ecent history has shown that pathologic complete response (pCR) in breast cancer is a critical drug development tool (DDT). Particularly, the I-SPY 2 trial has supported development of agents for FDA accelerated approval based on pCR data obtained in the neoadjuvant setting in breast cancer. Leukemias have similar characteristics in that tumor burden can be measured directly in the bone marrow as well as in the blood after treatment. As discussed below, the key concept of Minimal Residual Disease (MRD) is that there is a threshold of disease below which the likelihood of relapse is low. It is somewhat easier to identify leukemia cells than to measure pCR, because usually (not always) the leukemic cells express antigens that are distinct or in a pathological profile that distinguishes leukemic cells from normal cells.

cross the life sciences industry, companies are increasingly focused on how they can improve the speed and efficiency of their operations. This is especially true now with greater attention being paid to drug prices. From the clinical through the commercial phases, companies are seeking unique ways to reduce costs to maintain their margins; regulatory operations is no exception.

personally controlled health record that summarizes an individual’s health issues, medical interventions, investigations, medicines, allergies and adverse reactions and is legitimately accessible to healthcare providers anywhere has immense appeal.

espite significant efforts over the past decade–and the fact that the world’s major regulatory bodies long ago developed tailored regulatory frameworks for orphan and/or drugs for rare diseases, or DRDs–Canada remains one of the few developed countries without a specific orphan drug regulatory framework.

018 marks a banner year of marketing approvals of new drugs and implementation of important regulatory reform measures in China. On the other hand, the vaccine scandal sounded a significant alert that a stringent regulatory system is very much needed to safeguard the quality of medicinal products and ensure patient safety. Reform of the Drug Administration Law and pricing/reimbursement pressures will drive more changes in 2019 to benefit patients as well as innovative pharmaceutical companies.

DIA Regulatory Affairs Community Regulatory Information Management (RIM) Working Group

ife Sciences organizations are increasingly facing several business challenges: an expanding global footprint, evolving regulatory requirements, dynamic partnership models, and rapidly evolving technology. Many organizations are realizing the importance of regulatory information as a corporate asset and hence improving and innovating how regulatory information is captured and utilized across the regulatory and pharmaceutical value chains, commonly referred as regulatory information management (RIM). Organizations, both sponsors and the vendors that serve them, have come up with organic solutions that are fit for specific purposes; however, the resulting systems are often not harmonized in approach or terminology which compounds the complexity associated with ownership, flow of data across the enterprise, interoperability, quality responsibility, and governance of regulatory data, or any form of collaboration/integration.

Over a two-year period, the RIMWG has authored an Interim Consensus White Paper describing the evolution of the RIMWG’s discussions, debates, feedback, and decisions made by various global RIM stakeholders during framework development. The framework itself comprises the identification of RIM capabilities according to common themes, or “buckets”; an “RIM Elements” catalog (in process); and a data map that identifies data elements and determines how data flows through the organization and related systems (also in process). The key objective of this initiative is to define an “Ideal RIM State” along with a RIM Reference Model which collects process, data, and organizational constructs that both sponsors and vendors could use as a benchmark to compare their respective capabilities and offerings. Together, the collection of data and process flow recommends not a binding regulation, but rather a reference-able framework for that “Ideal RIM State” upon which organizations can begin to model and benchmark against. The intention here is to create a framework which could benefit sponsor organizations, as well as technology and service providers, in their effort to improve how we manage regulatory information as a corporate asset.

While RIM has broader reach and scope, this interim consensus white paper starts by addressing key RIM components that are traditionally understood as “in-scope” for RIM. Many topics such as correspondence and commitments, labeling, and advertising and promotion, are not included in this version of the white paper. However, as the paper evolves, we will be adding more comprehensive definitions and other components. We expect this to be a “living document” that will continue to live until it covers all components included within the scope of RIM. We welcome broader industry participation and the expertise of individuals willing to join our efforts as volunteers to further the cause of this working group and contribute to the creation of artifacts to benefit industry.

Key stakeholders from this RIMWG will be conducting a round table at the Regulatory Submissions, Information, and Document Management (RSIDM) Forum from 7:30 – 8:30AM on February 12 in Bethesda, Maryland. We welcome participation from the regulatory community at large: Join us at RSIDM and make your voice heard by providing input into the RIM Elements Catalog, data map, and the evolving white paper.

our heart is pounding at what feels like 100 beats per minute. Your palms are sweaty, and your anxious nerves start to kick in. Immediately before you stand in front of an audience, whether it is five or five hundred people, your heart sinks into your stomach.