| Patient-Reported Outcome Measures for Post-Acute Sequelae of SARS-CoV-2 | March 2025 |
| Natural History Study of STXBP1 Related Disorders | December 2024 |
| Platform Approach to Patient-Reported Data for Rare Disease Natural History Studies and Therapeutic Research | October 2024 |
| Use of MRI of Joints as Potential Clinical Trial Endpoints in Patients with Rheumatoid Arthritis | June 2013 |
| Use of Hyperpolarized Noble Gas MRI to Measure Lung Function in Patients with Cystic Fibrosis | July 2013 |
| Use of Bone Scan Assessment as a Potential Clinical Trial Endpoint or for Qualification as a Biomarker | July 2013 |
| Use of in vitro Testing to Predict Drug Sensitization Potential | October 2013 |
| Potential Approaches in Drug Development for Adrenomyeloneuropathy | December 2013 |
| Issues in the Laboratory Measurement of Dystrophin | July 2014 |
| Bile Salt Export Pump Inhibition as a Biomarker | May 2015 |
| Use of Disease Progression Modeling for Amyotrophic Lateral Sclerosis | June 2015 |
| Use of a Database of Clinical Trial Data | August 2015 |
| Use of a Database of Genomic and Clinical Data | September 2015 |
| Patient Reported Outcome Assessment for Primary Sclerosing Cholangitis | October 2015 |
| Drug Development for Mitochondrial Diseases | October 2015 |
| Use of Circulating Tumor Biomarkers | October 2015 |
| Patient Reported Outcome Assessment for early stage breast cancer | October 2015 |
| Using Genetic Data to Advance Drug Development | January 2016 |
| A Novel Analytic Method for Pharmacokinetic Data Interpretation | January 2016 |
| A Diagnostic Tool to Advance Cancer Drug Development | January 2016 |
| Approaches to Developing Therapies for Patients with Severely Debilitating and Life Threatening Conditions | February 2016 |
| Biomarkers for Traumatic Brain Injury Clinical Trials | March 2016 |
| Use of a Placebo Response Tool in Clinical Studies | April 2016 |
| Digital Pathology Software System | April 2016 |
| Mucolipidosis Type IV Translational Research | May 2016 |
| Natural History Studies in Primary Sclerosing Cholangitis | June 2016 |
| Development of Release Active Products | July 2016 |
| Multi-System Assessment of Postnatal Development Following Extremely Preterm Birth | July 2016 |
| Pediatric Cholestatic Liver Disease Endpoints | July 2016 |
| Disease Interception Therapy in Rheumatoid Arthritis | July 2016 |
| Symptom Measurement in Hepatocellular Carcinoma | July 2016 |
| Targeting Shared Molecular Etiologies Underlying Multiple Rare Diseases (SaME) | July 2016 |
| Natural History Studies in Angelman Syndrome | July 2016 |
| Update on the Pediatric Trials Consortium | August 2016 |
| Development of a Genomic Database | September 2016 |
| Disease Interception Therapy in Type 1 Diabetes | October 2016 |
| Atopic Dermatitis in Pediatric Patients | October 2016 |
| Disease Interception Strategy in Cataracts and Presbyopia | October 2016 |
| Registry for Pediatric Inflammatory Bowel Disease | November 2016 |
| C1M Assessments in Rheumatoid Arthritis | November 2016 |
| Tool to Evaluate Duplicates in Clinical Trials | December 2016 |
| Raman Spectroscopy to Analyze Compounded Drugs | December 2016 |
| Biomarkers to Identify Drug Induced Pancreatic Injury | January 2017 |
| A Tool to Assess Clinical Trial Site Performance and Data Quality | January 2017 |
| The Role of Inflammation and Immune Response in Duchenne | January 2017 |
| Approaches to Advance Regulatory Science Training | February 2017 |
| Novel Drug Development Tools and Biomarkers to Support Tuberculosis Drug Regimen | March 2017 |
| Development of Innovative Novel Excipients | March 2017 |
| Use of Biomarkers to Enrich for Cardiac Events in Cardiovascular Clinical Trials | March 2017 |
| Use of Virtual Reality Platform in Clinical Trials | March 2017 |
| Development of a Patient Reported Outcome Instrument for Alopecia Areata | April 2017 |
| Pediatric Microdosing Research Using Accelerator Mass Spectrometry (AMS) | May 2017 |
| Challenges Related to Drug Development for Cerebral Cavernous Malformations (CMM) Patient Community | May 2017 |
| Health Related Quality of Life Assessment in Diabetes | June 2017 |
| Physical Activity Accelermetry Assessment for Analgesic Clinical Trials (PAACT) | June 2017 |
| Outcome Measures for Type 1 Diabetes | July 2017 |
| 3D Bioprinted Human Tissue Models | August 2017 |
| Outcome Measures for Type 1 Diabetes | August 2017 |
| Chronic Obstructive Pulmonary Disease (COPD) Assessment Test | August 2017 |
| The Use of Wearable Devices to Collect Endpoint Data in Clinical Trials | September 2017 |
| Outcome Assessments in Acute Pancreatitis | October 2017 |
| Bring Your Own Device PRO Assessments | October 2017 |
| Adrenomyeloneuropathy (AMN) | October 2017 |
| Drug Development Tools for Charcot-Marie Tooth Disease | November 2017 |
| Genetic Prion Disease | November 2017 |
