EMAS Case Study The proposed study is a multisite, double-blind, randomized, placebo-controlled, parallel group study in children and adolescents with epilepsy with myoclonic-atonic seizures (EMAS). The primary endpoint is EMAS-associated seizure frequency over the treatment period.  Case Studies Multiple Sclerosis Case Study The proposed study is a randomized, double-blind, Bayesian, group sequential, non-inferiority (NI) trial comparing an investigational treatment to an active control in pediatric patients with multiple sclerosis (MS), borrowing strength from external data in adults and children. The primary endpoint is the annualized relapse rate (ARR). One interim analysis for efficacy is planned.   Case Studies Master Protocol Case Study This proposal is for a master protocol designed to study chronic pain.  This proposed master protocol permits multiple sub-studies to investigate proof of concept for several investigational products that may be intended to treat several types of chronic pain.   Case Studies Lupus Case Study The proposed study is a randomized, double-blind, Phase 2 study in patients with systemic lupus erythematosus (SLE), a rare disease with a high unmet need. Patients are to be randomized to one of four treatment groups: three doses of investigational product (IP) or placebo.  Case Studies DLBCL Case Study  The proposed trial is a randomized, open-label, multicenter trial in patients with first-line diffuse large Bcell lymphoma. Patients are to be randomized 2:1 to treatment vs. control. The primary endpoint of the study is Investigator-assessed progression-free survival (PFS), defined as the time from randomization to the first occurrence of progression or relapse, using the 2014 Lugano classification for Malignant Lymphoma (Cheson et al. 2014), or death from any cause, whichever occurs first.   Case Studies Critical Path to TB Drug Regimens Consortium (CPTR) CPTR Initiative for Anti-TB Drug Regimens is a cross-sector initiative launched to expedite the development of safer and shorter duration anti-tuberculosis drug regimens, addressing a critical public health challenge. Collaborations Multiple Sclerosis Outcome Assessments Consortium (MSOAC) MSOAC is promoting consensus science in MS, aiming to qualify a new measure of disability as an endpoint for MS therapy trials. Collaborations Polycystic Kidney Disease Outcomes Consortium (PKDOC) PKD Consortium for Imaging Biomarker Qualification is developing data standards and leveraging clinical data to support the qualification of imaging biomarkers for drug development trials. Collaborations Predictive Safety Testing Consortium (PSTC) PSTC for Drug Safety Tests is focused on obtaining regulatory acceptance of novel drug safety tests, validating innovative safety testing methods in collaboration with regulatory agencies. Collaborations Analgesic, Anesthetic and Addiction Clinical Trial Translations, Innovations, Opportunities and Networks (ACTTION) Initiative ACTTION for Therapeutic Interventions aims to identify, prioritize, coordinate, and sponsor activities to expedite the development of efficacious and safe analgesic, anesthetic, and addiction interventions, benefiting public health. Collaborations Heart Failure Collaboratory Aims to improve the ecosystem of heart failure clinical trials, advance scientific discussion and efficiency to ultimately foster the development of novel therapies and deliver new therapies to patients, focusing on enhancing the clinical trial landscape for heart failure. Collaborations Landscape Analysis of the Application of Artificial Intelligence and Machine Learning (AI/ML) in Regulatory Submissions for Drug Development From 2016 to 2021  This landscape analysis of regulatory submissions of drug and biological products to the US Food and Drug Administration from 2016 to 2021 demonstrated that AI/ML has been used successfully to perform a variety of tasks, such as informing drug discovery/repurposing, enhancing clinical trial design elements, dose optimization, enhancing adherence to drug regimen, endpoint/biomarker assessment, and post- marketing surveillance. Other Lessons Learned from the Model-Informed Drug Development (MIDD) Pilot Program The lessons learned from the MIDD pilot program are presented and reviewed in the following areas: leadership engagement and generating buy-in; staff education and fostering positive organizational culture; and process flexibility and ensuring  transparent communication. These findings advance and integrate MIDD approaches during drug development and regulatory review.  