In the rapidly advancing world of cell and gene therapy, a single treatment can alter a patient’s life trajectory. But what happens after the initial success? For many, the journey is just beginning. Long-term follow-up (LTFU) is not just a regulatory requirement—it’s a vital, ethical commitment to patients who become pioneers of new treatments. Yet, conducting this follow-up poses immense logistical, financial, and emotional challenges.
To explore this essential but often overlooked aspect of cell and gene therapy, the June 2025 Bridging the Gap webinar welcomed Dr. Carolyn Riley Chapman, a faculty member at Harvard Medical School and a lead investigator at the Multi-Regional Clinical Trial (MRCT) Center of Brigham and Women’s Hospital. Dr. Chapman’s insights were enriched by her background in genetics, bioethics, and policy development, and she currently leads a stakeholder initiative addressing LTFU challenges.
She was joined by moderator Albert Ribickas, Assistant Director of the Cell Therapy Facility at Moffitt Cancer Center, with recurring hosts Olga Bukatova from Azenta Life Sciences, Tom Whitehead from the Emily Whitehead Foundation, and guest panelists including George Eastwood and Dr. Patrick Hanley of Children’s National Hospital.
Why Long-Term Follow-Up Matters
As Dr. Chapman explained, “Gene therapies offer long-term hope, but also carry long-term risk. That’s why robust follow-up is not just recommended, but essential.”
Regulatory agencies require these extended studies – often spanning 10 to 15 years – to track long-term outcomes such as cancer risk, immunologic effects, or toxicities. But the real burden, she emphasizes, falls on patients and their families, who must remain engaged with the healthcare system for years, frequently without any immediate personal benefit. The logistical challenges for patients of maintaining compliance over such a prolonged period can be substantial.
“Things change over time—people move, sites lose staff, technologies evolve,” Dr. Chapman said. “We need systems that keep pace without overburdening patients.”
“Indeed, from a technology perspective, ensuring that biological samples, data, and even product handling remain consistent across a 15 year timeline is also an important challenge, and one where technology providers can contribute by helping future-proof those processes,” Bukatova added.
Tom Whitehead shared how his daughter Emily, the first pediatric CAR-T recipient, has embraced her role in research, but he acknowledged the unique difficulties many families face. “It only takes one bad appointment for a patient to never return,” he warned. He praised centers like CHOP for streamlining follow-ups to reduce that friction.
What Makes Follow-Up Work: Equity, Design, and Data
Panelists agreed that patient-centric design must be the cornerstone of any follow-up strategy. As George Eastwood put it, “We have to ask: how do we scale this in a way that respects each patient’s journey?”
Dr. Chapman introduced several promising approaches:
- Decentralized elements like apps, EMR integration, and telemedicine to reduce travel burdens.
- Patient-reported outcomes that reflect quality of life, not just safety metrics.
- Standardization of data collection across trials to enable broader impact and insight.
“There’s a real opportunity to harmonize outcomes and learn across products and diseases,” Dr. Chapman explained.
Who Should Bear the Burden?
Dr. Patrick Hanley raised a pressing ethical question: “Who pays for long-term follow-up when insurance coverage runs out?” He recounted a panel where patient advocate Jimmy Olaghere challenged experts to think beyond treatment delivery and consider sustained care over decades.
“This responsibility often falls on sponsors, but there’s a gap when trials originate from academia or smaller biotech efforts,” Dr. Chapman added. “We need solutions that ensure no patient is left unsupported.”
Dr. Chapman described this as the “Goldilocks Problem”: striking the right balance between maximizing value and minimizing burden — not too much, not too little, but just right. She emphasized that setting reasonable expectations for long-term follow-up is essential, not just for patients, but for every stakeholder involved.
Balancing Ethics and Innovation
The webinar also touched on emerging debates around accelerated access and ethical risk. Dr. Chapman referenced the newly appointed FDA Commissioner Marty Makary’s recent suggestion of a conditional approval pathway for gene therapies in ultra-rare diseases. “It’s a provocative idea,” she admitted. “But we have to carefully define under what conditions ethical flexibility is justified.”
Tom Whitehead emphasized the real-world urgency: “If the choice is hospice or trying something new, we should make that easier. Many patients don’t have time to wait for approvals.”
Key Takeaways from the Session
- Long-term follow-up is vital to both patient safety and broader learning but often lacks sufficient structural support.
- Patients face logistical and emotional burdens that can limit follow-up participation, particularly over 10-15 year timelines.
- Patient-centered strategies—including remote tools, streamlined protocols, and incentives—can improve engagement and equity.
- Data harmonization across trials could multiply the impact of LTFU and accelerate innovation.
- Ethical frameworks must adapt to ensure timely access while preserving safety and trust in an evolving therapeutic landscape.
Stay Connected
Want to hear more from the experts shaping the future of cell and gene therapy?
Register to watch this webinar on demand and receive updates about upcoming sessions in the Bridging the Gap series. Don’t miss the next installment as we continue to explore the science, policy, and human stories driving advanced therapies forward.
About the Guest Speaker
Carolyn Riley Chapman, Ph.D. MS.
Lead Investigator, Multi-Regional Clinical Trials (MRCT) Center of Brigham and Women’s Hospital
Dr. Carolyn Riley Chapman is a Lead Investigator at the Multi-Regional Clinical Trials (MRCT) Center of Brigham and Women’s Hospital and a faculty member at Harvard Medical School. Her work focuses on addressing ethical, regulatory, and logistical challenges in precision medicine research, including cell and gene therapies.
Prior to joining MRCT, she was on faculty at NYU Grossman School of Medicine, where she held roles in research ethics education and medical ethics. She has also served as Associate Director for Columbia’s Bioethics program, worked in biotech strategy at L.E.K. Consulting, and held leadership roles at Aton Pharma. Dr. Chapman holds a Ph.D. in Genetics from Harvard, an M.S. in Bioethics from Columbia, and a B.A. in Biology from Dartmouth, where she graduated summa cum laude. She completed a postdoctoral fellowship in medical ethics at NYU and holds a graduate certificate in survey research from the University of Connecticut.