Biosimilars have come to the forefront of discussion because of their potential to reduce health care costs (based on an extrapolation of the cost benefits from generic small-molecule drugs) and the inclusion of language establishing an abbreviated approval pathway for biosimilars in the Patient Protection and Affordable Care Act (PPACA), for which the US Food and Drug Administration (FDA) is expected to release general guidance this year. However, there are significant scientific and manufacturing challenges to ensuring that a biosimilar is equivalent to the innovator product. Additionally, the FDA has not yet developed a robust biosimilar approval pathway, raising questions regarding the types of studies (pre-clinical and clinical) that would be required to gain FDA approval. Furthermore, biosimilars exude safety concerns related to immunogenicity. Coupling these concerns with a skepticism regarding whether or not there is equivalent efficacy compared to the reference product, clinicians may be hesitant to utilize biosimilars. Potentially, a low uptake of biosimilars and a heavy investment in biosimilar development may minimize the cost benefit derived from a lower acquisition price of biosimilars.
The use of biologics is widespread in the active treatment of cancer and for supportive care management. As the use of biologics continues to grow and older biologics come off patent, consideration will need to be given to the incorporation of biosimilars into clinical practice and the safety and outcomes of patients with cancer. As the FDA implements elements of the BPCI Act, stakeholders must actively engage in discussions to ensure biosimilars are safe and effective for the treatment of patients with cancer.
On Friday, April 29, 2011, NCCN convened the NCCN Oncology Policy Summit: Biosimilars – Regulatory, Scientific, and Patient Safety Perspective in Washington, DC at the National Press Club. This invite-only Policy Summit was attended by patient advocates, providers, payors, employers, government, and industry representatives within the oncology community. These key stakeholders gathered for a day-long session to discuss current policy issues surrounding this important topic.
The Summit featured two expert roundtable discussions, moderated by William T. McGivney, PhD, Chief Executive Officer of NCCN, and several presentations by members of the NCCN Biosimilars Work Group, comprised of thought leaders from NCCN Member Institutions and external organizations, representing providers (physicians, pharmacists, and nurses), patients, manufacturers, payors, and government, who worked over the past several months to develop guidance regarding challenges of biosimilars to health care providers and other stakeholders. The Work Group’s efforts will culminate in a white paper later this year.
Andrew D. Zelenetz, MD, Chief of the Lymphoma Service at Memorial Sloan-Kettering Cancer Center and Chair of the NCCN Biosimilars Work Group, provided an overview of the role of biologics and biosimilars in oncology, the difference between biologics and small molecule drugs, and highlighted some important areas of stakeholder interest regarding biosimilars, including but not limited to preservation of innovation, safety and efficacy, availability of data for a wide variety of indications, access to medications, affordability, and how to extrapolate clinical utility from efficacy data.
During the second presentation, James M. Hoffman, PharmD, MS, BCPS, Medication Outcomes & Safety Officer at St. Jude Children’s Research Hospital and Work Group member, discussed the challenges and recommendations compiled by the Work Group, which will be further explored in the aforementioned white paper. It was noted that the Work Group believes that the overall goal of biosimilars is to increase affordability and access to biologic medications for patients, which are often important therapies for cancer care. Dr. Hoffman also expressed the Work Group’s support for defining a biosimilars approval pathway, which has been characterized in the Biologics Price Competition and Innovation Act of 2009 subtitle of the PPACA. Other recommendations addressed included education for patients, clinicians, and policy-makers as well as safety and efficacy of biosimilars.
The first expert roundtable panel addressed areas related to safety, patient, clinician, and payor issues. Panelists representing these areas discussed how biosimilars will be integrated into oncology practice, noting the importance of determining efficacy and safety. FDA approval and guideline recommendations were cited as evidence needed for use and extrapolation of biosimilars.
The second panel was introduced by presentations representing the innovator perspective and biosimilar manufacturer perspectives. FDA’s pending guidance on an approval pathway for biosimilars, what data will be required of biosimilars to prove high similarity to the reference product, extrapolation for off-label use, pharmacovigilance, and substitution practices were among subjects of debate addressed by the panel, moderator, and participating audience members.
Through dialogue and stakeholder engagement, the underlying consensus of the Summit was clear. It is essential that the oncology community work collaboratively to ensure biosimilars are safe and effective for the treatment of patients with cancer.
Based on the NCCN Biosimilars Work Group discussions and subsequent NCCN Biosimilars Policy Summit, NCCN published a white paper entitled "NCCN Biosimilars White Paper: Regulatory,Scientific, and Patient Safety Perspectives" as a JNCCN supplement.
Islah Ahmed, MD, Global Medical Director, Hospira, Inc.
Edward Louis Braud, MD, Oncologist, Springfield Regional Cancer Center
James Cross, MD, Head of National Medical Policy and Operations, Aetna
Nancy Davenport-Ennis, Chief Executive Officer & President, National Patient Advocate Foundation
Barry Dickinson, PhD, Secretary to the Council on Science and Public Health, American Medication Association
Steven Goldberg, MD, VP & Chief of Medical Affairs, Express Scripts
Scott Gottlieb, MD, Fellow, American Enterprise Institute for Public Policy
James M. Hoffman, PharmD, MS, BCPS, Medication Outcomes & Safety Officer, St. Jude Children's Research Hospital
Phil Johnson, MS, RPh, FASHP, Pharmacy Advocacy Director, Moffitt Cancer Center
Gary Lyman, MD, MPH, FRCP, Director, Comparative Effectiveness and Outcomes Research, Duke Comprehensive Cancer Center
Richard Markus, MD, PhD, Executive Medical Director, Amgen Global Development
Ursula Matulonis, MD, Director, Medical Gynecologic Oncology, Dana-Farber Cancer Institute
Denise Reinke, MS, NP, Nurse Practitioner Sarcoma Program, University of Michigan Comprehensive Cancer Center
Andrew Zelenetz, MD, PhD (CHAIRPERSON), Chief, Lymphoma Service, Memorial Sloan-Kettering Cancer Center