C3TI Compass (Knowledge Repository)
CDER Center for Clinical Trial Innovation (C3TI)
C3TI Compass provides information on completed CDER clinical trial innovation activities, maintains a comprehensive portfolio of ongoing efforts and knowledge resources, and supports knowledge-sharing. Its objective is to connect users to innovation information quickly, and to simplify the pathway for interested parties to engage with the FDA's mission of advancing clinical trial innovation.
View Frequently Asked Questions and Compass Glossary for more information.
| Title | Description | Format |
|---|---|---|
| A Bayesian Approach in Design and Analysis of Pediatric Cancer Clinical Trials | A Bayesian approach can be a flexible tool to formally leverage prior knowledge of adult or external controls in pediatric cancer trials. In this FDA-authored publication, a case example is provided to demonstrate how Bayesian approaches can be used to design, monitor, and analyze pediatric trials. | Case Studies |
| Actionable Steps to Drive Inclusion and Diversity in Clinical Trials | This article reviews the proceedings of a multi-stakeholder conference—including representatives from the FDA and NIH, practicing clinical investigators, pharmaceutical and device companies, community-based organizations, data analytics companies, and patient advocacy groups—about the current state of diversity in clinical trials and outlines actionable steps for improvement. | Other |
| Adaptive Design Clinical Trials for Drugs and Biologics: Final Guidance for Industry | This final guidance is provided for applicants submitting investigational new drug applications (INDs), new drug applications (NDAs), biologics licensing applications (BLAs), or supplemental applications on the appropriate use of adaptive designs for clinical trials to provide evidence of the effectiveness and safety of a drug or biologic. It also advises sponsors on the types of information to submit to facilitate FDA evaluation of clinical trials with adaptive designs, including Bayesian adaptive and complex trials that rely on computer simulations for their design. | Guidance Documents |
| Adjusting for Covariates in Randomized Clinical Trials for Drugs and Biological Products: Final Guidance for Industry | This final guidance describes FDA’s current recommendations regarding adjusting for covariates in the analysis of randomized, parallel group clinical trials that are applicable to both superiority trials and noninferiority trials. The main focus of the guidance is on the use of prognostic baseline covariates to improve statistical efficiency for estimating and testing treatment effects. | Guidance Documents |
| Advanced Research Projects Agency for Health (ARPA-H) Advancing Clinical Trial Readiness (ACTR) Initiative | ACTR initiative seeks to improve the nation’s ability to conduct clinical trials safely, quickly, and equitably. It will develop, evaluate, and integrate new tools and technologies to enable faster, less expensive, decentralized trials operating closer to/at points of care that are more representative across geography, age, gender, race, ethnicity, and socioeconomic status. | Other |
| Advancing Applications of Real-World Data (RWD) for Post-Marketing Safety Evaluations in the Sentinel System | This program aims to improve the quality and acceptability of Real-World Evidence (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 |
| Advancing the Use of Complex Innovative Designs (CIDs) in Clinical Trials: From Pilot to Practice Workshop (2024) | The purpose of this public workshop was to facilitate discussion on the use of external data sources, Bayesian statistical methods, and simulations in CIDs as well as trial implementation. This meeting met the performance goal of convening a public workshop on CIDs included in the seventh authorization of the Prescription Drug User Fee Act (PDUFA VII). | Public Meeting or Workshop |
| Alcohol Clinical Trials Initiative (ACTIVE) | The mission of ACTIVE is to develop consensus reviews and recommendations to improve the design, execution, and interpretation of trials of medications for the treatment of alcohol dependence. | Collaborations |
| Bayesian Methods in Human Drug and Biological Products Development in the Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER) | This FDA-authored article discusses the Bayesian framework for drug and biological product development, highlights key settings in which Bayesian approaches may be appropriate, and provides recent examples of the use of Bayesian approaches within the FDA’s CDER and CBER. | Case Studies |
| Biomarkers Consortium (BC) | BC convenes government, industry, patients and patient advocacy groups, and not-for-profit organizations to facilitate the development and the seeking of regulatory approval for disease biomarkers and surrogates, addressing one of the most pressing needs in disease diagnosis and treatment. | Collaborations |
