Biologics and Biosimilars: Rheumatologist Perceptions about ...

Biologics and Biosimilars: Rheumatologist Perceptions about Standards and Oversight A Survey of Rheumatologists by the Coalition of State Rheumatology Organizations (CSRO) June 29, 2015

Biologics and Biosimilars: Rheumatologist Perceptions about Standards and Oversight A Survey of Rheumatologists by the Coalition of State Rheumatology Organizations

Biologics and Biosimilars: Rheumatologist Perceptions about their Impact on Patients A Survey of Rheumatologists by the Coalition of State Rheumatology Organizations Survey Overview and Methodology The Coalition of State Rheumatology Organizations (CSRO) conducted a survey to measure the perceptions of rheumatologists about biologics and biosimilar drugs. Biologics are medicines made from living organisms and are far more complex and much more difficult to develop and manufacture than traditional chemical drugs. Biosimilars are medicines that are highly similar, but not identical to current biologics. Because of the uniqueness and complexity of biologics, biosimilars are not generic copies of biologic medicines. While there are a number of biosimilars approved in other countries, the U.S. Food and Drug Administration (FDA) recently approved the first biosimilar in the United States. Currently, there are four other biosimilar applications that FDA is reviewing for approval. CSRO used a web-based tool to distribute a 6-question survey to rheumatologists nationwide about issues that may impact the approval of biosimilars in the United States. The questions addressed issues such as physician notification of biosimilar substitution, interchangeability, naming, and labeling. The survey was distributed through state and regional CSRO member organizations to approximately 3,000 rheumatologists across the country. CSRO received 315 responses, a response rate of over 10 percent.

Issue Background As part of the Affordable Care Act (ACA), Congress enacted the Biologics Price Competition and Innovation Act (BPCIA) establishing authority for FDA to review and approve biosimilars. BPCIA requires that a biosimilar be “highly similar” to an already-approved innovative biologic product and that “there are no clinically meaningful differences” between it and the reference product “in terms of the safety, purity, and potency of the product.” The law also gives FDA authority to deem a biosimilar to be “interchangeable.” Interchangeability requires a significantly higher standard in order to confirm that an interchangeable biosimilar will achieve the same clinical result in any given patient. FDA has developed some processes to review and approve biosimilars, but many key decisions, such as the kinds of studies that must be done and standards for biosimilars, have not yet been made. FDA has not yet issued notice and comment rulemaking on interchangeability and naming, instead releasing draft guidance documents that provide no meaningful opportunity for input from patients and physicians. Clarity from FDA is needed because the complexity of each biologic medicine makes it impossible to produce exact copies and even slight variations in the manufacturing can cause a biosimilar to have a different effect. The first biosimilar was approved in March 2015, and a number of other applications are currently under review. As other biosimilars enter the market in the United States, they will offer more options to patients


suffering from chronic conditions, but many important questions about the oversight process remain unanswered.

Summary of Survey Findings A vast majority of respondents support further regulatory oversight before more biosimilars are approved. The ACA gives FDA authority to find a biosimilar interchangeable and the FDA will determine what standards must be met for a biosimilar to be interchangeable with the original biologic medicine. Over 82 percent of rheumatologists surveyed support “rigorous” FDA approval standards prior to designating a biosimilar as interchangeable, including clinical trial data specific for each condition. The FDA has not provided any guidance on the naming of biosimilars, an important issue for rheumatologists. More than 75 percent of rheumatologists support different product names for biosimilars than the reference product. The vast majority of rheumatologists agreed on the subject of labeling. The survey asked if the FDA should require a biosimilar to have a label that identifies the medication as a biosimilar and convey any important difference between it and the innovator biologic. Nearly 96 percent of respondents answered “yes.”

Conclusions and CSRO Recommendations This survey demonstrates that rheumatologists believe further guidance from the FDA on critical issues are needed before additional biosimilars come to market. Almost all rheumatologists surveyed believe both physicians and patients should be notified before any treatment is modified, and the vast majority of respondents believe there could be actual patient harm from switching medications without physician consent. Rheumatologists want to ensure that the interchangeable standard requires rigorous testing. The FDA should also mandate that all biosimilars have unique naming that is included on product labels. Clear identification systems will help providers and patients track what drug is being used, and will help identify differences in storage or administration instructions. Based on the conclusions drawn from this survey, CSRO offers the following policy recommendations for biosimilar approval: 

There should be a clearly distinguishable nonproprietary name for the biosimilar to ensure there is no confusion with the reference product, ensuring that both physician and patient are appropriately informed about the medication being dispensed;

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The label for a biosimilar must include the nonproprietary name and any differences in safety or effectiveness data that is unique to the biosimilar;

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Biosimilars should undergo clinical testing for each condition for which the reference product is approved.


About Rheumatology Rheumatologists are internists who are board certified and are qualified by additional training and experience in the diagnosis and treatment of arthritis and other diseases of the joints, muscles and bones. Rheumatologists treat arthritis, certain autoimmune disorders, musculoskeletal pain disorders and osteoporosis. There are more than 100 types of rheumatologic diseases, including rheumatoid arthritis, osteoarthritis, gout, lupus, back pain, fibromyalgia, and tendonitis. Many rheumatologic diseases are very serious and complex. They can be difficult to diagnose and treat, and many can change or evolve over time. Specialized care from an expert is often necessary, and only rheumatologists are experts in this field of medicine. Rheumatologists can reduce the severity of disease and save time and money. Prompt diagnosis and specially tailored treatment can improve quality-of life and long-term outcomes.

About Coalition of State Rheumatology Organizations (CSRO) The Coalition of State Rheumatology Organizations (CSRO) is a group of more than 30 state and regional professional rheumatology societies formed in order to serve practicing rheumatologists nationwide. CSRO advocates for excellence in rheumatologic disease care and patient access to the highest quality care for the management of rheumatologic and musculoskeletal diseases. For more information, visit www.CSRO.info
