PBSA calls for strong and clear review standards to protect patient safety

(June 09, 2015) Patients for Biologics Safety & Access (PBSA), a coalition of over 20 patient advocacy organizations dedicated to protecting patient access to safe and effective biologics, today sent a letter to the United States Food and Drug Administration (FDA) questioning the final guidance documents issued for biosimilars and concerns that the final guidances diminish patient safety protections. 

“As representatives of millions of American patients and their families, we applaud the FDA for issuing final guidances on important aspects of the Biologics Pricing Competition and Innovation Act (BPCIA). We remain concerned that instead of strengthening patient safeguards the final guidance documents diminish transparency and patient safety protections from the original draft guidances. The final guidances removed language that called for a biosimilar to be labeled as a biosimilar and to indicate whether or not the biosimilar is interchangeable with the reference product. In addition, the final guidance provides greater opportunity for the use of indication extrapolation for biosimilars,” said Larry LaMotte, Vice President of the Immune Deficiency Foundation, a member of PBSA.

“We are eager for new and affordable treatments, but patients with rare and chronic diseases are keenly aware of the possible risks associated with biosimilars. Biosimilars will not be successful without patient confidence in the approval process of these complex drugs. Taking steps to guarantee that there are strong and clear review standards to protect patient safety will bolster patient confidence in this new category of drugs,” added LaMotte.

Full text of the letter is below.

About Patients for Biologics Safety & Access

Patients for Biologics Safety & Access is a national coalition representing more than 20 patient advocacy organizations. The goal of the organization is to make sure the voices and interests of patients are heard as the FDA seeks to approve a new category of drugs known as biosimilars. For more information, visit www.BiosimSafety.org or @BiosimSafety

American Autoimmune Related Diseases Association

Committee of Ten Thousand

Crohn’s & Colitis Foundation of America

Dystonia Medical Research Foundation

GBS/CIDP Foundation International

Hemophilia Federation of America

Hepatitis Foundation International

Immune Deficiency Foundation

Jeffrey Modell Foundation

Lupus and Allied Diseases Association

Lupus Foundation of America

National Alliance on Mental Illness

National Organization for Rare Disorders

National Psoriasis Foundation

Platelet Disorder Support Association

Pulmonary Hypertension Association

Scleroderma Foundation

Spondylitis Association of America

United Spinal Association

US Hereditary Angioedema Association

***

June 09, 2015

Stephen Ostroff, M.D.

Acting Commissioner,

U.S. Food and Drug Administration

10903 New Hampshire Avenue

Silver Spring, MD 20993

Dear Dr. Ostroff:

Patients for Biologics Safety and Access (PBSA), a national coalition representing more than 20 patient advocacy organizations working to ensure that the voices and interests of patients are heard as the FDA implements the Biologics Price Competition and Innovation Act (BPCIA), thanks you for the release on April 28, 2015, of three final guidances on important aspects of implementing the (BPCIA). We have long urged the agency to move aggressively to issue guidances on a range of important patient safety issues related to biosimilars.  At the same time we want make you aware of some of our concerns and questions about several important aspects of these guidances.

We have compared the three final guidances to the draft versions of these guidances released in 2012. Our initial review of these documents leaves us concerned that instead of strengthening safeguards based on input from patients and other groups, several provisions of the final guidances appear to diminish transparency and patient safety protections from your previously issued draft guidances.

First, the 2012 draft guidance on Scientific Considerations of Biosimilarity called for labeling of biosimilars to identify the product as a biosimilar and to indicate whether or not the biosimilar is interchangeable with the reference product. We saw this as a very important element for patients to be fully informed about their care.  We are very concerned that the final guidance removes this language. This change violates a basic principle of transparency and denies patients important information about their health care.   

Second, patient safety protections appear weakened by language in the final guidances that provide greater opportunity for use of indication extrapolation for approval for biosimilar use in conditions for which the biosimilar has not been tested in clinical studies:

The final Scientific Considerations guidance (on page 20) no longer contains the language of the draft guidance that biosimilars “should be cautious with respect to the extrapolation of safety risk profiles across indications because patient populations for different indications may have different co-morbidities and receive different concomitant medications.” This is concerning because so many of our members face multiple comorbid conditions and must take many types of medications on an ongoing basis.  Focus on these patients should be heightened, not diminished.

The final Scientific Considerations guidance (on page 21) adds language stating that differences between conditions of use with respect to key factors such as immunogenicity of the product in different patient populations and differences in expected toxicities in each condition of use and patient population “do not necessarily preclude extrapolation.” Adding that, “A scientific justification should address these differences in the context of the totality of the evidence supporting a demonstration of biosimilarity.”

We are concerned about the potential impact of these provisions. And we have questions about how and why they were made. For example, we’d like to know what comments FDA received called for these changes.

We would also like to know the substantive justification for these changes.

While we are eager for new and affordable treatments, people with disabilities and patients with rare and chronic diseases are keenly aware of the possible risks associated with biosimilars. Biosimilars will not be successful without patient confidence in the approval process of these complex drugs. Taking steps to guarantee that there are strong and clear review standards and processes in place to protect patient safety and ensure efficacy of biosimilar medicines prior to making decisions about these applications is essential to building patient confidence.

Thank you again for issuing the guidances and for your attention to our concerns. We are eager to work with you to take meaningful steps to address our concerns.   

Sincerely,

Patients for Biologics Safety & Access

American Autoimmune Related Diseases Association

Committee of Ten Thousand

Crohn’s & Colitis Foundation of America

Dystonia Medical Research Foundation

GBS/CIDP Foundation International

Hemophilia Federation of America

Hepatitis Foundation International

Immune Deficiency Foundation

Jeffrey Modell Foundation

Lupus and Allied Diseases Association

Lupus Foundation of America

National Alliance on Mental Illness

National Organization for Rare Disorders

National Psoriasis Foundation

Platelet Disorder Support Association

Pulmonary Hypertension Association

Scleroderma Foundation

Spondylitis Association of America

United Spinal Association

US Hereditary Angioedema Association

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