| Children’s Registry for the Advancement of Therapeutics (CREATE) | CREATE is a drug-agnostic safety registry designed by a collaboration between universities, hospitals, health care providers, learning health networks, community organizations, the pharmaceutical industry and government agencies to support the completion of global post-marketing safety requirements, as well as collect safety information on all therapies used in pediatric Inflammatory Bowel Disease (IBD) patients. | Tools |
| Communities Advancing Research Equity (CARE) for Health | Care for Health seeks to improve access to clinical research to inform medical care, particularly for those in communities historically underrepresented in clinical research or underserved in health care. Informed by the health needs of these communities, CARE for Health will help to grow an evidence base that contributes to improved patient outcomes, provide communities access to the best available scientific research, and expand opportunities to participate in clinical trials and studies. | Programs |
| Complex Innovative Trial Design (CID) Pilot Meeting Program: Progress to Date | This FDA-authored publication highlights the five submitted meeting requests that have been selected for participation in the CID Pilot meeting program. The selected submissions, thus far, have all utilized a Bayesian framework. The reasons for the use of Bayesian approaches may be due to the flexibility provided, the ability to incorporate multiple sources of evidence, and a desire to better understand the FDA's perspective on such approaches. | Other |
| Decentralized Clinical Trials (DCT) Draft Guidance Webinar (2023) | As part of the FDA’s efforts to be responsive to the rapidly evolving clinical trial landscape and clarify the Agency’s recommendations on the conduct of DCTs, the FDA provided an overview of the draft guidance titled “Decentralized Clinical Trials for Drugs, Biological Products, and Devices” for small businesses and industry. | Public Meeting or Workshop |
| Digital Health Technologies (DHTs) for Drug Development: Demonstration Projects | To promote shared learning and understanding with external stakeholders, FDA continues to oversee and support numerous demonstration (i.e. research) projects on DHTs through multiple venues and programs include FDA’s Broad Agency Announcement, CERSI program, and other funding opportunities. This webpage lists current FDA-funded demonstration projects on DHTs. | Programs |
| Discussing Approaches to Enhance Clinical Study Diversity Public Workshop (2023) | The FDA, in collaboration with the Clinical Trials Transformation Initiative, hosted a virtual public workshop to solicit input on increasing the enrollment of historically underrepresented populations in clinical studies and encouraging clinical study participation that reflects the prevalence of the disease or condition among demographic subgroups. | Public Meeting or Workshop |
| Draft Glossary of Clinical Research Terms Related to Innovative Clinical Trial Design | Convened by the FDA and NIH, this draft glossary is intended to better facilitate communication within the clinical research community by helping to establish a common vocabulary to more uniformly characterize clinical research. In turn, the research community may be better situated to evaluate potential strengths and weaknesses of individual studies, and to clearly convey innovative aspects of clinical research in a meaningful way to funders, reviewers, and other interested parties. | Tools |
| Enhancing Adoption of Innovative Clinical Trial Approaches Workshop (2024) | The FDA and the Duke-Margolis Center for Health Policy convened a hybrid public workshop to discuss efforts to advance innovation of clinical trial design and conduct. The Center for Drug Evaluation and Research (CDER)'s portfolio of clinical trial innovation activities are wide ranging and span across drug development programs, therapeutic areas, and scientific disciplines. Stakeholders discussed barriers and challenges to incorporating successful or promising innovative clinical trial approaches in drug development. | Public Meeting or Workshop |
| Extramural Research Projects Funded by the FDA’s Office of Minority Health and Health Equity (OMHHE) | The OMHHE Research and Collaboration Program works with external partners to support research studies about minority health and health disparities. Research under this program provides insight into the scientific basis for individual therapies and seeks to advance the diversity of clinical trials. Studies also provide future directions for research and aid regulatory decision-making. This webpage lists current and completed FDA-funded projects with regard to minority health and health equity. | Programs |
| FDA | American Society of Clinical Oncology (ASCO) Annual Workshop on Getting the Dosage Right (2023) | The FDA and ASCO have co-sponsored several workshops that have highlighted the limitations to our current strategies for determining the recommended dosage(s) of anticancer agents for evaluation in registration trial. Specific topics discussed at this workshop includes determining the challenges to dosage optimization for combination therapies, identifying nonclinical data that support dosage optimization of the combination therapy assessing pharmacokinetic and pharmacodynamic interactions for dosage optimization of combination regimens, and evaluating trial designs to support dosage optimization of combination therapies. | Public Meeting or Workshop |