Programs Cardiac Safety Research Consortium (CSRC) CSRC is a public-private partnership dedicated to advancing the scientific study of cardiac safety issues, bringing together industry, academia, and government leaders to improve cardiac safety in drug development. Collaborations Global Pediatric Clinical Trials Network Pre-Launch Consortium (PTC) PTC for Innovative Drugs for Children is enabling the timely and efficient evaluation of innovative drugs, biologics, and devices for children, delivering regulatory-quality data needed for product labeling. Collaborations Critical Path for Alzheimer's Disease (CPAD) CPAD is bringing together diverse stakeholders to accelerate therapeutic innovation in Alzheimer's Disease under a data-driven, regulatory framework, aiming to advance treatment options for AD. Collaborations Electronic Patient-Reported Outcome (ePRO) Consortium ePRO Consortium for Clinical Trials is providing scientific leadership and best practice recommendations for electronic data capture technologies in clinical trials, supporting patient-focused outcomes data collection. Collaborations Coalition For Accelerating Standards and Therapies (CFAST) CFAST aims to accelerate clinical research and medical product development by creating and maintaining data standards, tools, and methods for research in important therapeutic areas, contributing to public health advancements. Programs Critical Path for Parkinson’s (CPP) CPP Consortium for Parkinson’s Prevention and Treatment is advancing the drug development landscape for Parkinson’s by collaborating globally to share data and resources, developing novel drug development tools. Collaborations Critical Path for Sickle Cell Disease (CP-SCD) CP-SCD Consortium is creating a regulatory science strategy to de-risk medical developments for Sickle Cell Disease, accelerating treatments and therapies through a wide-ranging partnership. Collaborations Critical Path to Therapeutics for the Ataxias (CPTA) CPTA for Ataxia Research and Development is bringing together experts to create regulatory tools and strategies for ataxia research, aiming to accelerate and innovate therapeutic development for these rare neurodegenerative disorders. Collaborations Duchenne Regulatory Science Consortium (D-RSC) D-RSC is creating an integrated database of patient-level clinical data from Duchenne Muscular Dystrophy (DMD) studies, developing standard terminology, and facilitating the development of clinical trial simulation tools for DMD. Collaborations Huntington’s Disease Regulatory Science Consortium (HD-RSC) HD-RSC is leveraging deep knowledge of HD to facilitate interaction and collaboration for treatment advancements, providing a neutral forum for the HD community. Collaborations International Neonatal Consortium (INC) INC aims to address the unmet need for evaluating therapies for neonates, uniting global stakeholders to generate consensus and develop tools for medical innovation. Collaborations Rare Disease Clinical Outcome Assessment Consortium (RD-COAC) RD-COAC is identifying tools and methodologies for collecting outcomes data in rare disease treatment trials, catalyzing drug development for measuring patient-relevant outcomes. Collaborations Type 1 Diabetes (T1D) Consortium T1D Consortium for Type 1 Diabetes Solutions is building actionable models of Type 1 Diabetes to enable more efficient and effective clinical trials, qualifying new biomarkers and endpoints from aggregated data analysis. Collaborations Transplant Therapeutics Consortium (TTC) TTC for Transplant Research and Development is facilitating a public-private partnership to speed up the development of new Immunosuppressive Therapies for transplant recipients, focusing on regulatory endorsement of novel endpoints for clinical trials. Collaborations Clinical Trial Transformation Initiative (CTTI) Public-Private Partnership The CTTI fosters an open forum for over 500 organizations and approximately 80 member organizations to discuss issues, exchange ideas, and come to a consensus on solutions. This unique approach blends diverse viewpoints, reshaping policies and procedures across the research community to run more efficient trials, generate high-quality evidence faster, and ultimately improve the health of people. Collaborations Clinical Trials Transformation Initiative (CTTI)'s Digital Health Technologies Project CTTI's Digital Health Technologies Project is increasing the use of clinical trial endpoints derived from digital technologies, enhancing the modernization of clinical trial methodologies. Collaborations Forum for Collaborative Research - The HIV Forum The HIV forum is bringing together experts in virology and immunology to advance research and development in HIV treatment, fostering collaboration among academia, regulatory agencies, industry, and patient communities. Collaborations Forum for Collaborative Research - The Liver Forum The Liver Forum is advancing regulatory sciences for the treatment of non-alcoholic fatty liver disease (NAFLD) and non-alcoholic steatohepatitis (NASH) by providing a venue for ongoing multi-stakeholder dialogue, facilitating science-based decision-making. Collaborations International Coalition of Medicines Regulatory Authorities (ICMRA) ICMRA collaborates globally to address human medicine regulatory challenges, fostering strategic direction, synergy, and resource leveraging. It enhances global communication, information sharing, crisis response, and regulatory