed MIDD approaches can be used in a specific drug development,” the FDA said. The agency plans to accept two to four meeting requests per fiscal quarter from the fourth quarter of 2018 to the fourth quarter of 2022. Because of the limited number of meeting slots, the agency will prioritize requests that focus on dose selection/estimation, clinical trial simulation and predictive or mechanistic safety evaluation. Applicants should be drug or biologic manufacturers with a relevant IND or pre-IND number. The meeting requests should include the product name, application number, chemical name/structure and proposed indications or context of product development. Applicants should also include a brief overview of the purpose and objectives of the potential meeting, proposed MIDD approaches for the product and a list of issues for discussion with the agency. The submission package should also address drug development issues such as dosing, safety predictions or clinical trial design and the proposed MIDD approach. The pilot will be administered jointly by CDER’s Office of Clinical Pharmacology and CBER’s Office of Biostatistics and Epidemiology. Read the Federal Register notice here: www.fdanews.com/04-16-18-Pilot.pdf.

Oracle Launches New Platform for Collection of Electronic Patient Data for Clinical Trial Use
Oracle Health Sciences launched a new service that will allow researchers to collect electronic patient data for use in clinical trials. The new service links existing clinical systems to patient engagement sources, with the goal of helping therapeutic teams access patient data on areas such as medication dosing habits during clinical trials. Oracle will collaborate with companies including MC10, CM and Validic on the service, called the mHealth Connector Cloud Service. The company is also exploring integration efforts with solution integrators and developers such as Accenture and POSSIBLE Mobile. “Being able to take what used to be patient-recorded data and outcomes via paper forms and site visits can now be done via mobile health sensors and wearables that have the potential to shorten trial times and reduce costs, while allowing sick patients to remain in the comfort of their homes versus traveling to and from trial sites,” said Oracle Health Science General Manager Steve Rosenberg. “To improve patient enrollment in clinical trials, study teams must put the patient at the center of everything they do, and emerging technologies such as wearables and sensors hold the key.”

Six Health Systems Establish Consortium to Expand Clinical Trial Access in NJ and PA
Six regional health systems announced the creation of a nonprofit clinical research consortium – Partners in Innovation, Education and Research (PIER Consortium) – a regional clinical trial system that will span New Jersey and Pennsylvania. The consortium comprising Atlantic Health System, Drexel University, Einstein Healthcare Network, Geisinger including AtlantiCare, Main Line Health and Thomas Jefferson University was formed to expand clinical trial access to larger numbers of patients and bring new treatments to market faster. The consortium believes “having contracts in place and physicians identified” could allow trials to begin and reach participation capacity sooner and notes that 80 percent of clinical trials do not finish on time. “The expertise shared across sites through PIER will allow clinical researchers to enroll patients in trials more quickly, and streamline the clinical trial process across institutions,” it said.

Vivli Beta Tests Clinical Trial Data Sharing Platform
Moves to increase clinical trial data transparency gathered momentum this week with the release by Vivli of the beta version of its clinical trial data-sharing platform to the public prior to its full rollout on July 19. The Vivli platform offers clinical researchers a way to store, request and analyze anonymized, secure clinical trial data with built-in privacy protections. Vivli promises to “host data for stakeholders that do not have the ability to do so, enable interoperability of data from multiple sources, coordinate and integrate existing data-sharing initiatives.” Johnson & Johnson is one of the companies feeding data into the new Vivli platform. “We hope that new platforms, such as that being created by Vivli, will lead to a broadening of engagement by other stakeholders in the R&D ecosystem,” said J&J’s Chief Medical Officer Joanne Waldstreicher. “We believe that the goal of data sharing — advancing science and medicine for the benefit of patients — will only fully be realized when all stakeholders who generate clinical data participate,” Waldstreicher said. Other groups are also tracking progress. Clinical trial transparency advocacy group AllTrials uses its TrialsTracker tool to monitor clinical trials that missed the transparency deadline. In the first two days of the FDA requirement going into effect, the tool identified 12 out of 117 trials that failed to show results. Since February, the FDA has required the sharing of clinical trial results on ClinicalTrials.gov, with possible fines of more than $10,000 daily for sponsors failing to publish the data.

Tufts Medical Center Joins TriNetX Health Research Network
Tufts Medical Center has joined the TriNetX health research network and will use the platform to help recruit eligible patients into clinical trials and to identify new ideas for trials. The cloud-based network gives biopharmaceutical companies and contract research organizations aggregate views of patient data allowing them to identify institutions such as Tufts as potential research partners. Clinical researchers at client institutions can offer their own patients opportunities to participate in clinical trials. Tufts Medical Center, the principal teaching hospital for the Tufts University School of Medicine, was selected in part for its history of scientific research and clinical advances, including research that led to the discovery of drugs that safeguard the body against rejecting transplanted organs. “We look forward to using TriNetX’s query and analytics capabilities to dig deeper into our clinical data both to recruit eligible patients into clinical trials and to generate new hypotheses for investigation,” said Will Harvey, Tufts Medical Director/Clinical Informatics and CTSI Director of Informatics Integration. “Additionally, we hope to attract more sponsored research studies that seek to take advantage of our unique patient populations.”

European Regulators Approve CRISPR Technology Trial for Beta Thalassemia for 2018
CRISPR Therapeutics has slated a European clinical trial of its gene-editing technology for a common blood disorder this year. Researchers aim to use the tool for patients with the inherited blood disorder beta thalassemia, which can cause bone deformities and anemia and affects thousands of children every year. Researchers will harvest stem cells from patients and engineer them with a goal of increasing levels of fetal hemoglobin, after which they will return the cells to the patients’ bodies. If the procedure is successful, beta thalassemia will be the first human disease treated with the gene-editing technology in Europe. Researchers have not yet established a specific location for the trials. “Certainly, 2018 promises to be the big year for clinical trials using CRISPR-based genome editing. Results presented by [CRISPR Therapeutics] at a hematology meeting showed that the method dramatically increased fetal hemoglobin in beta thalassemia patients’ cells,” geneticist Helen O’Neill of University College London told the Telegraph. “The therapy successfully edited over 90 percent of blood stem cells removed from patients, which were re-transfused.” The announcement comes after a January decision by the European Patent Office to revoke the main patent on CRISPR-Cas9 as filed by the Broad Institute of MIT and Harvard University, after it was found to have omitted the name of one of the inventors listed on previous applications. Chinese universities, meanwhile, have used CRISPR technologies on dozens of patients in clinical trials since 2015. The first U.S. CRISPR clinical trial began this year, sponsored by the University of Pennsylvania.