| FDA CDER & JHU CERSI Workshop | Addressing Challenges in the Design and Analysis of Rare Disease Clinical Trials: Considerations and Tools | The FDA’s Center for Drug Evaluation and Research (CDER) and the Johns Hopkins University’s Center of Excellence in Regulatory Science and Innovation (JHU CERSI) hosted a jointly sponsored virtual workshop on addressing challenges in the design and analysis of rare disease clinical trials. | Public Meeting or Workshop |
| Guidance Recap Podcast | E19 A Selective Approach to Safety Data Collection in Specific Late-Stage Pre-approval or Post-approval Clinical Trials | The FDA recently published the Guidance Snapshot for E19 A Selective Approach to Safety Data Collection in Specific Late-Stage Pre-Approval or Post-Approval Clinical Trials. The Guidance Recap Podcast provides highlights from this FDA guidance document directly from the authors. | Guidance Documents |
| Interacting with the FDA on Complex Innovative Trial Designs (CIDs) for Drugs and Biological Products: Final Guidance for Industry | This final guidance discusses the use of novel trial designs in the development and regulatory review of drugs and biological products, how sponsors may obtain feedback on technical issues related to modeling and simulation, and the types of quantitative and qualitative information that should be submitted for review. | Guidance Documents |
| International Consortium for Innovation & Quality in Pharmaceutical Development (IQ) Drug Induced Liver Injury (DILI) Initiative | This initiative is defining best practices for the detection, monitoring, management, and prevention of drug induced liver injury in clinical trials and pharmacovigilance programs. | Collaborations |
| International Life Sciences Institute (ILSI) - Health and Environmental Sciences Institute (HESI) Cardiac Safety Committee | ILSI - HESI Cardiac Safety Committee is improving public health by reducing cardiovascular-related adverse effects from drugs or chemicals, developing approaches for early detection and prediction. | Collaborations |
| Learning and Education to Advance and Empower Rare Disease Drug Developers (LEADER 3D) Initiative | This initiative is an effort of FDA’s Accelerating Rare disease Cures (ARC) Program. LEADER 3D aims to better understand the unique challenges in bringing rare disease products to market. LEADER 3D stakeholders participate in public workshops to share regulatory perspectives and knowledge on topics such as regulatory fitness for rare disease drug development, real-world data in rare disease drug development, and complex innovative design approaches. | Programs |
| Meta-Analyses of Randomized Controlled Clinical Trials (RCTs) to Evaluate the Safety of Human Drugs or Biological Products: Draft Guidance for Industry | This final guidance is provided for applicants submitting investigational new drug applications (INDs), new drug applications (NDAs), biologics licensing applications (BLAs), or supplemental applications on the use of meta-analyses of RCTs to evaluate the safety of human drugs or biological products within the framework of regulatory decision-making. Specifically, this guidance describes the factors FDA intends to consider when evaluating the strength of evidence provided by a meta-analysis studying the safety of drugs. | Guidance Documents |
| Multiple Endpoints in Clinical Trials: Final Guidance for Industry | This final guidance provides sponsors and review staff with the FDA’s thinking about the problems posed by multiple endpoints in the analysis and interpretation of study results and how these problems can be managed in clinical trials for human drugs, including drugs subject to licensing as biological products. | Guidance Documents |
| National Cancer Institute (NCI) Clinical Trials Innovation Unit (CTIU) | The NCI CTIU includes representatives from NCI, FDA, and National Clinical Trials Network (NCTN). It was created to accelerate clinical testing of new cancer prevention, diagnostic, treatment, and survivorship approaches. The projects selected for the CTIU are particularly suitable to innovative approaches with uncomplicated designs, using focused endpoints and data collection. | Collaborations |
| National Center for Advancing Translational Sciences (NCATS) Trial Innovation Network (TIN) | The TIN is an initiative of the Clinical and Translational Science Awards (CTSA) Program. The collaborative features a single IRB system, master contracting agreements, quality-by-design approaches, and a focus on evidenced-based strategies to recruitment and patient engagement. Launched to facilitate multi-site clinical studies, TIN is composed of three key organizational partners: CTSA Program Hubs, Trial Innovation Centers (TICs), and a Recruitment Innovation Center (RIC). | Collaborations |