science. Collaborations IQ Machine Intelligence for Quantitative Modeling in Drug Discovery & Development Applications Workshop (2022) Within the IQ Consortium, the Artificial Intelligence and Machine Learning (AI/ML) Working Group was formed with the aim to foster scientific dialogue on AI/ML applications and identify a set of good practices, so as to enable broader impacts in drug development. This workshop brought together experts from industry, academia, and the FDA to initiate a scientific dialogue and collaboration across disciplines to elevate the impact of ML. Public Meeting or Workshop International Rare Diseases Research Consortium (IRDiRC) IRDiRC is enabling accurate diagnosis, care, and therapy for rare diseases within one year of medical attention, addressing the challenges faced by people living with rare diseases. Collaborations Kidney Health Initiative (KHI) KHI is catalyzing innovation and development of safe and effective patient-centered therapies for kidney diseases, addressing the needs of patients with kidney conditions. Collaborations Lupus Accelerating Breakthroughs Consortium (ABC) Lupus ABC is working to bring lupus experiences into the spotlight, enabling collaboration to develop and pursue initiatives for improving health outcomes in lupus. Collaborations Outcome Measures in Rheumatology Clinical Trials (OMERACT) Pre-competitive Consortium Osteoarthritis Short-Term and Long Term Outcome Measures OMERACT for Autoimmune and Musculoskeletal Diseases is improving outcomes through advancing the design and quality of clinical studies, supporting the development of Core Outcome Sets for clinical trials. Collaborations Global Pediatric Clinical Trials Network The Institute for Advanced Clinical Trials for Children (I-ACT) was launched as the instantiation of the Advisory Report from Critical Path Institute’s Pediatric Trials Consortium. I-ACT’s mission/vision are fully aligned with the goals of RFA-FD-17-014 and focus on creating an integrated resource for pediatric product development, with sustainable global infrastructure to support all phases of pediatric clinical trials. Collaborations Pharmacuetical Users Software Exchange (PhUSE) PHUSE brings together representatives from the FDA, industry, and academia in a collaborative, non-competitive environment to find solutions to shared problems and challenges in clinical/non-clinical trials, data standards, data analysis, emerging technologies and beyond. Collaborations Pharmaceutical Inspection Co-operation Scheme (PIC/S) Good Clinical Practice (GCP) Work Group Participation This workgroup focuses on facilitating technical co-operation and harmonization of practices (including the development of guidance and training material), capacity building and information sharing, in particular through the creation of PIC/S Joint Visit inspection groups in these fields. Collaborations Setting International Standards of Quality of Life and Patient Reported Outcomes Endpoints- Innovative Medicines Initiative (SISAQOL-IMI) SISAQOL-IMI is establishing international standards for analyzing, interpreting, and reporting patient-reported outcomes data in cancer clinical trials. Collaborations Dental Quality Alliance (DQA) for Oral Health Care DQA for Oral Health Care is developing performance measures for oral health care, advancing measurement as a means to improve oral health, patient care, and safety. Collaborations Cures Within Reach - ReGRoW Pilot Program ReGRoW is supporting the growth of clinical research in low and lower-middle income countries, focusing on repurposed therapies for local patient treatments. Collaborations Project Site Selector – Navigating Oncology Drug Development through Troubled Waters Project Site Selector is an initiative focusing on identifying trends and rationale for selecting clinical trial sites across all phases of oncology drug development, aiming to optimize site selection processes. Programs C3TI Bayesian Supplemental Analysis (BSA) Demonstration Project This demonstration project will build experience across sponsors, FDA clinical reviewers, and statisticians in Bayesian methods to enhance statistical analysis and decision-making in clinical trials. Programs C3TI Selective Safety Data Collection (SSDC) Demonstration Project This demonstration project offers an innovative approach to facilitate the conduct of large-scale efficacy and safety trials through the purposeful reduction in the collection of certain types of data for drugs or biologics with a well-characterized safety profile. It will result in improved understanding of its real-world applicability, demonstration of its ability to facilitate efficient clinical trials, identification of potential challenges that programs encounter and ways to address those challenges, and promotion of best practices. Programs C3TI Streamlined Trials Embedded in clinical Practice (STEP) Demonstration Project This demonstration project will facilitate the adoption of pragmatic clinical trial approaches, focusing on their integration into