Connecting Drug Innovation & Patient Voices

Bringing patient perspectives into drug development and innovation is critical. The voices of patients, their caregivers, and their treating physicians can help us develop better, more impactful drugs in oncology.

Today in January 2024, FDA hosted its “Conversations on Cancer: Brining Innovation to People Facing Cancer” event – a unique web-based panel geared to connect the physician and patient perspectives with new oncology drug product approvals and product launches. The focus of this first panel was a recap of the 55 oncology approvals-- both innovator drugs and label expansions.

What is the Conversations on Cancer Series

The series is hosted by the FDA Oncology Center of Excellence (COE) and focuses on a range of webinar panel topics on oncology innovation.

Interested in learning more about the Conversation on Cancer and other Oncology COE events, click here.

January 2024 Conversation On Cancer Event

The panel kicked off with Dr. Margret Marino, Clinical Reviewer and Pediatric Hematologist-Oncologist as FDA, leading a discussion across a diverse FDA, physician, and patient panel on perspectives on patient experiences with new oncology therapies entering the market. Bernadette Sanger, a large B-cell lymphoma patient, emphasizes that new immunotherapies. Dr. Sonia Smith called newly approved immunotherapies a “a walking miracle” that helps us to envision a future where chemotherapy may not be needed.

Immunotherapies are a "a walking miracle" for cancer patients compared to existing chemotherapy drugs

Oncology Drug Approvals in 2023: the Good, the Bad, & the Ugly

Melanie Royce at FDA asked patients and clinicians about “the good, the bad, and the ugly” experiences for desmoid tumor patients. Amand Hosman, patient advocate for the Desmoid Tumor Foundation and patient herself, emphasized that new therapies for desmoid tumors approved in 2023 “changes my life,” and the Expanded Access program was essential in allowing her to access a life changing drug prior to its approval. She called the recent clinical trials “a really beautiful thing” that links research innovations with patients. Melanie Royce emphasized that, for cancer patients, clinical trials can be a meaningful way for patients to access new oncology therapeutics.

Clinical trials are a "really beautiful thing" that brings innovative ne therapies to patients

Dr. Mirinal Gounder from Memorial Sloan Cancer Center, who specializes in sarcoma clinical treatment, shared his experiences in how real world data (retrospectives) and clinical trials can be combined to take existing oncology drugs into new cancer types.

Learn more about the 55 oncology drug approvals in 2023 here.

The Role of Accelerated Approval in Modern Oncology

The physicians on the call emphasizes that Accelerated Approval is critical to oncology patients. Dr. Richard Pazdur, the director of the FDA's Oncology Center of Excellence (OCE), spoke to the role of the AIDS community in the 1990s Advocacy in shaping the regulatory landscape for the modern Accelerated Approval program, in particular approval of drugs on the basis of strong Phase 2 data and surrogate endpoints—in fact now about 70% of Accelerated Approvals are in oncology. He shared about his career experience from the 1980s to present, and the importance of speeding approvals to cancer patients.

About 70% of all Accelerated Approvals are in Oncology-- even though in the 1990s the program was first conceptualized for treating infectious diseases like AIDS

Does Social Media Play a Role in Oncology Patient Experience and Willingness to Try New Drugs?

Sandra Collines, a Desmoid tumor patient, and the FDA team had a great conversation on the power of social media – and the generational challenges—in leveraging social media to reach oncology patients and change perspectives on innovation. Dr. Vamsi Velcheti described that social media is a “challenge and an opportunity” in patient care, but a key challenge is who should professionals be reaching out to on advice for how to use social media and address information asymmetry.

For Oncology Blockbusters, Drug Innovation Doesn't Stop at Approval

Dr. Richard Pazdur commented that “drug approval is not the end, it [regulatory processes] continues on”. He described an example of patient reported safety issues in oncology, osteonecrosis of the jaw—which is now well-characterized, but was originally ‘discovered’ by a dental professional. He notes that patients being verbal to their doctors about adverse effects is critical to drug development and innovation, and oncology patients can be fearful of having the drug taken away from them or fearing ‘losing the fight’ with cancer. “Many times drug companies say over and over, the side effects are ‘manageable, manageable, manageable… what does that mean? I mean death is manageable, we call that undertakers… we don’t want to have filtered [safety] information from a drug company or people minimizing their experience”.

“Many times drug companies say over and over, the side effects are ‘manageable, manageable, manageable… what does that mean? I mean death is manageable, we call that undertakers… we don’t want to have filtered [safety] information from a drug company or people minimizing their experience”

Are Diagnostics and Biomarker Tests Accessible to All Patients?

Dr. Richard Pazdur also emphasized the advances in molecular testing, which may be approved but are not realistically happening in small, rural settings when patients are diagnosed with cancer “we can have the biggest and best innovation, and they are not reaching people.” Dr. Vamsi Velcheti noted that data shows as many as 20% of cancer patients that should be getting tumor testing are not in practice.

as many as 20% of cancer patients that should be getting tumor [diagnostic/biomarker] testing are not in practice

Dr. Mrinal Gounder shared his experiences that in very rare cancers, patients may be over-tested and know their biomarkers, but they live in small towns where they don’t have access to clinical trials and centers of expertise that match their biomarkers. He emphasizes that remote clinical trial technology and monitoring may enable us to start to bridge this gap.

Patient-Focused Events - Should they be a Priority for Professional Development in Industry teams?

In industry, brining the voice of the patient into drug development is essential to ensure we optimize the science, trial design, and outcomes for these populations-- and that we prioritize label expansions and innovator programs that will be most impactful for oncology patients and caregivers. These webinars provide a resource drug development teams can use to develop their teams and bring them closer to patients.

And lastly, for their valuable time and expertise -- congrats and thank you to the patients and physicians who joined FDA on this call to help bring these insights to a wider patient, healthcare, and industry audience, including:

Patient Advocacy & Patients

·?????? Amand Hosman, Desmoid Tumor Foundation and patient

·?????? Sandra Collins, Desmoid tumor patient

·?????? Bernadette Sanger Large B-cell Lymphoma patient

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Clinical Experts

·?????? Vamsi Velcheti NYU, thoracic malignancies

·?????? Mirinal Gounder, Memorial Sloan Cancer Center Sarcoma specialist

·?????? Sonali Smooth, University of Chicago hematologic oncology

How do you incorporate the voice of the patient in your innovation processes? Jump in the comments and let me know your thoughts.

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Angela Johnson, PhD, RAC is the head of global regulatory compliance for Cytiva and lecturer on regulatory strategy at Northeastern University. She has lead two venture-backed biotech regulatory teams to IPO and held leadership roles at IQVIA, GE Healthcare, and APAC consulting firms in drug development, and is an active member of the Regulatory Affairs Professionals Society (RAPS) and regulatory board member of the American Society of Cell & Gene Therapy (ASGCT), with more than 30 publications on regulatory and drug development.