FDA to review first biosimilar application
Could the regulatory and scientific logjam that’s keeping copycat biotech medicines off the market and out of the hands of patients and health providers in the United States finally be breaking?
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The Food and Drug Administration defines biosimilars as products that mimic their original, already-approved biologically engineered counterpart, “notwithstanding minor differences in clinically inactive components, and for which there are no clinically meaningful differences between the biosimilar and the approved biological product in terms of the safety, purity and potency.”
Thus far, that distinction has been academic within the U.S. pharmaceutical market, which has yet to see its first biosimilar approved for marketing by the FDA despite their common use in many other countries. But some four-and-a-half years after Congress and the White House enacted legislation calling on the agency to establish a review and approval pathway for biosimilars — and five years after passage of the Biologics Price Competition and Innovation Act of 2009 laying the groundwork for that approval process — there are signs that such a pathway is finally beginning to emerge.
In July, Novartis’ Sandoz unit became the first company to file for approval of a biologically engineered me-too pharmaceutical under the bio-similars pathway created by Congress and the Obama administration.
Sandoz announced that the FDA had accepted its biologics license application for filgrastim, a biosimilar modeled after Amgen’s Neupogen for the treatment of infection in patients receiving myelosup-pressive anticancer drugs. “The FDA’s acceptance of Sandoz’s filing is an important first step in increasing U.S. patient access to affordable, high-quality biologics,” the company noted in a statement.
Sandoz already markets filgrastim under the brand name ZARZIO in more than 40 countries outside the United States, according to the company. If approved by the FDA, the drug would be the first biosimilar available in the United States as early as 2015.
Meanwhile, investigational new drug applications for biosimilars continue to trickle into the FDA, with the agency receiving a total of eight INDs as of the end of June.
The Patient Protection and Affordable Care Act envisions what the FDA calls “an abbreviated licensure pathway for biological products that are demonstrated to be ‘biosimilar’ to or ‘interchangeable’ with an FDA-licensed biological product. But clearing that pathway for complex, biologically derived large-molecule compounds has been slow going. The agency, said FDA commissioner Dr. Margaret Hamburg, has labored to develop a review and approval process that sets “high standards for approval.”
“Building a new approval program of this complexity takes much time and deliberation, and that’s certainly been the case,” Hamburg said. “Congress deliberately set a very high bar for a biosimilar product approval. Manufacturers must demonstrate that their product is highly similar to, and differs in no clinically meaningful way, from the previously licensed product, known as the reference product, in terms of safety, purity and potency.”
Despite the slow, careful pace of progress at the FDA, the generic industry group predicts “a bright outlook for biosimilars,” with biologics amassing a total of some $60 billion in combined annual global revenues either already off patent or facing loss of patent protection over the next two years.
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WHO biosimilars naming proposal divides industries
What’s in a name? A lot, apparently, if the name applies to a biosimilar medicine.
Responding to requests from some countries’ regulatory agencies, the World Health Organization is mulling a proposal for a global identification system for biosimilar drugs that it says will help distinguish biosimilar and original biotech products, and avoid potential confusion among prescribing physicians and healthcare agencies. But the proposal is spawning debate among pioneer and generic drug companies worldwide, and opposing reactions from industry trade groups representing both sides of the debate.
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Specifically, the WHO’s new naming plan has raised concerns among companies that produce biosimilar medicines that their products could be elbowed out of the biotech market through neglect if they carry a different name than that of the original bio-engineered drug they’re designed to mimic. Industry groups like the Generic Pharmaceutical Association fear that creating distinct, separate names for biotech products and their biosimilar counterparts would confuse prescribing doctors and pharmacists, thus discouraging substitution of cheaper me-too biologic medicines and giving an unfair market advantage to branded biotech firms.
The issue revolves around the continued use of the same International Non-Proprietary Name for original, brand name biologics and their biosimilar counterparts. The WHO, which oversees that identification system, is proposing a nomenclature system that maintains the current practice by many developed countries of using the same INN to identify both the pioneer and follow-on biologic drug. But the global health group also is proposing the adoption of a secondary, voluntary identification code, dubbed a biological qualifier, to be attached to each drug to complement the INN identification system and “avoid proliferation of separate and distinct schemes developed by individual regulatory authorities.”
“This voluntary scheme is intended to provide a unique identification code (biological qualifier), distinct from the INN, for all biological substances that are assigned INNs,” the WHO noted in a statement. “The code will consist of four letters … assigned at random.”