point-of-care settings to make clinical trials more practical and applicable in real-world settings. Programs Accelerating Rare disease Cures (ARC) Program This FDA program drives scientific and regulatory innovation and engagement to accelerate the availability of treatments for patients with rare diseases. Programs IND Application Annual Reporting Proposed Rule The proposed rule seeks to revise FDA’s regulations for Investigational New Drug (IND) annual reporting and modify the format and content of the IND annual report to be generally consistent with those of the annual Drug Safety Update Report (DSUR) standards devised by the International Council for Harmonization of Technical Requirements for Pharmaceuticals for Human Use (ICH), aiming to align FDA's reporting standards with international norms. Other Oncology Center of Excellence (OCE) OCE unites experts across the FDA to conduct expedited review of medical products for oncologic and hematologic malignancies, aiming to speed up the review process for these critical areas. Programs Project Beyond Breakthrough Project Beyond Breakthrough identifies and institutes best practices for the expedited development of drugs and biological products designated as breakthrough therapies. The project focuses on the internal development of a pilot program aimed at early interactions to support dosage strategy selection for oncology products with Breakthrough Designation that have not yet been approved. Programs Project Optimus Project Optimus identifies opportunities to incorporate patient-reported outcomes (PRO) for a systematic and quantitative assessment of expected symptomatic adverse events and their impact on patients' functional abilities. Programs Project Orbis Project Orbis provides a framework for concurrent submission and review of oncology products among international partners. The FDA Oncology Center of Excellence (OCE) initiated Project Orbis in May 2019, aiming to streamline the review process for oncology products on an international level. Programs Project Pragmatica Project Pragmatica seeks to introduce functional efficiencies and enhance patient centricity by integrating aspects of clinical trials with real-world routine clinical practice through the appropriate use of pragmatic design elements, aiming to make clinical trials more relevant and integrated into everyday healthcare practices. Programs Center for Drug Evaluation and Research (CDER) Pilot Grant Program: Standard Core Clinical Outcome Assessments (COAs) and their Related Endpoints The purpose of this grant opportunity is to support the development of publicly available core sets of COAs and their related endpoints for specific disease indications or for disease impacts that span multiple diseases of similar phenotypes, aiming to standardize and enhance the use of COAs in clinical research.  Programs FDA Patient-Focused Drug Development Guidance Series for Enhancing the Incorporation of the Patient’s Voice in Medical Product Development and Regulatory Decision Making The FDA is developing a series of four methodological patient-focused drug development (PFDD) guidance documents to address, in a stepwise manner, how stakeholders can collect and submit patient experience data and other relevant information from patients and caregivers for medical product development and regulatory decision making. These guidance documents aim to standardize and enhance the use of patient experience data in the regulatory process, ensuring that patient perspectives are adequately considered in drug development and evaluation. Programs Advancing Real-World Evidence (RWE) Program This FDA program aims to improve the quality and acceptability of RWE-based approaches in support of new intended labeling claims, including the approval of new indications for approved medical products or to satisfy post-approval study requirements. Programs Complex Innovative Trial Design (CID) Meeting Program CID Meeting Program under PDUFA VII aims to advance the use of complex adaptive, Bayesian statistical methods, and other novel clinical trial designs, fostering innovation in clinical research methodologies. Programs Model-Informed Drug Development Paired Meeting Program The FDA MIDD Paired Meeting Program builds on the success of the MIDD Paired Meeting Pilot to continue to advance and integrate the development and application of exposure-based, biological, and statistical models derived from preclinical and clinical data sources in drug development and regulatory review. MIDD approaches use a variety of quantitative methods to help balance the risks and benefits of drug products in development, aiming to optimize drug development processes. Programs Rare Disease Endpoint Advancement (RDEA) Pilot Program The RDEA Pilot Program is specifically designed to advance drug development programs for rare diseases by providing a collaborative mechanism for sponsors to work with the FDA throughout the efficacy endpoint development process. Programs Biomarker Qualification Program Biomarker Qualification Program is working with external stakeholders to develop biomarkers as essential tools in drug development, aiming to streamline and improve the efficiency of the drug development process. Programs Center for Drug Evaluation and Research (CDER) Quantitative Medicine (QM) Center of Excellence (CoE)  The CDER QM CoE functions as a cooperative, coordinating body that spurs innovation and fosters comprehensive integration of QM approaches to advance therapeutic medical product development, inform regulatory decision-making, and promote public health.  