Michbio is calling on the FDA to revise its emergency use authorization (EUA) labeling for COVID-19 convalescent plasma (CCP), saying that “hundreds, if not thousands, of in-date, ready to transfuse CCP units across the country have been rendered unusable by the specifics of the EUA,” which requires the antibody titer to be displayed on each unit. The labeling requirement differs from what was in use for CCP distributed for the expanded access program (EAP) led by the Mayo Clinic. “To make matters worse, the EAP was closed upon the EUA issuance, and thus no existing units can be utilized under the auspices of that protocol,” MichBio President Stephen Rapundalo said. The FDA based its grant of the EUA, in large part, on data from the Mayo EAP, which had enrolled more than 90,000 patients. “Time to transfusion data is the most important factor that lead to the EUA,” Rapundalo said. “This labeling issue, if not rapidly corrected, will lead to significant delays in transfusion of patients across the country for the foreseeable future or put transfusion services licenses at risk for willfully violating FDA requirements.”
The Institute for Clinical and Economic Review (ICER) released a draft evidence report assessing the comparative clinical effectiveness and value of Roctavian (valoctocogene roxaparvovec, Biomarin Pharmaceutical Inc.) and Hemlibra (emicizumab, Genentech) in treating hemophilia A. The report comes at the halfway point of ICER’s eight-month assessment of the drugs. ICER noted that it will continue with its review of Roctavian even though the FDA recently handed Biomarin a complete response letter for the drug. The draft report compares both drugs with Factor VIII prophylaxis, finding Hemlibra incremental or better than Factor VIII and the evidence for Roctavian as “promising but inconclusive.” Comments on the draft evidence report should be submitted to ICER by Sept. 23.
The NIH’s National Cancer Institute (NCI) is partnering with Cancer Research UK to fund Cancer Grand Challenges, an international initiative to address unanswered questions in cancer research. The NCI and Cancer Research UK expect to co-fund about four awards for each round of the challenges, with each selected multidisciplinary team being awarded about $25 million over five years. The partnership is expected to support three rounds of awards, with a new round of challenges announced every other year, according to the NCI. A series of international workshops will be held to get input from cancer research thought leaders and people affected by cancer. The final challenges will be selected from among the most compelling ideas generated from the workshops. A patient committee will provide input and ideas throughout the process. The NCI and Cancer Research UK plan to announce the list of new challenges in October, giving research teams through April 2021 to express interest in pursuing the challenges.
With the FDA prioritizing its response to COVID-19, the number of events triggering biopharmaceutical recalls in the U.S. dropped to 67 in the second quarter of 2020, a 28% decline from the first quarter, according to a report by Stericycle Expert Solutions Inc., of Indianapolis. An even bigger drop was seen in the number of units impacted. The 67 events resulted in the recall of 17.5 million drug units, down nearly 66% from the first quarter. Failed drug specifications accounted for 24% of the events, making that once again the top cause for drug recalls. (Failed specifications have been the leading cause of drug recalls 14 times in the past 18 quarters.) But when it came to the number of units recalled, the presence of foreign materials was the leading factor, due primarily to a single recall of 8.8 million units due to foreign material.
The U.S. SEC adopted two final rules Aug. 26 expanding its “accredited investor” definition and modernizing, for the first time in more than 30 years, its description of business, legal proceedings and risk factor disclosures registrants are required to make under Regulation S-K. The amendments to the investor rule are intended to “improve the definition to identify more effectively investors that have sufficient knowledge and expertise to participate in investment opportunities that do not have the rigorous disclosure and procedural requirements and related investor protections,” the SEC said. For instance, some people may now qualify as accredited investors based on certain professional certifications or credentials. The Regulation S-K changes are intended to improve the readability of disclosure documents while discouraging repetition and the disclosure of information that’s not material, the SEC said.