On October 1, 2019, FDA issued its second Patient-Focused Drug Development (PFDD) draft guidance – the next in a series of four methodological guidance documents, which discuss the collection of patient experience data to support decision-making related to drug development and approval (see some of …
The American Conference Institute’s (“ACI’s”) popular “FDA Boot Camp” – now in its 34th iteration – is scheduled to take place from September 18-19, 2019 at The Bostonian Hotel, Boston, MA. The conference is billed as the premier event to provide folks with a roadmap …
Last week, Global Genes, a global rare disease patient advocacy organization, announced the 2019 RARE Champion of Hope Awardees, which included Hyman, Phelps & McNamara, P.C.’s very own, James Valentine. This is a great honor, bestowed upon “true champions for rare disease”, “people who inspire …
On May 24, 2019, FDA approved Avexis’s BLA for its gene therapy, Zolgensma (onasemnogene abeparvovec-xioi), for treatment of spinal muscular atrophy (SMA). This represents the first approval of a systemically-administered somatic gene replacement therapy. (The first gene therapy approved by FDA was Luxturna in Dec. …
On May 8, 2019, the FDA published a new draft guidance for industry titled “Submitting Documents Using Real-World Data and Real-World Evidence to FDA for Drugs and Biologics.” This draft guidance was produced as a joint effort from CDER and CBER and is intended to provide …
On March 25, 2019, FDA issued a draft guidance, “Rare Diseases: Natural History Studies for Drug Development,” to help inform the design and implementation of natural history studies that can be used to support the development of drugs and biological products for rare diseases (hereinafter …
On March 16, 2019, Advances in Therapy published a commentary authored by HP&M Attorney James E. Valentine along with co-authors Marielle Contesse, Tracy Wall, and Mindy Leffler. The paper sets forth the case, and provides a methodologic overview, for incorporating qualitative patient and caregiver video …
About 6 weeks after Vanda filed its complaint, the company’s lawsuit has joined its clinical trial: on hold. Last week, the District Court of D.C. granted FDA’s motion for a voluntary remand of the case challenging FDA’s requirement to perform 9-month large animal studies prior …
The American Conference Institute’s (“ACI’s”) popular “FDA Boot Camp” – now in its 33rd iteration – is scheduled to take place from March 25-27, 2019, at the Park Lane Hotel in New York, NY. The conference is billed as the premier event to provide folks …
As avowed dog-lovers and FDA law enthusiasts, a recent Complaint filed in the District Court of D.C. caught our attention. Vanda Pharmaceuticals filed a Complaint on February 6, 2019 alleging that FDA’s insistence that Vanda perform a 9-month dog study with its drug candidate tradipitant …
With a little over a year under FDA’s belt implementing its Comprehensive Regenerative Medicine Policy Framework (which we blogged about here), CBER has updated its current thinking with the issuance of final versions of the two Regenerative Medicine Advanced Therapy (“RMAT”)-related guidance documents: Expedited Programs for …
As we await our nation’s State of the Union address, FDA’s own Commissioner Scott Gottlieb and Center for Biologics Evaluation and Research (CBER) Director Peter Marks issued a joint statement providing a state of union of sorts on the development and regulation of cell and …
Next Thursday, February 7, the Food and Drug Law Institute will conduct a webinar entitled, “Patient Assistance Programs, Recent Enforcement and Best Practices.” The webinar will cover the various forms of patient assistance programs, the legal authorities that apply to them, recent enforcement actions, best …
Many companies with investigational drugs have posted an expanded access policy on their websites as required, but more than a few still have this task left over from the 2018 to-do list. As the time for New Year’s resolutions and new to-do lists is upon …
On December 13, 2018, the National Academies of Sciences, Engineering, and Medicine released the proceedings of a May 2018 expert workshop entitled “Advancing the Science of Patient Input in Medical Product R&D: Towards a Research Agenda”. The workshop, hosted by the Academies’ Forum on Drug …