Programs Critical Path Innovation Meetings (CPIM) CPIM is a means by which the Center for Drug Evaluation and Research (CDER) and investigators from industry, academia, scientific consortia, patient groups, and government can communicate to improve efficiency and success in drug development. The goals of the CPIM are to discuss a methodology or technology proposed by the meeting requester and for CDER to provide general advice on how this methodology or technology might enhance drug development. Programs FDA, MHRA, and Health Canada Good Clinical Practice Workshop: Global Clinical Trials - Considerations and Lessons Learned from the Changing Landscape (2022) This workshop provided insight into key topics, compliance trends and the opportunity to hear first-hand from regulators about lessons learned from the changing clinical trial landscape. Topics covered include: key aspects of building resilience in clinical trials, risk-based approach to sponsor oversight, use of real-world data/real-world evidence, updates regarding decentralized trials, changes in clinical trial activities and inspections, sponsor oversight of vendors, potential uses of artificial intelligence and machine learning in clinical trials, clinical and bioanalytical challenges in bioequivalence trials, and updates in guidance and inspection approaches.  Public Meeting or Workshop Center for Drug Evaluation and Research (CDER) Public-Private Partnership (PPP) and Consortia Coordination This coordination facilitates consistency and continuity throughout CDER as the Center engages in PPPs and consortia, ensuring a unified approach in these collaborative efforts. Collaborations Artificial Intelligence and Machine Learning (AI/ML) for Drug Development Discussion Paper The FDA published this discussion paper as part of a multifaceted approach to enhance mutual learning and to establish a dialogue with FDA stakeholders on AI/ML in drug development. Three main topics are highlighted in this discussion paper: Landscape of current and potential uses of AI/ML, considerations for the use of AI/ML, and next steps and stakeholder engagement. Other Support for clinical Trials Advancing Rare disease Therapeutics (START) Pilot Program START Pilot Program is a communication pilot for sponsors to regularly communicate with the FDA on clinical development issues in rare diseases, focusing on study design, control group selection, and patient population fine-tuning. Programs FDA Glossary of Terms This glossary provides a detailed list of terminologies used by the FDA as well as their definitions, aiming to clarify and standardize the language used in regulatory processes and communications. Trainings Collection of Race and Ethnicity Data in Clinical Trials and Clinical Studies for FDA-Regulated Medical Products: Draft Guidance for Industry The purpose of this draft guidance is to provide FDA’s expectations for, and recommendations on, use of a standardized approach for collecting and reporting race and ethnicity data in submissions including information collected and reported from clinical studies and clinical trials for FDA-regulated medical products. Using standard terminology for race and ethnicity helps ensure that data are collected and reported consistently in submissions to FDA.  Guidance Documents Considerations for the Conduct of Clinical Trials of Medical Products During Major Disruptions Due to Disasters and Public Health Emergencies: Final Guidance for Industry, Investigators, and Institutional Review Boards (IRBs) This final guidance aims to assist sponsors, IRBs, and clinical investigators in ensuring trial participant safety, maintaining GCP compliance, and minimizing risks to trial integrity during major disruptions like disasters and public health emergencies. Guidance Documents Considerations for the Use of Real-World Data and Real-World Evidence to Support Regulatory Decision-Making for Drug and Biological Products: Final Guidance for Industry  This final guidance discusses the applicability of FDA’s investigational new drug application (IND) regulations under part 312 to various clinical study designs that utilize Real-World Data (RWD), providing clarity on how RWD can be used in clinical studies. Guidance Documents Decentralized Clinical Trials for Drugs, Biological Products, and Devices: Draft Guidance for Industry, Investigators, and Other Stakeholders This draft guidance aims to improve inclusivity, diversity of participation, and