| Clinical Assessment Tools for Alopecia Areata | December 2017 |
| Disease Severity Score for Influenza and other Acute Respiratory Viruses | January 2018 |
| Translational Pharmaceutics based drug development in the US | February 2018 |
| Development of Prophylactic Treatments for Asthma and Other Allergic Diseases | March 2018 |
| Hutchinson-Gilford Progeria Syndrome Biomarker Development | April 2018 |
| Biomarkers and Clinical Outcome Assessments for Fragile X Syndrome | April 2018 |
| A Patient-Centric Approach to Long-term Follow-up Studies | May 2018 |
| Clinical Outcome Assessment tools for Batten Disease | July 2018 |
| Clinical Research Networking Technology Platform | October 2018 |
| Electronic product information in clinical trials | October 2018 |
| A Patient-Centric Approach to Long-term Follow-up Studies | October 2018 |
| Clinical Outcome Assessments for Prader-Willi Syndrome | November 2018 |
| Definitions for clinically meaningful levels of hypoglycemia | December 2018 |
| Drug development tools to aid rare disease natural history development | December 2018 |
| Development of COA through personalized digital health coaching in clinical trials and drug development | February 2019 |
| Voice activated PRO tools for Charcot-Marie-Tooth (CMT) Disease | February 2019 |
| Challenges associated with the Development of Novel Immunosuppressive Drugs (ISD) for use in Kidney Transplantation | March 2019 |
| Personalized oligonucleotide therapies for genetic neurodegenerative diseases | May 2019 |
| Digital drug development tools for Parkinson’s Disease | May 2019 |
| New approaches to analgesic drug development | September 2019 |
| Statistical Approaches to Biosimilar Clinical Study Design | October 2019 |
| Clinical Outcomes for Chronic Obstructive Pulmonary Disease | October 2019 |
| Disease Interception in Non-Small Cell Lung Cancer (NSCLC) | November 2019 |
| Self-Management Self-Test (SMST) in Psychiatric Disorders | December 2019 |
| Master Protocol to Promote Expanded Access in Oncology | February 2020 |
| The Use of the Sequential Parallel Comparison Design in the Development of treatments for Major Depressive Disorder | February 2020 |
| Clinical Outcome Assessments of for Cutaneous Lupus Erythematosus | February 2020 |
| Use of Artificial Intelligence (AI) to created Synthetic Controls in Clinical Trials | March 2020 |
| Clinically Meaningful Endpoints for Wound Healing Clinical Trials | June 2020 |
| Advancing Development of Therapies for Spinal Muscular Atrophy | August 2020 |
| Goal Attainment Scaling (GAS) | August 2020 |
| Mass-Action Law Pharmacodynamics (MAL-PD) | October 2020 |
| Cyclin Dependent Kinase-like 5 (CDKL5) Deficiency Disorder (CDD) Observational Study | December 2020 |
| The Use of Serum Neurofilament-light (sNfl) in Progressive Multiple Sclerosis (MS) Trials | December 2020 |
| Embedding Qualitative Patient Interviews in Neurofibromatosis Clinical Trials to Facilitate Drug Development | February 2021 |
| Down Syndrome Associated Alzheimer’s Disease (DS-AD) | March 2021 |
| Fabry Disease Structural Endpoints for Assessment of Drug Efficacy in Fabry Nephropathy clinical Trials | April 2021 |
| Optimizing pain measurements in neonates to expedite analgesic drug development | June 2021 |
| Obtaining Reliability and Acceptance of Novel Endpoints using Digital Health Technologies | July 2021 |
| Basket Trials to Study Sleep Wake Instability across Multiple Rare Disorders | November 2021 |
| Innovative Conceptual Approaches to Clinical Trial Design and Analysis for an Integrated Research Platform in Non-Alcoholic Steatohepatitis (NASH) | January 2022 |
| Rare Genetic Dilated Cardiomyopathy | February 2022 |
| Extracellular RNA Splice Variant Biomarkers of Myotonic Dystrophy | April 2022 |
| Use of Digital Pathology in Good Laboratory Practice (GLP) Environment for Nonclinical Studies | June 2022 |
| Advancing Nocturnal Scratch as a Digital Endpoint for Atopic Dermatitis | July 2022 |
| Transfer RNA-Readthrough Treatment for Rare Genetic Diseases | September 2022 |
| Advanced Computational and in-vitro modeling in Drug Discovery & Drug Development | January 2023 |
| Sjögren’s Tool for Assessing Response (STAR) | February 2023 |
| Artificial Intelligence (AI) image analysis software for supporting the evaluation of oncology drug efficacy in clinical trials | June 2023 |
| Precision Psychiatry Approach to Clinical Development | September 2023 |
| Natural History Studies for Primary Sclerosing Cholangitis | October 2023 |
| Oral Tyramine Challenge Testing of MAO Inhibitors | January 2024 |
| Virtual Clinical Trials | March 2024 |
| Electroencephalography (EEG)-based artificial intelligence (AI)-powered method to improve the efficiency of clinical trials of Major Depressive Disorder (MDD) | July 2024 |
| Targeting Condensates to Treat Disease: Implications, Opportunities and Challenges | September 2024 |
