Working with Regulators a Focus on the FDA

Working with Regulators A Focus on the FDA Based on the educational program of the Cancer Policy Institute at the Cancer Support Community In partnership with Uniting a Community (UaC) Policy, Advocacy, Education and Action Network September 15, 2015 A Toolkit for Advocates 1 TABLE OF CONTENTS PAGE I. Introduction. .3 II. The FDA 101. .4 III. History and Timeline. .13 IV. Innovative Approaches. 15 V. The Intersection of the FDA and Congress. .18 VI. The FDA and Elevating the Patient Voice. .19 VII. Case Study I: Duchenne Muscular Dystrophy. 26 VIII. Case Study II: Breakthrough Therapy Designation . 28 IX. Key Messages. .29 X. Glossary of terms. 30 XI. Partners and Sponsors. 32 ABOUT THE CANCER SUPPORT COMMUNITY The Cancer Support Community (CSC) is an international nonprofit dedicated to providing support, education and hope to people affected by cancer. CSC offers a menu of personalized services and education for all people affected by cancer. Its global network brings the highest quality cancer support to the millions of people touched by cancer. These support services are available through a network of professionally-led, community-based centers, hospitals and community oncology practices as well as online at www.cancersupportcommunity.org and over the phone at 1.888.793.9355, so that no one faces cancer alone. 2 WORKING WITH REGULATORS: A FOCUS ON THE FDA A TOOLKIT FOR ADVOCATES I. Introduction “The leaders of the FDA want to learn from patients and the advocacy community. They want to have the conversation with you.” –Linda House, MSM, BSN, RN, President, Cancer Support Community Working with Regulators: A Focus on the FDA is the second in a series of meetings hosted by the Cancer Policy Institute at the Cancer Support Community in partnership with the members of Uniting a Community. The goal of these seminars is to provide patient advocates with usable information about how to access, work with and have an impact on the regulatory process. The focus is on providing basic information on the U.S. Food and Drug Administration’s (FDA’s) organization, operations and interactions with Congress and the health care community. The regulatory world is increasingly complex and interactions should be driven by evidence. There is still a powerful role for patient testimonials, but today the individual voice must be elevated to a collective voice that addresses the key factors that determine how decisions are made and policy is implemented. It is critical to have a working knowledge of the agencies making these decisions as well as the skills and resources to interact successfully with the regulators and the process. This is particularly important when dealing with the FDA, a huge and very influential agency charged with assuring that drugs and medical devices are safe and effective before they are made available and marketed to the American public. The FDA is currently engaged in or involved with a number of programs and initiatives designed to improve that process and to accelerate the rate at which promising new therapies become accessible to patients who need them. The agency is also on the cusp of several key congressional decisions related to clinical research and the upcoming reauthorization of user fees. THIS TOOLKIT PROVIDES BASIC INFORMATION ABOUT: • The roles and responsibilities of the FDA. • The FDA’s organization and how to access decision makers. • Key initiatives that are changing how patients access new therapies. • The FDA and Congress. • Advocacy. • Resources for working with the FDA. 3 WORKING WITH REGULATORS: A FOCUS ON THE FDA A TOOLKIT FOR ADVOCATES II. The FDA 101 “The FDA wants what you want—safe, effective therapies. They are not there to say no. Slamming the FDA is not helpful. It is much more useful to work with them.” –Joshua Sharfstein, MD, Associate Dean for Public Health Practice and Training, Johns Hopkins Bloomberg School of Public Health THE FDA “A place where law, science and other things—public health, politics, THE FOOD AND DRUG beliefs—intersect.” ADMINISTRATION –Susan Wood, PhD, Associate Professor and Executive 10903 New Hampshire Avenue Director, Jacobs Institute of Women’s Health, George Silver Spring, MD. 20993 Washington University 888-463-6332 The FDA is the government agency “responsible for protecting www.fda.gov the public health by assuring the safety, efficacy and security of human and veterinary drugs, biological products, medical devices, our nation’s food supply, cosmetics, and products that emit radiation. The FDA is also responsible for advancing the public health by helping to speed innovations that make medicines more effective, safer, and more affordable and by helping the public get the accurate, science-based information they need to use medicines and foods to