FDA, Sponsors Look to Expand Patient Input to Clinical Trials
Applied Clinical Trials
Patient focused drug development is playing a larger role in study design and outcome measurement.
Patient focused drug development (PFDD) is moving into the mainstream, promising to alter the conduct of clinical trials and FDA regulatory policies. PFDD appears increasingly useful and accepted in designing studies and in assessing outcomes and treatment benefits most important to patients with a certain condition. Building on the experience gained from a series of FDA meetings to solicit patient perspectives for treating chronic conditions, sponsors are querying patient groups to help define the key goals of clinical studies and to avoid research programs that yield less useful results. FDA is bringing in patients to consult with review divisions and to join FDA-sponsor meetings to help shape research studies and product labeling.
To further this trend, FDA is expanding its patient representative program beyond participation in advisory committee meetings. The agency has identified some 200 patient representatives based on their experience, FDA training, and clearance on conflicts of interest. Last year, members of this cadre were involved in 10 consultations with FDA review divisions and in additional meetings with sponsors, explained Richard Klein, head of FDA's patient liaison program in the Office of Health and Constituent Affairs. Qualified patient representatives not only have experience with a disease or condition, but are active in patient advocacy organizations, knowledgeable about treatment options, and able to grasp basic scientific principles, Klein pointed out at the recent conference on PFDD sponsored by the University of Maryland Center of Excellence in Regulatory Science & Innovation.
One FDA initiative is to develop a "roadmap" to patient-focused outcome measurement in clinical trials, reported Ashley Slagle of the Office of New Drug (OND) Study Endpoints and Labeling Development staff in the Center for Drug Evaluation and Research (CDER). The roadmap aims to establish an orderly pathway for selecting or developing instruments to accurately measure treatment benefit, she explained. Key criteria are the natural history of the disease or condition, the affected patient population, treatment alternatives, and current care standards.
Behind the Buzz: Why Clinical Research Leaders Flock to SCOPE Summit
February 7th 2025In this episode, we meet with Micah Lieberman, Executive Conference Director for SCOPE Summit (Summit for Clinical Ops Executives) at Cambridge Innovation Institute. We will dive deep into the critical role of collaboration within the clinical research ecosystem. How do we bring together diverse stakeholders—sponsors, CROs, clinical trial tech innovators, suppliers, patients, sites, advocacy organizations, investors, and non-profits—to share best practices in trial design, program planning, innovation, and clinical operations? We’ll explore why it’s vital for thought leaders to step beyond their own organizations and learn from others, exchanging ideas that drive advancements in clinical research. Additionally, we’ll discuss the pivotal role of scientific conferences like SCOPE Summit in fostering these essential connections and collaborations, helping shape the future of clinical trials. Join us as we uncover how collective wisdom and cross-industry partnerships are transforming the landscape of clinical research.
A Guide to Patient Engagement & Recruitment Solutions
March 18th 2025Clinical trials have become increasingly complex over the past decades, making participant recruitment more challenging than ever. This guide explores common recruitment obstacles and offers strategies to overcome them using a patient engagement and recruitment solution.
Reaching Diverse Patient Populations With Personalized Treatment Methods
January 20th 2025Daejin Abidoye, head of solid tumors, oncology development, AbbVie, discusses a number of topics around diversity in clinical research including industry’s greatest challenges in reaching diverse patient populations, personalized treatment methods, recruitment strategies, and more.
Bridging the Gap: How Chiesi Used External Collaboration to Get Closer to Patients
March 18th 2025Chiesi partnered with Citeline to enhance clinical trial awareness and patient engagement through its FREUD initiative by launching a Trial Portfolio Website, activating 150+ sites, and generating 600+ qualified referrals—learn more about how Citeline helped improve study discoverability, streamline recruitment, and strengthen Chiesi’s presence in key therapeutic areas.
Citeline Patient Engagement & Recruitment Solutions
March 18th 2025Watch Citeline’s 15-minute presentation from Clinical Leader’s Patient Engagement & Recruitment Solutions Expo, where VP Matt Holms explores Direct-to-Data (DTD) patient recruitment. This session takes a deep dive into how complex algorithms, tokenized data, and large data assets can optimize enrollment, highlighting the importance of early strategic recruitment planning and breaking down the unique benefits and real-world applications of DTD.