| Natural History Studies and Registries in the Development of Rare Disease Treatments Workshop (2024) | The Reagan-Udall Foundation for the FDA, in collaboration with FDA’s Rare Diseases Team within the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine; Office of New Drugs; Center for Drug Evaluation and Research and the National Institutes of Health’s Division of Rare Diseases Research Innovation within the National Center for Advancing Translational Sciences, hosted a public workshop. The workshop brought together rare disease patient advocates, academic researchers, regulated industry, and other key stakeholders to discuss considerations for the use of natural history study and registry data in rare disease drug development programs. | Public Meeting or Workshop |
| Public Workshop: Understanding Priorities for the Development of Digital Health Technologies (DHTs) to Support Clinical Trials for Drug Development and Review (2023) | The FDA and the Duke-Robert J. Margolis, MD Center for Health Policy hosted a virtual public workshop to understand the priorities for the development of DHTs to support clinical drug trials, including accessibility, diversity, and clinical outcomes measures using DHTs. | Public Meeting or Workshop |
| Publications from the Office of Biostatistics (OB) Staff | The OB conducts independent research on statistical methodologies relevant to CDER’s scientific mission and regulatory review process for publication. OB research spans such broad areas as clinical trial design, Bayesian statistics, signal detection, causal inference, meta-analysis, bioequivalence, benefit-risk assessment, patient focused drug development, psychometrics, and product quality assessment and control. This webpage includes a select list of peer-reviewed scientific journal articles that include OB statisticians. | Other |
| Rare Diseases: Considerations for the Development of Drugs and Biological Products: Final Guidance for Industry | This final guidance is intended to assist sponsors of drugs and biological products for treatment of rare diseases in conducting efficient and successful drug development programs through a discussion of selected issues commonly encountered in rare disease drug development. | Guidance Documents |
| Real-World Data (RWD) and Real-World Evidence (RWE)-focused Demonstration Projects | To promote shared learning and understanding with external stakeholders, FDA continues to oversee and support numerous demonstration (i.e. research) projects through multiple venues and programs including the broad agency announcements, funding opportunity announcements, CERSI grants, and inter-agency agreements. This webpage lists ongoing and completed demonstration projects focused on RWD and RWE. | Programs |
| Real-World Data (RWD) as External Controls Case Study: BALVERSA (erdafitinib) | Eerdafitinib is a New Molecular Entity (NME) approved for the treatment of adult patients with locally advanced or metastatic urothelial carcinoma (UC) that included patient-level RWD as an external control. However, the FDA review concluded that the RWD submitted were uninterpretable due to data quality and completeness issues (such as the lack of standardized approach in assessing tumor response, a small sample size, differential selection of comparison groups, and missing data) and deemed the results inconclusive. Therefore, there is no discussion of the RWE in the BALVERSA US Prescribing Information These conclusions raise awareness around challenges and issues when using external control data to potentially aid in making submissions using RWD of better quality (pp. 1-27) | Case Studies |
| Real-World Data (RWD) as External Controls Case Study: BRINEURA (cerliponase alfa) | Cerliponase alfa is a biologics NME approved for the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2 disease) that included patient-level RWD as an external control. The FDA requested multiple comparative analyses between the clinical trial and the historical control. Despite finding some differences in patient characteristics between the two cohorts and the use of a different version of the clinician-reported outcomes (ClinROs) than the one used in the clinical trial, the BRINEURA US Prescribing Information includes the comparative analysis results on the motor domain of the CLN2 rating scale. These conclusions raise awareness around challenges and issues when using external control data to potentially aid in making submissions using RWD of better quality (pp. 9-27). | Case Studies |
| Real-World Data (RWD) as External Controls Case Study: XPOVIO (selinexor) | Selinexor is a is a New Molecular Entity (NME) approved to treat patients with relapsed refractory multiple myeloma that included patient-level RWD as an external control. However, the FDA review concluded that the evidence generated from the RWD was inadequate to provide context or comparison for the overall survival observed in the single-arm trial. Therefore, there is no discussion of the RWE in the XPOVIO US Prescribing Information. These conclusions raise awareness around challenges and issues when using external control data to potentially aid in making submissions using RWD of better quality (pp. 83-91). | Case Studies |
