Download Lupus and Allied Diseases Association

RE: BLA 125544 for CT-P13, a proposed biosimilar to REMICADE (infliximab)
February 9, 2016
Kathleen A. Arntsen, Patient Advocate
President and CEO of Lupus and Allied Diseases Association (LADA)
Patients for Biologics Safety and Access (PBSA)
Alliance for Safe Biologic Medicines (ASBM)
Based on my experiences as both an autoimmune patient and advocate, I offer the
following comments and thank you for the opportunity to provide my unique perspective
regarding this BLA. I also submitted more comprehensive written comments which
should be in your materials.
I am just a patient who knows firsthand that we desperately need new drugs to treat
complicated autoimmune diseases like lupus and have closely followed the Biosimilar
pathway. These drugs hold tremendous promise and therapeutic advantages for people
suffering from conditions of unmet need like me, just as biologic medicines have for
millions of individuals living with life-threatening and life-diminishing autoimmune
diseases. Lupus is an extremely complex, chronic inflammatory autoimmune disease
affecting virtually any organ system of the body with few current available treatment
options.
Like many others with lupus, I suffer from several other autoimmune disorders and comorbid conditions; I currently take 38 medications per day and have unique allergies and
sensitivities to both active and inactive ingredients in drugs, requiring careful monitoring
by my healthcare providers. My entire digestive tract is impaired and it takes five
different drugs to allow me to digest food each day. I haven’t eaten fruits or vegetables
in nineteen years and have refused a colostomy. I have an infusa-port for bi-weekly 7hour infusions for renal complications and am blind in my right eye from the shingles
virus and adverse reactions to eye drops. I do not qualify for a corneal transplant so have
an expensive custom made prosthetic device to protect my cornea.
Given that the FDA has not yet finalized guidance on issues that impact patient safety
such as indication extrapolation, interchangeability, naming, and labeling; as you review
this application, please keep in mind complex autoimmune patients like me who do not
fit the norm and are labeled “outliers” by their treating physicians.
You must remain vigilant in protecting patient safety while promoting unfettered access
to vital and innovative treatments by recognizing the complexity of biologics;
snowballing with each generation, as well as the intricacy and vulnerability of the
potential patient populations. At this initial juncture of biosimilar development, it is
critical for both patients and physicians to be confident that these drugs are safe and as
effective as the original innovator biological medication. Therefore, it is essential to
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validate that the chemical, structural and biological parameters are highly similar to the
reference product and consider whether dissimilarities have meaningful clinical
relevance. It is your responsibility to review the science and analytical data and determine
the acceptable amount of uncertainty.
Please understand no one size fits all products exist for complex autoimmune patients
like me, our immune response to treatments is unique, contrary and at times adverse.
Biosimilars are not precise replicas of the originator biologic, subsequently their
performance may not be equivalent in every disease population, resulting in unexpected
divergent effects. Because of this I strongly believe that each biosimilar should be
considered individually for each disease population, not combined together as a group of
variable diseases. Patients like me are so hypersensitive that even the slightest change in
manufacturing, dose or method of delivery, subcutaneous, injectable vs. infusible, can
provoke immunogenicity or heightened immune response and disease complications.
There must be sufficient proof of clinical efficacy, safety, purity, potency and tolerability
provided for each distinct disease population to grant indication extrapolation; not just
projected clinical safety and efficacy data.
To illustrate this point, I refer to the 2014 Mercy University Hospital Study in Cork,
Ireland in which 29% of patients on inflectra required surgery compared to 0% on
infliximab; 80% on inflectra required hospital readmission vs. 5% on infliximab; 60% on
inflectra required increased steroids vs. 8% on infliximab; and 93% on inflectra had an
increase in C-Reactive protein vs. 100% on infliximab having a decrease in CRP.
Therefore, I echo ECCO’s position statement, no pun intended, that use of most
biosimilars in IBD will require testing in this particular patient population and cannot be
extrapolated from other disease populations.
As the FDA determines final guidance on interchangeability, I ask for a policy requiring
rigorous criteria that include non-clinical and clinical data. In order to be designated as
“interchangeable” biosimilars must unequivocally produce the same clinical result in any
given patient as the biologic reference product. Additionally, if given more than once to a
patient, the risk in terms of safety and effectiveness of switching between the
interchangeable and the reference product must not exceed that of using the reference
product without switching. Therefore, it is essential that the strongest patient safety
standard possible be applied when determining guidance for interchangeability.
Pharmacovigilance is essential for all biological medicines because these treatments may
produce idiosyncratic reactions or immunogenic reactions in patients like me who may
also be hypersensitive to changes in production methods or impurities. It is critical for
you to recognize that adverse effects are difficult to predict and may only occur after
many years of treatment and in reality biological medicines will be administered to
patients like me suffering from serious, life-threatening diseases; taking a multitude of
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concomitant medications, including immunomodulatory drugs who are not participating
in a controlled clinical study.
Due to the heterogeneous nature of autoimmune diseases like lupus, no two cases are
alike and treatment is highly individualized; effectively treating patients like me is like
balancing on a pinhead. Only healthcare professionals familiar with a patient’s personal
medical history should be making treatment decisions. Numerous times therapies have
failed me over the years, forcing me and my physicians to take a step backwards and
think outside of the box to treat my complicated medical picture. Individuals with
complex care needs like me require unique strategies and personalized medicine to
manage their care.
I am alive today because I have educated myself and am an integral part of my treatment
team and the catalyst of my health care. However, it also takes a tremendous amount of
self-motivation to manage my medical care, maintain my dignity and attempt to have any
quality of life.
I am living proof that the determination of the most appropriate medical treatment is best
accomplished by open and transparent communication between healthcare professionals
and patient. Therefore, I believe that it is imperative that healthcare providers and
patients have all necessary information to make completely informed decisions regarding
the choice to use an originator biologic or biosimilar. Moreover, biosimilar labeling
information should clearly identify whether it is a biosimilar and state that its therapeutic
determination is based on extrapolation of the reference product’s data. This crucial
information must be in a format that is appropriate for each audience, thus promoting
transparency and facilitating educated and informed decision-making by all parties.
I also feel that automatic substitution of biosimilars for biologics is potentially harmful
and can disrupt continuity of care; resulting in detrimental consequences such as
increased symptomology, medical complications, and higher health care costs.
Substitution should only occur when the FDA has designated a biologic product as
interchangeable and proper patient protections are upheld including communication
between pharmacists and prescribers to guarantee complete transparency. I also believe
that the prescriber should have the authority to prevent substitution by specifying so
when it is in the best interest of the patient not to have a biosimilar.
I acknowledge that newer, innovative treatments offer therapeutic advantages over
conventional medicines and that we are in dire need of more efficacious, accessible, and
affordable treatments. It took 56 years for a lupus drug to be approved, yet still today
most of our treatments are off-label, borrowed from other diseases. We are unequivocally
frustrated with the pace of drug development for conditions of unmet need. However, as
millions in the lupus, autoimmune and unmet disease communities fervently await the
development of new therapies, we also recognize that much like these complex
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conditions, the biosimilar approval process is intricate and warrants a thoughtful,
innocuous and vigilant course.
In conclusion, I thank you again for the opportunity to share my perspective as you
evaluate this BLA and applaud the FDA for continually recognizing the importance of
the patient voice during the drug review process.
Respectfully Submitted,
Kathleen A. Arntsen, President/CEO
Lupus and Allied Diseases Association, Inc.
P.O. Box 170
Verona, N.Y. 13478
[email protected]
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