With the FDA already reviewing its first biosimilar applications, several patient advocacy groups are banding together to ensure the agency listens to the patient voice as it makes approval decisions and develops future biosimilar guidance.

The top priority for the 22 organizations that launched the Patients for Biologics Safety & Access (PBSA) coalition is to make sure adequate patient safety protections are built into the FDA's biosimilar regulatory path. "Biosimilars hold promise for patients with chronic and life-threatening diseases, but that promise should not compromise the safety of patients," said Marcia Boyle, president and founder of the Immune Deficiency Foundation, a PBSA member.

While the coalition isn't questioning the overall safety of biosimilars, it is calling for safeguards to help with pharmacovigilance and to inform patients and doctors as to which drug has been dispensed. Echoing biosimilar safety concerns raised in the past, the PBSA is pushing for regulatory steps to ensure proper tracking of adverse events for all biologics and unique nonproprietary names to eliminate confusion. (See BioWorld Today, May 14, 2012.)

In addition, the coalition wants patients and doctors to have the information necessary to make informed decisions about the biologics they take and prescribe. It also supports incentives to encourage the ongoing development of innovative biologics.

When issues such as biosimilar naming and substitution have come up at public forums, the discussion often turned to a debate about competition. For instance, when several drugmakers developing biosimilars explained the pharmacovigilance need for unique names and physician notification of biosimilar substitution at an FTC hearing earlier this year, some generic drugmakers accused them of trying to erect market barriers to protect the interests of the innovators. (See BioWorld Today, Feb. 5, 2014, and Feb. 6, 2014.)

While the issues aren't new, what is new about the PBSA's effort is its focus on patients. "As the FDA creates a regulatory pathway to market for biosimilars, we want to make sure the voices and interests of patients are front and center," Boyle said.

WAITING FOR GUIDANCE

If the FDA sticks to its work plan, patients may get an opportunity over the next few months to comment on much-awaited biosimilar guidance. The agency has said it plans to issue draft guidances this year on biosimilar labeling and interchangeability, plus a Q&A on implementation of the Biologic Price Competition and Innovation Act. It also hoped to finalize four draft guidances issued in 2012 and 2013.

In August, a few senators wrote to the FDA, questioning the agency's pace in developing biosimilar guidance on "key scientific policy questions" such as naming, labeling, interchangeability and indication extrapolation. Most of these questions should be answered before the first biosimilar is approved, according to the letter, which was signed by Republican members of the Senate Health, Education, Labor and Pensions Committee. The FDA is expected to approve the first U.S. biosimilar in the first half of next year. (See BioWorld Today, Aug. 5, 2014.)

"We have heard there is some difference of opinion on these matters, making it even more important that these policies, which are integral to the success of the biosimilar pathway, be released in draft form as soon as possible," the letter said.

Regardless of when the guidances are released, the ongoing safety concerns reveal the need for biosimilar makers to engage with patient groups like PBSA, which has a membership list that includes organizations such as the Arthritis Foundation, Hepatitis Foundation International, Lupus Foundation of America and the National Organization for Rare Disorders.

ENGAGING PATIENTS

"We have found that early engagement on biosimilars education with doctors, advocacy groups and patients is paramount to success of this new U.S. biosimilars industry," Juliana Reed, vice president of global government affairs at Hospira Inc., told BioWorld Today.

Hospira's education efforts are based on its experience of making and marketing biosimilars in Europe and Australia, where "biosimilars have been transformative to decreasing costs for healthcare systems and driving patient access to important biologics," Reed said. She pointed out that millions of doses of biosimilars have been administered safely to patients in Europe over the past eight years.

Meanwhile, as part of its commitments under the FDA Safety and Innovation Act, the FDA is seeking public comment on how it can best get the views of patients during the development process of all medical products, including biosimilars, and better consider patients' perspectives in regulatory discussions.

The FDA already has formed a working group to align strategies across the agency for patient participation and to explore ways to increase that participation. It also is using a variety of tools to involve the patient community, which includes family and caregivers, in benefit-risk assessments of drugs and devices, the FDA said in a notice to be published in Tuesday's Federal Register. Comments are due by Dec. 4.