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  1. Drug Safety and Availability

FDA initiates the Lysosomal Diseases Pre-Consortium at the Critical Path Institute

[9/1/2022] FDA’s Center for Drug Evaluation and Research (CDER) is pleased to announce its partnership with the Critical Path Institute (C-Path) to explore the feasibility of launching the Lysosomal Diseases Consortium, a public-private partnership among FDA, leading academic institutions, pharmaceutical industries, patient groups, and non-government organizations to address unmet drug development needs for patients living with lysosomal diseases – inherited metabolic diseases that are characterized by an abnormal build-up of various toxic materials in the body’s cells as a result of enzyme deficiencies.

C-Path is an independent, nonprofit, public-private partnership that aims to accelerate the pace and reduce the costs of medical product development through the creation of new data standards, measurement standards, and methods standards that aid in the scientific evaluation of the effectiveness and safety of new therapies.

The proposed Lysosomal Diseases Consortium will be under the umbrella of the Accelerating Rare disease Cures (ARC) Program and will focus on generating actionable solutions to accelerate drug development for lysosomal diseases, and to ensure that those solutions reflect current scientific knowledge of lysosomal diseases and regulatory standards for implementation of drug development. During the current exploratory phase, C-Path will initiate a one-year pre-consortium effort to determine the feasibility of ensuring a sustainable operational consortium. The pre-consortium will bring together key stakeholders to conduct a comprehensive landscape analysis of the ecosystem of lysosomal diseases. Through the stakeholders’ collective input and feedback, the group will help determine the high-level goals and objectives; generate a research plan to focus on identified common unmet medical product development needs; and develop an operations and governance plan for the envisioned multi-stakeholder consortium.

CDER values input and feedback from knowledgeable stakeholders and is committed to working with advocacy groups, academics, industry, patients, and other partners to help identify solutions for the challenges in rare disease drug development.

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