NIH RA, Lupus Research Program Releases Cellular Datasets to Industry

An NIH research collaboration of rheumatoid arthritis and systemic lupus erythematosus has made its datasets, which characterize disease cells, available to the public. The program hopes researchers mine the data to accelerate the development of products and explore potential targets for new treatment options. The Phase I study, part of the Accelerating Medicines Partnership, analyzed individual cells from the lining of joints affected by rheumatoid arthritis and from kidneys damaged from lupus, hoping to better understand the specific pathways at play, and help to provide a new approach to understanding autoimmunity. The data also has potential implications for precision medicine, the NIH said. The program is one of three launched in 2014 as part of a public-private partnership, led by the NIH’s National Institute of Arthritis and Musculoskeletal and Skin Diseases, in cooperation with the National Institute of Allergy and Infectious Diseases. Investigators are currently conducting Phase II studies, including larger cohorts of patients. The Foundation for the NIH manages the partnership with industry, including AbbVie, Bristol-Myers Squibb, Merck, Pfizer, Sanofi and Takeda Pharmaceuticals, as well as nonprofits such as the Arthritis Foundation, Lupus Foundation of America, Lupus Research Alliance and Rheumatology Research Foundation. The Phase I data are freely available through the NIAID-sponsored Immunology Database and Analysis Portal, at www.immport.org. Genomic data are also being submitted to be made available through the NIH’s database of Genotypes and Phenotypes, at: www.ncbi.nlm.nih.gov/gap.

FDA Adopts ICH’s E6(R2) Addendum to its GCP Guideline

The FDA adopted an update to the ICH’s E6 guideline on good clinical practice, including an addendum on advances in clinical trial design and processes to improve efficiency. In addition, the document recommends that sponsors develop a systematic, prioritized, risk-based approach to monitoring clinical trials. Dubbed E6(R2), with the final version adopted by the international group of regulators in November 2016, the guidance includes standard operating procedures for electronic trial data system setup, installation and use, as well as descriptions of system validation, functionality testing and data collection. It also reflects the increased use of electronic communications since the initial publication of the guideline in 1996. Other amendments include a recommendation that sponsors document their rationale for choosing on-site or centralized monitoring, or a combination of the two. Reports of on-site and/or centralized trial monitoring should be provided to the sponsor with documentation of the results in enough detail to verify compliance with the monitoring plan. The full guidance is available here: www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM464506.pdf.

Market Research Report Predicts Large Expansion in Global Clinical Trials Management

The size of the global clinical trial management (CTM) market is estimated to more than double over the next five years, reaching $1.37 billion by 2023, according to a report by Allied Market Research. Clinical trial management accounted for $570 million in 2016, with North America being the largest contributor. The sector is expected to grow by 13.4 percent annually by 2023, with China and the Asia-Pacific region seeing the largest gains, the firm said. The growth is being driven in part by the need for novel drugs and medical devices, as well as increasing trends in outsourcing and the globalization of clinical trials. Additionally, demand has increased due to the growing R&D expenditures and the benefits of employing clinical trials management strategies. However, according to the report, high costs and a lack of skilled professionals is projected to hamper growth efforts.Software is currently the dominant segment in the market. Enterprise systems contribute the most toward market growth, but site-based systems are expected to overtake, through their ease of use.

NIH Launches Global Study to Track AMD Progression

The NIH, through the National Eye Institute, is launching a five-year clinical study to examine the natural history of early age-related macular degeneration (AMD). The study plans to follow 500 people — visualizing structures within the eye, and measuring their function — to identify biomarkers of progression before the disease advances to later stages and causes loss of vision. AMD is the leading cause of vision impairment and blindness among people age 50 and older in the U.S., the NIH said, with 10 to 20 percent of early AMD patients progressing to late-stage disease within five years, influenced by a combination of age, genetic and other factors. Researchers have identified 52 independent genetic variants associated with AMD, but further research is needed to determine how these variants influence disease development and progression, the NIH said.The study, NCT03092492, is funded by the NEI, with sites located throughout the U.S., United Kingdom, Australia, Germany and Italy.