FDA issued a draft guidance, Demonstrating Substantial Evidence of Effectiveness for Human Drugs and Biological Products (Draft Guidance), on December 19, 2019, as an expansion of its 1998 guidance, Providing Clinical Evidence of Effectiveness for Human Drug and Biological Products (1998 Guidance). The 1998 Guidance provided examples of evidence that FDA could consider to be confirmatory evidence to potentially support FDA approval of a marketing application based on one adequate and well-controlled clinical trial. The new Draft Guidance provides further detail on clinical trial design considerations, as well as forms of confirmatory evidence that sponsors may consider when proposing to rely on a single adequate and well-controlled clinical trial.
As part of the US Food and Drug Administration’s (FDA’s) overall reorganization of the Office of New Drugs, the former Office of Hematology and Oncology Products (OHOP), the FDA office responsible for approving cancer therapies, was recently restructured and renamed the Office of Oncologic Diseases (OOD).
Per Dr. Richard Pazdur, the acting OOD director, the reorganization will allow for greater stakeholder engagement and streamline the drug review process. OOD is now composed of six divisions, including three divisions of oncology.
US President Donald Trump signed a pair of appropriations bills into law on December 20, including bipartisan legislation intended to facilitate the development of generic and biosimilar products. The bill, previously known as the CREATES Act (H.R. 965/S. 340), allows developers of 505(b)(2) New Drug Application (NDA) and Abbreviated New Drug Application (ANDA) products, as well as biosimilar products, to sue companies holding NDAs or Biological License Applications (BLAs) (each, a License Holder) that refuse to provide “sufficient quantities” of an approved reference drug or biologic on “commercially reasonable, market-based terms.” “Sufficient quantities” are those the developer determines it needs to conduct testing and other regulatory requirements to support an application. “Commercially reasonable, market-based terms” are defined as (1) the nondiscriminatory price at or below the most recent wholesale acquisition cost (WAC) for the product, (2) a delivery schedule that meets the statutorily defined timetable, and (3) no additional conditions on the sale.
FDA on September 23 issued a Drug Supply Chain Security Act (DSCSA)-related compliance policy stating it will not take enforcement action against wholesalers that do not have systems in place to verify product identifiers of saleable returned product prior to further distribution until November 27, 2020. FDA’s decision was necessary because existing drug distribution systems are not prepared to handle and verify the large volumes of returned product in the supply chain in the United States. The extended compliance period also allows wholesalers to issue transaction statements for returned product without certification statements concerning verification processes.
Now is the time for pharmaceutical manufacturers to review their Open Payments/Sunshine Act internal compliance procedures, data collection forms and databases, and reporting and recordkeeping templates for payments and transfers of value made to healthcare providers. On August 14, 2019, the Centers for Medicare and Medicaid Services (CMS) published a Proposed Rule seeking to expand the Open Payments program, including the number and types of covered recipients and payment categories. As a result, drug manufacturers have at least two reasons to quickly conduct a comprehensive internal examination of their Open Payments program.