The Evidence Forum

Using Patient Experience Data to Demonstrate the Value of CAR-T Cell Therapies in Clinical Trials of Hematological Cancers

Recent Trends in Drug Safety: The Growing and Evolving Role of Real-World Evidence

Pediatric Drug Development: Trends and Perspectives in the European Union

Advancing Patient-centered Approaches to Oncology Studies: Exploring an Example

The Evolution of Technology Enablement in Long-term Follow-up Studies

Overcoming Challenges in Real-World External Control Arm Design

Network Meta-Analysis of RWE: The Challenge of Related Publications

Advancing Treatments for Chronic Kidney Disease and Rare Renal Disorders in Adults and Children: Trends and Opportunities

Advancing Rare Disease Treatments in the COVID-19 Era and Beyond

The Evolution of Pregnancy and Lactation Safety Studies

Reimbursement Landscape for NGS

Emergence of SARS-CoV-2 Variants

Reshaping the Future of Consumer Health

Mini-Global Value Dossiers (GVDs)

The Need for Flexible Pregnancy Safety Studies

Emergency Use Authorization and Antigen Diagnostic Tests for COVID-19

Enhancing Clinical Trial Diversity in the Era of Decentralized Trials

A New Framework for Modeling Disease-Modifying Treatment Strategies for Parkinson’s Disease

Implementation Science: A Primer

What You Need to Know About Real-World Databases in Japan

Pre-Approval Information Exchange (PIE)

Four Common Missed Opportunities When Designing and Developing a REMS Program

Everything Old is New Again

Post-Marketing Safety Registries: What, When, Why, How?

Health Technology Assessment During the COVID-19 Pandemic

Supporting Clinical Trial Site Selection During the COVID-19 Pandemic Using WAVE, a Custom Hybrid Epidemiologic Model

Transformation of Clinical Trial Design and Operations: Interviews with Science 37, Medable and Takeda

COVID-19 Symptom Reports: Comparing and Contrasting Three Groups of Patients

An Initial Framework to Describe and Classify Integrated Scientific Advice Procedures

Rare Diseases and Orphan Drugs: Where are We Now?

Prevent or Wait and Treat: Modeling Health Conditions with Preventive and Acute Therapeutic Options in R

Pediatric Drug Development: Trends and Perspectives in the United States

Can Clinical Studies Go Virtual?

Proactive Management of Study Complexity and Amendment Risks Can Return Millions on the Investment

How Health-Related Social Media Can Complement Traditional Real-World Evidence Approaches to Offer Unique Patient Insights

Value is in the Eye of the Beholder

Advancing Early Stage Oncology Research with Adaptive Designs and Master Protocols

Incorporating Innovation into the 505(b)(2) Development Pathway

Digital Approaches in the Era of COVID-19

Leveraging Decentralized RWE Data Collection Strategies during the COVID-19 Pandemic and Beyond

A Systematic Approach to Assessing Real‑World Research Questions Supported by Digital Enablement

Leveraging Simulation to Patch a Clinical Trial Broken by COVID-19

Evidence Generation Using Innovative, Technology-Driven Data Collection

Surprises You Don’t Want When Adopting eCOAs for Use in Clinical Trials: Cautions for Decision Making and Planning

Digital Therapeutics:
Past, Present, and Future

Integrated Scientific Advice during the COVID-19 Pandemic

Trends in Medical Device Purchasing, Evaluation of Value, and Advice for Manufacturers

Patient Preference Studies in HTA Decision Making: A NICE to Have?

Version 4.1 of the AMCP Format: Introducing a Trio of Dossiers to Support a Product Throughout its Lifecycle

The Role of Integrated Scientific Advice for the Early Determination of RWE Requirements in HTA and Payer Assessments

Pragmatic Randomized Trials: Considerations for Design and Implementation

Research Operations for Secondary Use of Clinical Sites’ EMR

Advancing Healthcare through Innovation and Collaboration

Increase Study Awareness and Patient Engagement with the Use of Branded Study Materials

Virtual Trials and Real-World Evidence Data Collection: Identifying Core Needs and Defining “Virtual Trials”

Protocol Design in Real-World Evidence: The Indispensable Link Between Strategic Need and Study Execution

Enhancing Patient Centricity of HTA: Opportunities in Europe

Patient Preferences in Health Technology Assessment in Europe: Recent Advances and Future Potential

The Growing Need for Real-World Evidence in Medical Devices

The Growing Influence of the Institute of Clinical and Economic Review on Payer Decisions in the US

Health Economics in China: Changing Pharmaceutical Pricing and Reimbursement

Natural History Studies in Rare Diseases and Genetic Biomarkers

How Social Media Can Be Used to Understand What Matters to People with Rare Diseases

FDA Updates Draft Guidance on Rare Diseases: Some Key Takeaways You Need to Know

Patient Engagement in Clinical Trial Protocol Design and Recruitment Strategies: What Does It Mean for Orphan Drug Manufacturers?

Evidence Requirements for Orphan Drugs from a Payer Perspective: How Can Early Scientific Advice Help?

Can Early Collaboration between Multiple European Stakeholders Increase Access for Patients Living with Rare Diseases?

Clinical Outcome Assessment Selection for Rare Disease Trial Programs

Adapting an Existing Instrument for a Rare Disease: A Valuable Resource within Your Reach

Registries in Rare Disease Research: Approaches to Optimize Success

More Evidence Needed for Orphan Drugs in Germany: A Hurdle for Access and an Opportunity for Real-World Evidence

Rare Disease Treatments Aren’t So Rare Any More

The EU HTA Harmonization Initiative: What is the Significance to Manufacturers of the New Directive?

Bucher versus Bayesian NMA Approaches for Indirect Treatment Comparisons

Clinical Trial Simulation in Early Market Access Planning

Meaningful Treatment Benefit from the Patient’s Perspective

Machine Learning – Biopharma Applications

Disease Simulation in Drug Development – External Validation Confirms Benefit

New Trends in Drug Safety

How Similar is Similar? A Deeper Dive into Bucher vs. Bayesian NMA

HTA Scientific Advice – Is it Becoming More Important?

Early Access Programs: Opportunities and Challenges for Real-World Data Collection

Are Patients at the Center of Your Trials? Using Patient Engagement and Insights to Improve Clinical Research

Market Access – Is Your Healthcare Communication and Data Dissemination Strategy the Missing Piece?

Pre-Approval Communication of Health Care Economic Information to U.S. Payers: Opening Doors for Patient Access

Is Real-World Evidence Needed in Comparative Effectiveness Research? Yes, But …

Market Access Policy: EU HTA – Looking Back to Better See Ahead

The First Re-Evaluations in France Based on Real-World Evidence: Another Good Example of Starting with the End in Mind

Pregnancy Registries and Lactation Studies: Best Practices to Support Product Labeling

Using Qualified PRO Measures in Drug Development: An Update on the EXACT and E-RS

The Drive Toward Pragmatism in Randomized Trials

21st Century Cures Act Innovation, Breakthroughs, and Research in Under-Represented Populations

Leveraging Real-World Evidence for Regenerative Medicine and Advanced Therapy Success Beyond the Regulator

A Perspective on the 21st Century Cures Act: Patient-Focused Drug Development

U.S. Presidential Mandate on Value-Based Drug Pricing – Moonshot or Wormhole?

Value Frameworks: Will They Work in the U.S.? What are Stakeholders Saying?

Access Options for Investigational Products: Key Considerations

Wearable Devices and Mobile Technology in Clinical Trials

Real-World Studies Need Patients Too! Unique Considerations for Patient Engagement and Retention