FDA announces new efforts to strengthen its expanded access program
Food and Drug Administration commissioner Scott Gottlieb today outlined efforts to further strengthen the expanded access program, while noting the progress the agency has been made over the last five years.
FDA has authorized more than 9,000 applications across drugs, biologics and devices through the agency’s expanded access program, and authorized approximately 99% of all the requests it has received across all application types, Gottlieb said.
Gottlieb also highlighted key findings from the FDA’s independent assessment of the program and new steps the agency is taking to improve upon its efforts.
“The assessment found that external stakeholders’ overall perceptions of FDA’s expanded access program — and FDA’s role in administering it are very positive,” Gottlieb said. “Stakeholders across the healthcare system highlighted FDA’s commitment to expediting the review of EA applications, the FDA’s collaborative nature and our focus on continuous improvement.”
Physicians with direct experience submitting an EA application to the FDA reported positive impressions of the program, and patients and their advocates described the program as a crucial route to access investigational therapies when other alternatives have been exhausted.
“Manufacturers, patient advocates, IRB representatives and physicians all noted that FDA has taken key steps to reduce the administrative burden associated with submitting requests and recognized our commitment to facilitate medically appropriate access via the EA program,” Gottlieb said.
Stakeholders also reported some challenges across the physician and patient journey through the program, that, if properly addressed, could meaningfully enhance the program.
The assessment found that confusion with program navigation, multi-stakeholder coordination and administrative burden were the most frequently-cited challenges. The assessment recommendations include improving FDA’s public website content and investing in resources to support patient/physician program navigation.
In response to the feedback, FDA’s EA web pages will be updated to improve usability through streamlining of content and a more user-friendly organization. This includes a reduction in duplication, as well as the addition of new pages with such commonly requested information as forms and keywords.
“These updates will begin rolling out today and will continue in the future as we identify new opportunities to improve the usability of information on our EA webpages,” Gottlieb said.
FDA also plans to formally establish an agency-wide Patient Affairs Staff and Health Care Provider Affairs Program, under the oversight of the Office of Clinical Policy and Programs. “This will enhance our engagement with these important external stakeholder groups. The Patient Affairs Staff is already in place and charged with serving as the ‘home base’ and primary point of entry for patients and physicians starting the EA process and navigating them through the steps,” Gottlieb said.
The FDA already streamlined the required supporting documentation for expanded access requests submitted by a physician for access to a drug or biological for the treatment of an individual patient.
“These changes reduced the administrative burden for these physicians,”Gottlieb said. “Following these changes, we estimate that it takes about 45 minutes, on average, to complete a patient application form. That form typically requires just one attachment.”
Pointing out that the FDA also simplified the process for Institutional Review Board, or IRB review, Gottlieb said, “For single patient EA, we’ve modified the IRB review process to permit just one IRB member — the chair or another appropriate person — to concur with the treatment use rather than the entire board.”
The FDA also established an agency-wide Expanded Access Coordinating Committee, which facilitates cross-center communication and promotes discussion to rapidly address cross-cutting issues related to expanded access to promote consistency and best practices.
Cardinal Health posts 8% growth in Q1
Cardinal Health on Thursday reported its first quarter fiscal year 2019 results, posting revenues of $35.2 billion — an increase of 8% from the same period last year.
For the quarter, GAAP diluted earnings per share increased 439% to $1.94, while non-GAAP diluted EPS increased 18% to $1.29.
“We are pleased that the first quarter provided a solid start to fiscal year 2019,” Cardinal Health CEO Mike Kaufmann said. “Operating performance came in as expected, and we are on track in executing on our strategic initiatives to deliver increased value to our shareholders, our customers and their patients. We look forward to making further strides over the balance of the year and building on Cardinal Health’s essential role in health care.”
First-quarter revenue for theDublin,Ohio-based company’s pharmaceutical segment increased 9% to $31.4 billion, due to sales growth from pharmaceutical distribution and specialty solutions customers, partially offset by the divestiture of the company’s China distribution business, the company said.
Segment profit for the quarter decreased 12% to $409 million, which reflects the negative impact from the company’s generic program performance, according to the company.
First-quarter revenue of the medical segment increased 2% to $3.8 billion, which was driven primarily by new and existing customers, Cardinal Health said.
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