Skip to content

Answering a Compelling Need: Expediting Life-Saving Treatments to Patients

Answering a Compelling Need: Expediting Life-Saving Treatments to Patients



 Senator Michael Bennet (D-CO)


Janet Woodcock, Director, CDER, FDA
Ellen Sigal, Chair, Friends of Cancer Research
Jay Siegel, Head of Global Regulatory Affairs, Johnson and Johnson
Jeffrey Leiden, CEO, Vertex Pharmaceuticals
William Elder, Jr., Advocate, Cystic Fibrosis Foundation
Moderator: Kate Rawson, Senior Editor, The RPM Report

This is a widely attended event.

On July 24, 2013, Friends of Cancer Research (Friends) hosted a congressional briefing titled “Answering a Compelling Need: Expediting Life-Saving Treatments to Patients” to discuss the Food and Drug Administration’s (FDA’s) Breakthrough Therapy designation. The newest of several programs FDA employs to expedite the development and review of promising drugs,  Breakthrough Therapy designation gives particular attention to drugs exhibiting early clinical evidence of substantial improvement over existing treatment. Just over a year after Breakthrough Therapy’s signature into law as part of the Food and Drug Administration Safety and Innovation Act (FDASIA), this briefing examined the policy’s progress and continued implementation. Friends provided outlines of the Breakthrough Therapy designation and other FDA Expedited Review Programs, as well as a report Friends recently published describing the clinical endpoints used for oncology drug approvals over the past decade.

The Breakthrough Therapy designation has already seen greater-than-expected utilization. Since January, twenty-five drugs have been granted Breakthrough status. Panelists from FDA, the pharmaceutical industry, and patient advocacy groups gathered to discuss the successes, the implications, and the ongoing challenges of expedited drug development and approval.

Long-time medical research advocate Senator Michael Bennet (D-CO) gave opening remarks, praising the bipartisan nature of the discussion, the work of its participants, and the rapid impact of Breakthrough Therapy. He thanked the panelists for their ongoing efforts in regulatory reform, highlighting the humanitarian and economic value of bringing safe, effective drugs to market as efficiently as possible.

Dr. Janet Woodcock, Director of the FDA Center for Drug Evaluation and Research began the panel discussion by outlining the purpose and promise of the Breakthrough Therapy designation. As new drugs become increasingly targeted, developers and regulators see unprecedented clinical impact as early as Phase One trials. “If the drug is so much better than anything out there for that disease, we don’t want to wait seven years while it’s tested in Phase One, Two, and Three.”

She noted that FDA involvement must be cross-disciplinary and timely, as expedited drug development could leave manufacturing requirements, an aspect of drug development that is often addressed toward the end of the process, as the rate-limiting step in bringing new drugs to market.

Dr. Jeffrey Leiden, CEO of Vertex Pharmaceuticals, the developer of the Breakthrough-designated cystic fibrosis drug Kalydeco, described communication with FDA under the new program: “It’s a different kind of conversation. It’s iterative. It’s continuous. It’s pick up the phone if you have a problem. It’s collaborative. Everything is on the table: the trial size, the trial design, the statistical analysis, the manufacturing process. And that makes the progression of the trial and the progression of the development immeasurably smoother and easier.”

Dr. Jay Siegel, Head of Global Regulatory Affairs for Johnson & Johnson, sponsor of Breakthrough-designated cancer drugs ibrutinib and daratumumab, echoed the sentiment, stating that ibrutinib’s expected approval date has been accelerated by up to two years. “It’s not just that we can pick up the phone and call, but they’ll pick up the phone and call us and say ‘Here’s a way you might do this faster.'” William Elder, Jr., a cystic fibrosis patient taking Kalydeco and speaking on behalf of the Cystic Fibrosis Foundation, discussed his experience with an expedited drug. “This Breakthrough designation changes everything. It changes the way we see our future.”

In response to a question by Moderator Kate Rawson, Senior Editor for The RPM Report, Director Woodcock described the number of Breakthrough-designated drugs as the result of a fundamental scientific shift. “We’re really seeing a new kind of drug development that’s driven by understanding the basic science of the disease and intervening on those pathways. There’s a tremendous recognition by the clinical staff that this is different.” Though she acknowledged that it is still vital to review all drugs efficiently, Breakthrough drugs present such an enormous opportunity that “We can’t afford not to do this.”

Dr. Ellen Sigal, Founder and Chairperson of Friends, pointed out that active FDA involvement with Breakthrough-designated drugs is resource intensive and expressed concern for the impact of the Sequester on already tight budgets. “This is about a collaborative scientific process. None of these Breakthroughs came out of the air. They’re a result of the hard work of the NIH, academic researchers, patients, industry and FDA coming together to improve people’s lives.” To cut research and regulatory funding is short-sighted. “If we treat the right population with the right drugs, we will save money, in addition to a lot of pain and suffering.”

Dr. Leiden agreed. “The next generation of these remarkable precision medicines won’t be there if we cut away at the beginning of the process at the NIH and at the FDA.”

Dr. Siegel expressed concern for mismatched expectations between FDA and foreign regulators, particularly regarding Breakthrough-designated drugs. Where FDA attempts to move patients from control groups to effective medicines when possible, European regulators may require longer-term monitoring before any trial adjustments could be made. “Our hope is that foreign regulators will catch up.”

Even after initial approval, panelists anticipated challenges regarding how insurance providers would determine reimbursement for drugs that went through the Breakthrough program. Because of their accelerated development process, Breakthrough drugs can demonstrate the safety and efficacy required to reach the market prior to developing long-term measures on outcomes sought for cost-benefit analysis both overseas and, increasingly, in the United States.

Further discussion touched on the role companion diagnostics play in drug development. Though they will be required for many Breakthrough drugs, their development and manufacturing are not directly subject to the benefits of the Breakthrough Therapy designation.

Dr. Sigal noted Friends’ ongoing work with CDER and the FDA Center for Devices and Radiological Health (CDRH) to explore strategies to expedite the co-development of diagnostic tools with drugs that have received a Breakthrough Therapy designation. Friends will hold a public forum on the subject on September 6. Additional topics of discussion included a desire by both FDA and industry to see the manufacturing process improved, the benefits of publicly-funded research to drug development, and the possibility of large-scale clinical trial networks to ease the trial process for drugs treating rare conditions, where patient volunteers are often scarce.

Overall, panelists were supportive of the Breakthrough Therapy designation and optimistic for the future of drug development and review. So long as the collaboration, the innovation, and the funding that made Kalydeco possible continue, the future of medicine is bright. Said Dr. Leiden, “I think we’re standing at the threshold of the most exciting era in biomedicine that we’ve ever seen. And that’s partly scientific, it’s partly regulatory. That’s what Breakthrough is about.” Dr. Sigal wrapped up the conversation emphasizing that all parties do their part and “keep this going, because ultimately this collaboration is going to help patients.”

Inside Health Policy – Stakeholders, FDA Working on ‘Breakthrough’ Dx Proposal

Reuters – U.S. Drugmakers Cheer ‘Speed Lane’ for Breakthrough Therapies

Pink Sheet Daily – FDA’s Breakthrough Designations Face Question of Review Resources

Bloomberg – Breakthrough Status May Spur J&J Drug to Market Early

FierceBiotech – Johnson & Johnson, Vertex Execs Speak Up for ‘Breakthrough’ Status in Washington

Pink Sheet – FDA Breakthrough Troubles Emerge in Effort to Speed Development