maintain and improve their health. FDA also has responsibility for regulating the manufacturing, marketing and distribution of tobacco products to protect the public health and to reduce tobacco use by minors.” The Food and Drug Administration. (2015, December 7). About the FDA. Retrieved from: http://www.fda.gov/AboutFDA/WhatWeDo/default.htm The FDA is part of the executive branch of the U.S. government. It is one of the agencies of the Department of Health and Human Services, and a part of the Public Health Service. “The FDA has a different mission than the other agencies in the health cadre of government. We are the regulators. The rest are service providers.” –Richard Klein, Director, Patient Liaison Program, Office of Health and Constituent Affairs, FDA WHAT THE FDA DOES: • Reviews and approves new drugs. • Inspects manufacturing facilities. • Conducts field inspections. • Monitors post-market adverse events. • Maintains oversight of drug advertising. • Recalls unsafe or ineffective products. • Manages drug shortages. • Provides information to the public. • Conducts research to promote the science of drug and device evaluation. 4 WORKING WITH REGULATORS: A FOCUS ON THE FDA A TOOLKIT FOR ADVOCATES FDA PUBLIC HEALTH ACTIVITIES: Review activities: WHAT DOES THE FDA REGULATE? • Human drugs. The scope of the FDA’s regulatory authority is very broad. The FDA’s responsibilities are closely related to those of several • Human biologics. other government agencies. Often frustrating and confusing • Over-the-counter (OTC) drugs. for consumers is determining the appropriate regulatory agency to contact. The following is a list of traditionally • Vaccines. recognized product categories that fall under the FDA’s • Blood products. regulatory jurisdiction; however, this is not an exhaustive list. • Cell and gene therapy. In general, the FDA regulates: Foods, including: • Tissue therapy. • Dietary supplements. • Medical devices. • Bottled water. • Food additives. • Radiation-emitting equipment. • Infant formulas. • Other food products (although the U.S. Department of Agriculture • Veterinary drugs. plays a lead role in regulating aspects of some meat, poultry and • Cosmetic regulation. egg products). • Food safety. Drugs, including: • Prescription drugs (both brand-name and generic). Enforcement activities: • Non-prescription (over-the-counter) drugs. • Field inspections. Biologics, including: • Vaccines. • Drug advertising. • Blood and blood products. • Cellular and gene therapy products. • Recalls of defective products. • Tissue and tissue products. • Seizures/prosecutions. • Allergenics. Medical Devices, including: Public health activities: • Simple items like tongue depressors and bedpans. • Laboratory research. • Complex technologies such as heart pacemakers. • Dental devices. • International coordination. • Surgical implants and prosthetics. • In vitro diagnostic kits. • Drug shortage management. Electronic Products that give off radiation, including: • Information/guidance for industry. • Microwave ovens. • Information for health care professionals. • X-ray equipment. • Laser products. • Information for public. • Ultrasonic therapy equipment. • Mercury vapor lamps. Regulated products are: • Sunlamps. • Safe and efficacious. Cosmetics, including: • Color additives found in makeup and other personal care products. • Honestly, accurately and informatively • Skin moisturizers and cleansers. represented. • Nail polish and perfume. • In compliance with laws and regulations. Veterinary Products, including: • Livestock feeds. • Pet foods. • Veterinary drugs and devices. Tobacco Products, including: • Cigarettes. • E-cigarettes. • Cigarette tobacco. • Roll-your-own tobacco. • Smokeless tobacco. 5 WORKING WITH REGULATORS: A FOCUS ON THE FDA A TOOLKIT FOR ADVOCATES The FDA regulates over $1 trillion worth of products, which account for 20 cents of every dollar spent by American consumers. WHAT THE FDA DOES NOT DO “Don’t expect the FDA to do things it doesn’t have the legal authority to do, and remember, the FDA is required to make its decisions based on science. It does this through both internal and external evaluation.” –Susan Wood, George Washington University • Develop drugs and devices. • Design or run clinical trials. • Regulate the practice of medicine. • Regulate the cost and price of medicines or devices. “Changes in policy are often not just up to the FDA. It is more complex. The Department of Health and Human Services (HHS), the White House, Congress can all have a role, but the FDA can be your best advocate.” –Joshua Sharfstein, Johns Hopkins Bloomberg School of Public Health 6 WORKING WITH REGULATORS: A FOCUS ON THE FDA A TOOLKIT FOR ADVOCATES THE ORGANIZATION OF THE FDA FOOD AND DRUG ADMINISTRATION OFFICE OF THE

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