| Real-World Data (RWD): Assessing Electronic Health Records and Medical Claims Data to Support Regulatory Decision-Making for Drug and Biological Products: Final Guidance for Industry | This final guidance is intended to provide sponsors and other interested parties with considerations when proposing to use electronic health records (EHRs) or medical claims data in clinical studies to support a regulatory decision for effectiveness or safety. | Guidance Documents |
| Real-World Evidence (RWE) Submissions to FDA’s Center for Drug Evaluation and Research (CDER) | As part of the reauthorization of the Prescription Drug User Fee Act (PDUFA VII), FDA committed to reporting aggregate and anonymized information on submissions to CDER that contain RWE. This webpage describes submissions to CDER containing RWE that meet specified reporting criteria. This report is not intended to include all submissions to CDER containing analyses of RWD. Columns will be added annually to represent submissions by fiscal year (FY) from FYs 2023 through 2027. | Other |
| Society of Clinical Data Management (SCDM) | FDA's Center for Drug Evaluation and Research (CDER) and SCDM have collaborated to host annual meetings, Regulatory Town Halls, Leadership Forums, and sessions on various topics related to decentralized clinical trials (DCTs), digital health technology (DHT) use in the clinical trials, artificial intelligence/machine learning (AI/ML), data governance, real-world data (RWD), quality by design, quality management, and risk-based monitoring to promote quality and excellence in clinical data management. | Collaborations |
| Streamlining Drug Development and Improving Public Health through Quantitative Medicine Workshop: An Introduction to the Center for Drug Evaluation and Research (CDER)'s Quantitative Medicine Center of Excellence (QM CoE) (2024) | This virtual workshop was hosted by the newly established FDA CDER QM CoE. The purpose of this workshop was to introduce the CDER QM CoE, provide an overview of the scope, goals, and current state, while gaining feedback from the public on needs and opportunities in education, outreach, and policy. | Public Meeting or Workshop |
| The Amyloidosis Forum | The Amyloidosis Forum, formed in 2019, is a public-private partnership (PPP) between the Amyloidosis Research Consortium (ARC) and the FDA. The overarching goal of this forum is to accelerate the advancement of new therapeutics by addressing scientific gaps and unmet research needs in the precompetitive domain that aid in the evaluation of the efficacy and safety of new therapies. Current areas of focus include improving endpoints for amyloid light-chain (AL) amyloidosis and evolving study designs in a changing landscape for ATTR amyloidosis. | Collaborations |
| Complex Innovative Design (CID) Case Study: A Study in Patients with Epilepsy with Myoclonic-Atonic Seizures (EMAS) | The proposed study is a multisite, double-blind, randomized, placebo-controlled, parallel group study in children and adolescents with epilepsy with EMAS. The primary endpoint is EMAS-associated seizure frequency over the treatment period. | Case Studies |
| Complex Innovative Design (CID) Case Study: A Study in Pediatric Patients with Multiple Sclerosis (MS) | 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 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 |
| Complex Innovative Design (CID) Case Study: Master Protocol to Study Chronic Pain | 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 |
| Complex Innovative Design (CID) Case Study: A Study in Patients with Systemic Lupus Erythematosus (SLE) | The proposed study is a randomized, double-blind, Phase 2 study in patients with 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 |
| Complex Innovative Design (CID) Case Study: External Control in Diffuse Large B-Cell Lymphoma (DLBCL) | The proposed trial is a randomized, open-label, multicenter trial in patients with first-line DLBCL. 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 | Heart Failure Collaboratory is fostering the development of therapies by creating a collaborative environment for scientific discussion and efficacy in heart failure clinical trials. | 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 |
| International Consortium for Innovation & Quality in Pharmaceutical Development (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 |
| Investigational New Drug (IND) Application Annual Reporting Proposed Rule | The proposed rule seeks to revise FDA’s regulations for 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 (PFDD) 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 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. | Guidance Documents |
| 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. | Tools |
