Gottlieb Outlines the FDA’s Future Approach to Precision Medicine in Davos

The FDA plans to base future approvals of precision medicines largely on clinical trials that examine long-term durability and safety issues, Commissioner Scott Gottlieb said during a panel discussion at the World Economic Forum in Davos, Switzerland. Gottlieb said the key regulatory issues will be less about determining efficacy, especially with products demonstrating a strong proof-of-principle and early observations of effectiveness in small trials — and instead more focused on product issues related to potential off-target effects and their implications. The agency will also seek accelerated approvals for precision medicines designed to optimize benefits for particular groups of patients, especially through genomic or molecular profiling. “I think we’re really at an inflection point right now, where we’re … defining the modern rules for how these technologies are going to be regulated,” he said. “We’re going to be looking at accelerated approval endpoints for earlier approval on questions of efficacy, with more vigorous long-term follow up in some of these constructs where we have authority to do that under accelerated approval.”

Federal Lawsuit Claims FDA Inspection Negligence Hurt Research Firm’s Reputation

A drug research firm is suing the FDA for $50 million in federal court, arguing an agency disciplinary letter led to the “annihilation” of the company’s reputation and finances, including the scuttling of the company’s potential sale, based on evidence fabricated by a rogue employee. In September 2015, FDA staff inspected Semler Research Center’s drug testing facility and corporate offices in Bangalore, India, finding a spreadsheet they described as evidence of data manipulation for five studies. According to the complaint, the FDA notified drugmakers in April 2016 that the company’s clinical and bioanalytical studies were “not acceptable as a result of data integrity concerns” and began requesting information from customers who had undertaken studies with Semler. These actions led the European Medicines Agency and the World Health Organization to withdraw approvals of generics whose studies were conducted by Semler, the complaint said, with the exception of drugs serving immediate needs such as HIV and malaria. Semler claimed the data was entered by an “unauthorized, unknown person,” and the information was “verifiably inaccurate and non-representative of the study data.” The lawsuit accuses the FDA of negligence for failing to properly investigate the spreadsheet’s authenticity.

FDA’s Oncology Center of Excellence Marks First Anniversary

In its first year, the FDA’s Oncology Center of Excellence (OCE) has worked to coordinate cancer treatment reviews across the agency’s product centers, centralizing clinical assessments to help expedite development and speed approvals as research advances. “In this new era of cancer therapeutics development, biologic products like gene and cellular therapies and vaccines, and devices like next-generation sequencing in vitro diagnostics, are increasingly being integrated with drug therapies into patient care,” wrote OCE Director Richard Pazdur on the center’s anniversary, following 16 new approvals in 2017, including the first cell-based gene therapies and tumor site-agnostic treatments, as well as 30 supplemental approvals, two biosimilars and multiple in vitro diagnostics. The OCE has scheduled a public listening session for March 15 to hear recommendations from the industry regarding their expectations of the center, specifically, what stakeholders desire in terms of structure, function, regulatory purview and activity.

FDA Says Sponsors Seeking QIDP and Fast-Track Designations Must Apply for Both

Drug sponsors seeking the FDA’s Qualified Infectious Disease Product and Fast Track designations for antibiotics undergoing clinical trials must specifically request both designations, according to a new draft guidance from the agency. The QIDP designation makes a drug eligible for Fast Track and Priority Review, and also extends exclusivity for five years upon approval. Sponsors can request the designation prior to submitting an IND, but a request for Fast Track may only be made concurrently with or after an IND. An efficacy supplement to an NDA may also be granted the five-year exclusivity extension, if the supplement is for an indication that has already received a QIDP designation, and if the supplement qualifies for three-year exclusivity or orphan drug exclusivity. The FDA’s full question-and-answer guidance is available here: https://www.fda.gov/ucm/groups/fdagov-public/@fdagov-drugs-gen/documents/document/ucm594213.pdf.