National Organization for Rare Disorders Urges FDA To Consider Rare Disease Treatment Implications When Setting Biologics Naming Policy

WASHINGTON, June 4, 2014 /PRNewswire-USNewswire/ -- In a letter submitted today to the U.S. Food and Drug Administration (FDA), the National Organization for Rare Disorders (NORD) urged Commissioner Margaret Hamburg to give serious consideration to the concerns of the rare disease community when setting policy regarding official names for biologics, including biosimilars.

With over 7,000 rare diseases identified and 30 million Americans affected, the patient population represented by NORD is extraordinarily heterogeneous. Without thoughtful and consistent naming protocols for biologics, there is the potential for significant confusion among treatment options and increased adverse events, both of which could jeopardize patient safety. Distinguishable naming of all biologics is imperative to recognize for health care professional to deliver the degree of customized care that is routinely required for patients with complicated, uncommon and less well-studied diseases.

"Every patient deserves the care best suited for their medical situation and most likely to give them the best outcomes," said NORD President and CEO Peter L. Saltonstall. "Biologics are often the most advanced and effective treatments for patients we represent and everyone in the treatment continuum should be able to readily identify the specific drug product a patient was given."

The letter raises a number of concerns specific to rare disease patients. Rare disease patients usually have unique treatment courses and while similar medications might work equally well, such a judgment can only be made through tracking of outcomes made possible through distinguishable names.

According to the letter, distinguishable names for biologics would:

• Support the medical community's vital post-approval learning curve regarding which medicines are best for their rare disease patients;
• Support surveillance and tracking of adverse events given that rare disease patients often do not respond to medications in the same way as other individuals might; and
• Reinforce a critical distinction in the biosimilars law between biosimilars (similar not identical) and interchangeable biosimilars (similar but demonstrated to have comparable clinical results).

The full text of the letter is below.

About NORD:
The National Organization for Rare Disorders (NORD) was established in 1983. It is a nonprofit organization representing all Americans affected by rare diseases. NORD's services include advocacy, awareness, education, patient services and research to improve the lives of all individuals and families affected by rare diseases. NORD's website is at www.rarediseases.org.

The Honorable Margaret Hamburg
Commissioner
U.S. Food and Drug Administration
10903 New Hampshire Avenue
Silver Spring, MD 20993

Re: Naming of Biologics

Dear Commissioner Hamburg,

The National Organization for Rare Disorders (NORD) urges you to adopt distinguishable names[1] for biologics, including biosimilars. Our position stems from the existing lack of consistent naming protocols for biologics and the potential for significant physician and patient confusion once biosimilars become available.[2] The need for distinguishable names is independent of FDA's latest guidance on levels of evidence of biosimilarity.

With over 7000 rare diseases identified and 30 million Americans affected, the population represented by NORD is extraordinarily heterogeneous. We believe strongly that every patient deserves the medical care that is best suited for their medical situation and that is most likely to give them the best results. Based on the reports we receive from member organizations, as well as individuals, it is increasingly difficult for rare disease patients to receive optimum care if any degree of customization is required.

In light of this problem of access to optimum care, distinguishable names for biologics, including biosimilars, addresses three important concerns to the rare disease community.

Distinguishable names for biologics support the medical community's vital post-approval learning curve about which medicines are best for their rare disease patients. Health care providers need to know that a prescribed medicine was actually given to the patient and whether a substitution was made and to what alternative product. This can't be achieved unless biologic productsespecially ones with similar therapeutic purposescannot be distinguished, tracked and studied.

This is a critical issue for a large segment of the rare disease community: patients who are treated--on or off-label--with medicines that have a primary indication that is not for their specific rare disease. Which drug will work for what patients in what therapeutic regimen is, for these patients, almost entirely based on a post-approval trial and error process conducted by clinicians and researchers. Similar medications might work equally well, but such a judgment can only be made based on tracking of results that relies on distinguishable biologic names.

Distinguishable names for biologics support surveillance and tracking of adverse events. Rare disease patients often do not respond to medications the same way other individuals might. Idiosyncratic reactions to medications occur frequently.

As a group, rare disease patients are more dependent on "medical necessity" determinations than others. Quite often, this is based on changing medications because of adverse events. For an individual, this might be an unpleasant, expensive and perhaps life-threatening event, but the record is then established for that individual that alternative therapy is needed. What rare disease patients need is accurate surveillance and clinical tracking data so that subsequent patients don't have to fail therapy/experience an adverse event before justifying a medical necessity determination to receive a different medication.

Distinguishable names for biologics reinforce a critical distinction in the biosimilars law between biosimilars (similar not identical) and interchangeable biosimilars (similar but demonstrated to have comparable clinical results). NORD welcomes biosimilars because many rare disease patients will benefit from having additional therapies that might be available at a substantial discount to current products. However, there is no assurancelegal, clinical or scientificthat biosimilar products will work the same as the reference product in rare disease patients. Even when interchangeable biosimilars are approved by FDAlikely well into the futureit still will not always be the case that rare disease patients will respond the same to a biosimilar in the same way as they do to the reference product.

Having distinguishable names promotes the needed learning curve (our first point) and assists with tracking of adverse events (our second point). Critically, distinguishable names for biologics serves as a reminder to payers, states and health care facilities that market access has been granted only on the basis of similarity and not on the basis of substitution or interchangeability (our third point). This is very different from the situation with generic drugs, where common names are used and FDA's approval findings include whether interchange or substitutions is supported by evidence.

Commissioner, thank you for your attention to this issue, which is of the utmost importance to rare disease patients. If we can supply additional information, please do not hesitate to let us know.

[1] We consider the specific format to be a technical issue and have no position at this time on how FDA should assure that biological products be distinguishable, e.g. prefixes, suffixes, etc.

[2] Closely related to the naming issue, we urge FDA to require the labeling of biosimilars to specify the reference product so that biosimilars cannot inadvertently be confused within a therapeutic category of similar, but not identical originator products.

SOURCE National Organization for Rare Disorders (NORD)