| 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 Populations in Clinical Studies: Draft Guidance for Industry | This draft guidance describes the form, content, and manner of diversity action plans, the applicable medical products, and clinical studies for which a diversity action plan is required, the timing and process for submitting diversity action plans, and the criteria and process by which FDA will evaluate sponsors’ requests for waivers from the requirement to submit a Diversity Action Plan. | 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: Final Guidance for Industry | This final 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 (EHR) Data in Clinical Investigations: Final Guidance for Industry | This final guidance is intended to assist sponsors, clinical investigators, contract research organizations (CROs), 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 FDA’s Center for Drug Evaluation and Research (CDER) can review COAs and provide advice on the development or modification of COAs outside the Investigational New Drug (IND)/New Drug Application (NDA)/Biologics License Application (BLA) pathway. | Programs |
| Introduction to ICH E19: Selective Collection of Safety Data in Clinical Trials - YouTube Video | This video explains the internationally harmonized guidance on the use of selective safety data collection that may be applied in specific late-stage interventional clinical trials that are pre-approval or post-approval. | Trainings |
| Bringing Clinical Research to Patients Webinar (2023) | The FDA's Office of Women’s Health hosted this webinar in collaboration with the Center for Drug Evaluation and Research (CDER's) Office of Medical Policy to discuss the new strategies, such as Decentralized Clinical Trials (DCTs) and Real-World Evidence (RWE), currently available for bringing clinical research to all patients. | Trainings |
| Challenges, Strategies, and Regulatory Considerations for the Design of Rare Disease Clinical Trials - YouTube Video | As part of the Accelerating Rare disease Cures (ARC) Program, the Center for Drug Evaluation and Research (CDER's) Rare Diseases Team (RDT) inaugurated the Learning and Education to Advance and Empower Rare Disease Drug Developers (LEADER 3D) initiative to better understand and address the unique challenges in bringing rare disease products to the market. This video is the first resource as part of the LEADER 3D effort. The video provides information about demonstrating substantial evidence of effectiveness and approaches to clinical trial designs that are informative to challenges inherent to rare disease drug development. | Trainings |
| Complex Innovative Trial Design (CID) Pilot Meeting Program: The Process - YouTube Video | This video provides an overview of the process for applying for the FDA’s CID Pilot Meeting Program. | Trainings |
| Conducting Clinical Trials with Decentralized Elements: Final Guidance for Industry, Investigators, and Other Interested Parties | This final guidance provides recommendations regarding the implementation of decentralized elements in clinical trials. By enabling remote participation, decentralized clinical trials may enhance convenience for trial participants, reduce the burden on caregivers, expand access to more diverse patient populations, improve trial efficiencies, and facilitate research on rare diseases and diseases affecting populations with limited mobility. | Guidance Documents |
| FDA Guidance Snapshots | Guidance documents contain the Agency’s current best thinking on a particular topic. To support transparent communication and dissemination of FDA guidance documents, FDA launched the Guidance Snapshot Pilot for a subset of cross-cutting guidance documents on topics that seek to modernize drug clinical trials and accelerate drug development. Guidance Snapshots are a communication tool that provide highlights from guidance documents using visuals and plain language. This webpage includes a select list of guidance snapshots to support the efficient application of their respective guidance documents’ recommendations. | Guidance Documents |
| Integrating Randomized Controlled Trials (RCTs) for Drug and Biological Products Into Routine Clinical Practice: Draft Guidance for Industry | This draft guidance is intended to support the conduct of randomized controlled drug trials with streamlined protocols and procedures that focus on essential data collection, allowing integration of research into routine clinical practice. This may improve convenience and accessibility for participants and allow for enrollment of more representative populations, resulting in more generalizable trial results. | Guidance Documents |
| The Role of Artificial Intelligence (AI) in Clinical Trial Design and Research Q&A with FDA Podcast | AI and machine learning are gaining traction in clinical research, changing the clinical trial landscape, and is increasingly being targeted in areas where FDA is actively engaged, including clinical trial design, digital health technologies, and real-world data analytics. This podcast discusses recent advances and the use of technology in clinical trial design. | Trainings |
| Using Bayesian Statistical Approaches to Advance our Ability to Evaluate Drug Products Q&A with FDA Podcast | In this podcast, Jennifer Clark, a Lead Mathematical Statistician in the Center for Drug Evaluation and Research (CDER) at the FDA, discussed the recent changes in the use of Bayesian statistics and the therapeutic areas in which Bayesian statistics can be particularly helpful. | Trainings |