Sponsors in the US, the EU, and in some select cases Japan, are able to apply for Accelerated Approval from regulators based on a surrogate endpoint, using less clinical data than would typically be required. This approach is intended to speed products to market that treat serious or life-threatening conditions – like cancer – and that offer a benefit over current treatments. As a result, patients have benefited from earlier access to critical medications, and sponsors have enjoyed the accompanying market advantage.
Since the first oncology treatment was approved via the Accelerated Approval process in 1994, the Accelerated Approval program has been instrumental in the timely approval of a large number of cancer therapies, moving from more established types of therapy such as hormonal and chemotherapy agents to novel classes of cancer therapy such as monoclonal antibodies, tyrosine kinase inhibitors, and immune checkpoint inhibitors.
A study performed on drugs approved between 1995 and 2008 found that, indeed, the median development time for nonbiologic NMEs was markedly shorter via Accelerated Approval than Regular Approval. Another benefit to Accelerated Approval for sponsors is that they are able to consult with the FDA more often and at higher levels within the agency throughout the development process. In addition to considerations on the required clinical trials, the FDA is often willing to make some other concessions in what is required in the Chemistry, Manufacturing & Controls (CMC) package, production requirements, and the post-approval commitments for the New Drug Application (NDA) or the Biologics License Application (BLA) under the Accelerated Approval program.
The pursuit of the Accelerated Approval development path are not without risk, however, and the decision to seek this program should be made carefully with a complete understanding of the regulatory, medical, and logistical implications. The greatest risk for sponsors in seeking Accelerated Approval using a surrogate endpoint is that the confirmatory clinical trial might fail to confirm the intended clinical benefit or reveals a new safety issue not detected in the earlier smaller trial population. Challenges in population selection, endpoint selection, diagnostic assay, and trial design can all be barriers to success for a promising asset in successfully achieving Accelerated Approval. Even after Accelerated Approval is achieved, full marketing approval remains a challenge as confirmatory trials compete with a newly marketed agent.
In this webinar we review a number of the benefits and considerations related to pursuing accelerated approval, offer advice on how to access the applicable expedited program, effectively interact with the Agency, follow the regulatory pathway most expeditiously, and highlight examples that illustrate both the advantages and challenges of pursuing Accelerated Approval.
- Understand expedited development/review options in the U.S. and EU
- Identify potential benefits and risks of pursuing accelerated approval in oncology indications
- Learn about the planning process for pursuing accelerated approval
- Learn about how to execute trials designed for the accelerated approval pathway
Robert Millham, MS, MSc, PharmMed, Senior Vice President & General Manager, Hematology & Oncology, inVentiv Health
Robert Millham is the Therapeutic Area Leader for Hematology and Oncology. He has 17 years of Pharmaceutical industry experience, in roles including laboratory, clinical development and clinical operations. His most recent role before joining inVentiv Health was Vice President, Oncology Clinical Operations, in Pfizer Worldwide Research & Development. In that role, he oversaw the provision of operations support for clinical trials for Pfizer’s Oncology portfolio from phase I through IV. Robert was with Pfizer from 1998 through 2015, working in laboratories on biomarker development and validation. He joined Pfizer Oncology in 2004, and served in roles including Translational Medicine Lead, Clinical Lead, and Medical Team Lead before assuming his present role in Development Operations in 2013. Prior to joining Pfizer, he worked for companies in Biotech and Agri-Biotech. He is an author or co-author on eight peer-reviewed publications and over 20 peer-reviewed abstracts and presentations. He started his professional career as a high school chemistry and biology teacher, first in the suburbs of Chicago and later in northern Vermont. Robert holds degrees in biology (Middlebury College), microbiology (University of New Hampshire) and Pharmaceutical medicine (Hibernia College).
