The DIA 2017 Annual Meeting was held on June 18–22, 2017, in Chicago, IL. This multidisciplinary event attracted more than 4000 professionals from around the globe from academia and regulatory agencies, and from the pharmaceutical, biotech, and medical device industries. It featured more than 160 sessions grouped under 11 tracks: data/big data/eHealth; disruptive innovation; medical affairs and scientific communication; patient engagement; regulatory; safety and pharmacovigilance; special populations; strategic planning/execution and partnerships; translational sciences; preclinical/clinical and product development; value and access; and quality. CCSA senior scientist Ying Tang attended the meeting. She shares some topics of interest below.
Real-world evidence and the next generation of decision-making. There has been great interest among industry, researchers, and regulators in harnessing the rich information from real-world data that can be collected from the healthcare system. Randomized controlled trials (RCTs) have long been considered the gold standard to determine the safety and effectiveness of medical products, and used as the basis for regulatory approval. The panel discussed the strengths and limitations of real-world data and scenarios where real-world data may be most valuable in providing complementary evidence to RCTs.
International regulatory convergence. The FDA and EMA signed a mutual recognition agreement in February 2017 on GCP inspections. This agreement encourages harmonization. The FDA can rely upon the inspections conducted by EMA, and vice versa, to reduce duplication and maximize efficiency.
Updates from the China Food and Drug Administration (CFDA). Representatives from various divisions of CFDA discussed major initiatives and progress made since the publication of the regulatory reform plan in August 2015, entitled “The Opinions on Reforming Review and Approval Process for Drugs and Medical Devices.” China joined ICH as a full regulatory member in December 2016. Topics discussed included the current practice for GCP and GLP inspections, and their four-level adverse drug reaction monitoring system.
Using totality of evidence in the clinical and CMC context to support regulatory approval. Situations were discussed where a totality of evidence approach can be used in lieu of full evidence to support regulatory approval. This approach is being used in FDA’s review of botanical products because of some unique aspects/challenges associated with this type of product. A botanical product contains a heterogeneous mixture of components and sometimes its active constituents are unknown. Conventional CMC approaches for small molecules are often insufficient. Demonstration of therapeutic consistency from batch to batch can be supported by a totality of evidence approach, which takes into consideration multiple factors including controls in raw materials, manufacturing process, bioassays, and clinical data. To date, there have been more than 600 pre-INDs/INDs submitted to FDA, and only two NDAs submitted and approved.
Evolution of novel registration endpoints as diseases become chronic. There is a need for novel endpoints to capture the extended survival benefit of newer and more effective therapies. Minimum residual disease in myeloma and novel efficacy endpoints in the immuno-oncology setting were used as case studies to illustrate approaches for developing surrogate endpoints acceptable to regulators.