convenience for trial participants by bringing trial-related activities closer to the patient, making clinical trials more accessible and patient-friendly. Guidance Documents Digital Health Technologies for Remote Data Acquisition in Clinical Investigations: Final Guidance for Industry, Investigators, and Other Stakeholders This final guidance supports the use of digital health technology (DHT) in clinical trials to obtain physiological and other data directly from patients, enhancing the data collection process and patient engagement in trials. Guidance Documents Diversity Plans to Improve Enrollment of Participants from Underrepresented Racial and Ethnic Populations in Clinical Trials: Draft Guidance for Industry  The purpose of this draft guidance is to provide recommendations to sponsors developing medical products on the approach for developing a Race and Ethnicity Diversity Plan to enroll adequate numbers of participants in clinical trials from underrepresented racial and ethnic populations in the United States. Guidance Documents Enhancing the Diversity of Clinical Trial Populations - Eligibility Criteria, Enrollment Practices, and Trial Designs: Final Guidance for Industry This final guidance provides recommendations on strategies for clinical trial sponsors to employ and enhance the enrollment of underrepresented populations, supporting new drug applications or biologics license applications. Guidance Documents Informed Consent: Final Guidance for IRBs, Clinical Investigators, and Sponsors This final guidance is designed to assist institutional review boards (IRBs), clinical investigators, and sponsors in complying with FDA’s informed consent regulations, ensuring ethical and legal standards are met in clinical investigations. Guidance Documents Investigator Responsibilities - Safety Reporting for Investigational Drugs and Devices: Draft Guidance for Industry This draft guidance is intended to help clinical investigators comply with the safety reporting requirements for investigational new drug application studies and investigational device exemption studies. It provides recommendations to help investigators identify safety information that needs to be reported to sponsors and institutional review boards, aiming to clarify and standardize the safety reporting process in clinical trials. Guidance Documents Key Information and Facilitating Understanding in Informed Consent: Draft Guidance for Sponsors, Investigators, and Institutional Review Boards (IRBs) This draft guidance provides recommendations related to two provisions of the revised Federal Policy for the Protection of Human Subjects (the revised Common Rule) by the U.S. Department of Health and Human Services (HHS) and identical provisions in FDA’s proposed rule “Protection of Human Subjects and Institutional Review Boards.” The FDA’s proposed rule, if finalized, would harmonize certain sections of FDA’s regulations on human subject protections and institutional review boards (IRBs), to the extent practicable and consistent with other statutory provisions, with the revised Common Rule, in accordance with the 21st Century Cures Act. The guidance addresses the provisions of the revised Common Rule that require informed consent to begin with key information about the research and to present information in a way that facilitates understanding and identical provisions in FDA’s proposed rule. Guidance Documents M11 Template: Clinical Electronic Structured Harmonized Protocol: Draft Guidance This draft guidance on the International Council for Harmonization of Technical Requirements for Pharmaceuticals for Human Use (ICH) M11 is intended for interventional clinical trials of drugs, vaccines, and drug/device combinations planned to be registered as drugs. The template is suitable for all phases of clinical research and all therapeutic areas. Existing ICH Guidelines and International Organization for Standardization (ISO) 14155 were considered in its development. The draft guidance is designed to enable modifications suitable for the particular trial. Guidance Documents Master Protocols for Drug and Biological Product Development: Draft Guidance for Industry This draft guidance provides recommendations on the design and analysis of trials conducted under a master protocol as well as guidance on the submission of documentation to support regulatory review. Guidance Documents Master Protocols: Efficient Clinical Trial Design Strategies to Expedite Development of Oncology Drugs and Biologics: Final Guidance for Industry This final guidance provides recommendations to sponsors of drugs or biologics for the treatment of cancer regarding the design and conduct of clinical trials intended to simultaneously evaluate more than one investigational drug and/or more than one cancer type within the same overall trial structure (master protocols) in adult and pediatric cancers. Guidance Documents Optimizing the Dosage of Human Prescription Drugs and Biological Products for the Treatment of Oncologic Diseases: Draft Guidance for Industry  This draft guidance is intended to assist sponsors in identifying the optimal dosage(s) for human prescription drugs or biological products for the treatment of oncologic diseases during clinical development prior to submitting an application for approval for a new indication and usage.   