Carolyn Finkle, MSc, Senior Vice President, Regulatory Affairs, inVentiv Health
Carolyn Finkle has over 28 years scientific and industry experience including 18 years in management of drug and biologic development, quality and regulatory affairs for companies in the U.S. and Canada, including VP, Global Regulatory Affairs, Catalent Pharma Solutions, Senior Director, International Regulatory Affairs, AstraZeneca, Vice President, Global Product Development Strategy and VP North America Consulting, PAREXEL International, VP Regulatory Affairs at Celsion Corporation, VP Preclinical Development at TherImmune Research Corporation, VP Drug Development at GeminX Biotechnologies, Director of Preclinical Development at ConjuChem and Manager of Preclinical Development at BioChem Pharma (acquired by Shire).
Carolyn Finkle holds a M.Sc. Chemistry from the University of Toronto, Canada and has a B.Sc. Chemistry from the University of Ottawa, Canada. She has worked in academic research at Stanford University, the University of Toronto, Max Bell Research Institute, Toronto General Hospital and Chiba University, Japan, prior to her industry managerial appointments.
Carolyn Finkle currently serves as faculty for UCSF-Stanford Center of Excellence in Regulatory Science and Innovation (CERSI) Graduate Course: Regulatory Considerations for Drug Development: Phase 2 and Phase 3 for Approval, Georgetown University Adjunct Lecturer and graduate course co-director of the Georgetown University Master’s Program in Clinical and Translational Research and Course Director of the Pharmaceutical Education and Research Institute (PERI), as well as, contributor to the UCSF American Course of Drug Development and Regulatory Sciences (ACDRS). Carolyn has co-authored multiple white papers on biosimilarity, accelerated approval, 1 book chapter on First-in-Human global regulatory requirements, over 18 abstracts and 14 papers for industry associations and scientific publications and chaired DIA sessions (global regulatory) at the annual meetings. She has received numerous awards and scholarships throughout her career.
Joseph O’Connell, MD, Vice President, Medical & Scientific Affairs, Hematology & Oncology, inVentiv Health
Dr. O’Connell completed his Medical Oncology Fellowship at Memorial Sloan Kettering, where his clinical development experience included work on Phase 1-3 studies in hematologic malignancies and solid tumors, and key studies in the development of effective anti-emetics to support delivery of chemotherapy in the outpatient setting. Thereafter, he practiced adult medical oncology for over 15 years in community and academic settings, the latter as a member of the Thoracic Oncology Clinic of Yale Comprehensive Cancer Center. Dr. O’Connell served as the president of the Connecticut State ASCO Affiliate, where initiatives included promoting payer and provider dialog on pay-for-performance initiatives and representing the Association in State Legislature testimony in support of access for underinsured patients.
Dr. O’Connell joined Pfizer in 2007 in Clinical Development, leading several Phase 1-3 studies for major late-stage assets. Responsibilities included formulation of clinical development plans, opinion leader engagement across North America, Asia and Europe, face-to-face global regulatory discussions, oversight of safety monitoring, input to commercial and market access efforts including launch planning, and participation in business development review. Thereafter, Dr. O’Connell worked in Medical Affair for Pfizer in a field-based role interacting with community and academic HCPs, US payers, and national pathway organizations.
Presently, Dr. O’Connell is VP/ Global Therapeutic Head, Hematology-Oncology division of inVentiv Health Clinical, a role which includes strategic input for large and small Pharmaceutical developers for Phase I-IV protocols, overall development plans, and support of market access discussions.
Who Should Attend?
Senior level professionals from biopharmaceutical companies working within:
inVentiv Health is a global professional services organization designed to help the bioPharmaceutical industry accelerate the delivery of much-needed therapies to market. Our combined Clinical Research Organization (CRO) and Contract Commercial Organization (CCO) offer a differentiated suite of services, processes and integrated solutions that improve client performance. With more than 14,000 employees and the ability to operate in more than 90 countries, we enable clients to successfully navigate an increasingly complex environment. For more information, visit inVentivHealth.com.