Reaction was mixed, and generally broke along the divide between the pioneer biotech industry and biosimilars manufacturers and interest groups. The Biotechnology Industry Organization, representing branded biotech makers, endorsed the WHO proposal for the BQ identifier, “under which nonproprietary names of biological products that are similar to each other in structure and function are distinguishable,” noting that “distinguishable nonproprietary names enhance patient safety.”
The generic and biosimilars industries were less than enthusiastic, however. While supportive of the use of the INN for both biotech and biosimilar medications, they view the proposal for a second BQ marker as simply a tool that could be wielded by the biotech industry to head off competition from follow-on biologics.
“A biosimilar that is designed to be chemically and pharmacologically identical to the reference product should not have a different INN,” GPhA asserted. “Unique INNs … would divorce the biosimilar from its shared regulatory history with the reference originator product on which its approval is fundamentally based.”
GPhA concluded that a separate naming system would confuse prescribers, “thwart competition” and “make the collection of post-approval data and its analysis for adverse events … much more difficult.”
Protecting personal healthcare data
The adoption of personal medical devices and healthcare kiosks that capture consumer health data — like blood pressure and glucose — coupled with data points with a patient’s health record at the pharmacy represents a significant opportunity to enhance disease state management programs.
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However, that same functionality also represents an issue concerning patient privacy as it relates to HIPAA, especially given the ongoing rise in breaches of healthcare data. The number of breaches this year is already close to 12 million records, noted Dan Munro, a Forbes contributor who has covered HIPAA breaches extensively, including the breach of 4.5 million records at Community Health Systems last August. “At this rate, we’ll probably get to 14 million or 15 million records this year alone.”
Hackers aren’t interested in the innocuous personal healthcare measurements generated by personal medical devices or healthcare kiosks. But the devices that capture that data provide a possible entry point to patient health records. “Any device, any endpoint becomes a vulnerability,” Munro said. “The issue isn’t the data itself, the issue is the device as a gateway into the network, which is typically secure.”
And upon accessing those records, hackers can use them to hijack a person’s medical profile. “What they’re looking for is two things,” Munro said. “One is the potential to leverage that [data] quickly for fraud, and the second is for illegal drug use. Prescriptions become another mechanism for which the data has supreme value.”
It’s an issue of significant concern to manufacturers of the devices and healthcare kiosks that capture those data points. “In this day and age if you store data all in one place, it’s not a matter of if you’ll get hacked but when,” said Khan Siddiqui, chief technology officer and chief medical officer for Higi, a kiosk manufacturer. “There are best practices [regarding] how you store [the data] and build the infrastructure to make it extremely difficult for anybody to create a breach,” he said. Higi employs monitoring systems that look for any malicious programming or viruses, he added. “So from an infrastructure point of view, we’ve [implemented] a lot of security layers to really understand what is happening to the data to prevent these kinds of breach scenarios.”
PharmaSmart has a comprehensive program linking the clinical data points captured by their healthcare kiosks to a pharmacy’s patient profile, but the patient data submitted to the profile is de-identified data, which keeps the system in compliance with HIPAA regulations. “The only data PharmaSmart has access to is de-identified data,” Ashton Maaraba, COO and general manager of PharmaSmart, told Drug Store News. And it’s a one-way submission of data, Maaraba said; PharmaSmart does not have access to the pharmacy’s patient profile.
Protecting medical devices from hackers is also an issue for the Food and Drug Administration, which in October finalized recommendations to manufacturers for managing cybersecurity risks to better protect patient health and information.
The final guidance, titled “Content of Premarket Submissions for Management of Cybersecurity in Medical Devices,” recommends that manufacturers consider cybersecurity risks as part of the design and development of a medical device, and submit documentation to the FDA about the risks identified and controls in place to mitigate those risks. The guidance also recommends that manufacturers submit their plans for providing patches and updates to operating systems and medical software.
“There is no such thing as a threat-proof medical device,” said Suzanne Schwartz, director of emergency preparedness/operations and medical countermeasures at the FDA’s Center for Devices and Radiological Health. “It is important for medical device manufacturers to remain vigilant about cybersecurity and to appropriately protect patients from those risks.”
The FDA’s concerns about cybersecurity vulnerabilities include malware infections on network-connected medical devices or computers, smartphones and tablets used to access patient data; unsecured or uncontrolled distribution of passwords; failure to provide timely security software updates and patches to medical devices and networks; and security vulnerabilities in off-the-shelf software designed to prevent unauthorized access to the device or network.
The agency is planning a public workshop this fall to discuss how government, medical device developers, hospitals, cybersecurity professionals and other stakeholders can collaborate to improve the cybersecurity of medical devices and protect the public health.
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