Guidance Documents Rare Diseases Natural History Studies for Drug Development: Draft Guidance for Industry This draft guidance is intended to help inform the design and implementation of natural history studies that can be used to support the development of safe and effective drugs and biological products for rare diseases. Guidance Documents Real-World Data: Assessing Registries to Support Regulatory Decision-Making for Drug and Biological Products: Final Guidance for Industry  This final guidance provides considerations for sponsors proposing to design a new registry or use an existing registry to support regulatory decision-making about a drug’s effectiveness or safety.  Guidance Documents FDA's Sentinel Initiative Sentinel, the FDA’s national electronic system, continues to transform the way researchers monitor the safety of FDA-regulated medical products by enhancing participation to a wider array of scientific expertise, translating new technologies from emerging fields such as data science and big data, creating laboratories to develop new approaches to using electronic health records, and cultivating a robust scientific community to uncover novel ways to leverage the system’s core capabilities beyond drug safety. Programs Good Clinical Practice (GCP) Inspection Collaboration with International Regulators for Drug Development GCP Inspection Collaboration with International Regulators for Drug Development is one of Center for Drug Evaluation and Research (CDER)'s collaborations with the European Medicines Agency (EMA), the Pharmaceuticals and Medical Devices Agency (PMDA) in Japan, the United Kingdom’s Medicines and Healthcare Products Regulatory Agency (MHRA-UK) and Health Canada to optimize regulatory resources and oversight in the evaluation of clinical trial conduct. Collaborations Artificial Intelligence and Machine Learning (AI/ML) for Drug Development FDA Webpage The FDA is committed to ensuring that drugs are safe and effective while facilitating innovations in their development. As with any innovation, AI/ML creates opportunities and new and unique challenges. To meet these challenges, the FDA has accelerated its efforts to create an agile regulatory ecosystem that can facilitate innovation while safeguarding public health, aiming to adapt to and leverage the advancements in AI/ML for regulatory purposes. Other Patient Preferences in Benefit-Risk Assessments during the Drug Life Cycle (PREFER) Consortium PREFER is promoting the development and adoption of patient preferences in decision-making about medical products, fostering collaboration among pharmaceutical companies, academic institutions, and patient representatives. Collaborations Accelerating Medicines Partnership (AMP) AMP is a public-private partnership between NIH, FDA, and various organizations to transform the model for developing new diagnostics and treatments, addressing current challenges in medical innovation. Collaborations E19 A Selective Approach to Safety Data Collection in Specific Late-Stage Pre-Approval or Post-Approval Clinical Trials This guidance is intended to provide internationally harmonized guidance on the use of selective safety data collection that may be applied in specific pre-approval or post-approval late-stage clinical trials.  Guidance Documents Use of Electronic Health Record Data in Clinical Investigations Guidance for Industry This guidance is intended to assist sponsors, clinical investigators, contract research organizations, institutional review boards (IRBs), and other interested parties on the use of electronic health record data in FDA-regulated clinical investigations. Guidance Documents Innovative Science and Technology Approaches for New Drugs (ISTAND) Pilot Program The Innovative Science and Technology Approaches for New Drugs (ISTAND) Pilot Program accepts submissions for qualification of types of drug development tools (DDTs) that are out of scope for existing DDT qualification programs but may still be beneficial for drug development. Programs Clinical Outcome Assessment (COA) Qualification Program The COA program manages the qualification process for COAs intended to address unmet public health needs; works directly with requestors in guiding COA development for qualification; and encourages a collaborative, multidisciplinary setting where CDER can review COAs and provide advice on the development or modification of COAs outside the IND/NDA/BLA pathway. Programs Introduction to ICH E19: Selective Collection of Safety Data in Clinical Trials - YouTube Video Facilitating efficiencies in clinical trial conduct through selective safety data collection under ICH E19 Training

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