FDA’s mission to expand access to new drugs

Sep 15, 2018
FDA’s mission to expand access to new drugs

In 2017, the FDA approved 16 new oncology drugs and biologics, including the first two cell-based CAR-T cell therapies. A total of 30 new indications were also approved, along with the first two oncology biosimilars and three next-generation sequencing (NGS) platforms.

FDA Commissioner Dr. Gottlieb said: “This science is bringing forward more novel opportunities to more meaningfully address human disease at a pace that’s faster than ever before. But humankind is still much closer to the beginning than to the end of these endeavors.”

He went on to highlight a number of FDA initiatives intended to modernize the drug regulation process. These include the Oncology Center of Excellence (OCE) which has just marked its first birthday and has been tasked with breaking down “outdated silos” to foster a disease-focused approach.

Dr. Gottlieb also spoke about the FDA’s commitment to widen access to clinical trials and referenced recent calls for more pre-market studies on overall survival endpoints.

“I disagree. And I suspect some of the patients who face long odds, for whom available therapy gives them just a slim chance of long-term survival, might also disagree,” he said.

Building a regulatory system that serves those facing “long odds” depends on breaking down barriers between research and practice, as well as harnessing real-world data.

Dr. Gottlieb added: “It means enabling better access to experimental therapies being tested in clinical trials by modernizing eligibility criteria and conducting clinical trials in the communities where patients live. In my opinion, this transformation isn’t optional.”

Two new pilot projects will, he hoped, help the FDA drive that transformation. One is aimed at focusing submissions on data that’s most relevant to assessing safety and effectiveness.

“The goal is to improve the overall quality of applications and make sure resources and review time are being focused on evaluating data that’s most meaningful to clinicians and patients.”

The OCE is also piloting a “real-time oncology review,” which is currently focused on new efficacy supplements for approved cancer drugs.

“As soon as a sponsor locks their database and has decided they want to file for FDA approval, they’d start sharing the bottom line data with the agency,” he explained.

These are just some of the FDA’s current programs to modernize and streamline the approval process, he added.

“We’ll have many more announcements to make soon about how we’re embracing science to improve drug regulation,” concluded Dr. Gottlieb.

FDA’s mission to expand access to new drugs

Stakeholder Engagement is a function within Corporate Affairs at Astellas that focuses on creating, building and maintaining third-party relationships. We serve as a conduit between Astellas and external stakeholders to help improve patient outcomes, improve access issues and address patients’ unmet needs head on.

Leave a Reply