Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Aldeyra, Alvotech, Astrazeneca, Checkpoint, Genenta, Intellia, Santhera, Serda, Valneva.
The U.S. development path for rare disease treatments is strewn with numerous challenges, not least of which are the regulatory hurdles. For companies developing promising candidates to treat ultra-rare diseases and the patients who are running out of time, the regulatory obstacles in the U.S. may seem almost insurmountable. And new concerns about drug development in general could make those barriers even higher.
Regulatory snapshots, including global submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Bigfoot Biomedical, Boomerang Medical, Convatec, Cordx, Neuro20.
Test developers who are seeking coverage by public and private payers often resort to clinical practice guidelines as support for their pleas for coverage, but payers aren’t always persuaded by these guidelines. Lon Castle of Evicore Health told test makers that while these guidelines are often helpful, many of them are well ahead of the evidence, and that test developers would do well to check the data behind the guidelines before reciting them to payers.
Boomerang Medical Inc. knocked out its first target with a U.S. FDA breakthrough device designation for its bioelectronic device for treatment of inflammatory bowel disease (IBD). The technology stimulates the parasympathetic nervous system to reduce inflammation for individuals with both types of IBD, Crohn’s disease and ulcerative colitis.
Europe has given device makers more time to meet requirements set out in the 2017 Medical Devices Regulation (MDR). The European Parliament approved in mid-February an extension of the transition periods for medical device and IVD manufacturers to comply with the new rules, which aim to enhance safety for patients and ensure the “smooth functioning of the internal market.”
The question of the U.S. FDA’s authority to regulate lab-developed tests (LDTs) has been percolating for more than a decade, but the recent failure of Congress to pass legislation granting the agency explicit authority to do so is seen in some quarters as a missed opportunity. The FDA’s Elizabeth Hillebrenner said that while the agency would prefer to regulate LDTs under new statutory authorities, the agency sees a public health problem with the current state of affairs, and thus, “we are moving forward with rulemaking.”
Although the U.S. FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) voted unanimously, 12-0, March 1 that the data GSK plc presented was adequate to support the safety of its respiratory syncytial virus vaccine, several panelists cautioned the FDA against viewing the vote as a recommendation to license the vaccine before more data are available.
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