Emerging Issues in Food and Drug Law: Implementation of FDASIA Innovative Drug Approvals: Breakthrough Therapies Arnold & Porter LLP, Washington DC

Transcription

1 Emerging Issues in Food and Drug Law: Implementation of FDASIA Innovative Drug Approvals: Breakthrough Therapies Arnold & Porter LLP, Washington DC Ryan M. Hohman, JD, MPA Managing Director, Friends of Cancer Research

2 Courtesy of the American Association of Cancer Research 2011 Cancer Progress Report

3 A Unique Model to Advance Biomedical Research Each year Friends of Cancer Research (Friends) convenes conferences, forums and working groups, to address critical issues in the research, development and delivery of new drugs. These annual venues bring together leaders from federal health and regulatory agencies, academic research centers, patient advocacy organizations and the private sector to propose consensus solutions and develop a clear path forward on critical issues surrounding the development and regulation of drugs and therapies. Through our unique collaborative model, we have created a path to better drug development and approval through scientific, cultural, regulatory and legislative solutions.

4 TARGET CANCER: New Drugs Stir Debate on Rules of Clinical Trials By Amy Harmon September 18, 2010 Two Cousins, Two Paths: Thomas McLaughlin, bottom, was given a promising experimental drug (PLX4032) to treat his lethal skin cancer in a medical trial; Brandon Ryan, top, had to go without it..last year, each learned that a lethal skin cancer called melanoma was spreading rapidly through his body, the young men found themselves with the shared chance of benefiting from a recent medical breakthrough. Only months before, a new drug had shown that it could safely slow the cancer s progress in certain patients. Both cousins had the type of tumor almost sure to respond to it. And major cancer centers, including the University of California, Los Angeles, were enrolling patients for the last, crucial test that regulators required to consider approving it for sale. But when Mr. Ryan, 22, was admitted to the trial in May, he was assigned by a computer lottery to what is known as the control arm. Instead of the pills, he was to get infusions of the chemotherapy drug that has been the notoriously ineffective recourse in treating melanoma for 30 years. With chemotherapy, you re subjecting patients to a toxic treatment, and the response rates are much lower, so it s important to answer Are you really helping the patient? said Dr. Charles L. Sawyers, chairman of human oncology at Sloan-Kettering. But with these drugs that have minimal side effects and dramatic response rates, where we understand the biology, I wonder, why do we have to be so rigorous? This could be one of those defining cases that says, Look, our system has to change. Vemurafenib (PLX4032) received FDA approval for the treatment of late-stage melanoma on August 17, 2011

5 Getting Breakthrough Therapies to Patients A profound therapeutic breakthrough fundamentally alters the way oncologists think about a disease in terms of prognosis, treatment options, and quality of life of patients

6 Getting Breakthrough Therapies to Patients The 2011 Conference included a panel entitled: Development Paths for New Drugs with Large Treatment Effects Seen Early. The panel proposed scientific strategies to ultimately expedite FDA approval for a drug showing dramatic responses in the early stages of development while maintaining drug safety and efficacy standards. Goals of Breakthrough Therapy Designation Goal 1: Expedite drug development process for products that show remarkable clinical activity early Goal 2: Minimize the number of patients exposed to a potentially less efficacious treatment

7 Getting Breakthrough Therapies to Patients On March 8, 2012, Friends of Cancer Research Exec. Director introduced Breakthrough Before House Energy and Commerce Committee On March 22, 2012, Friends of Cancer Research held a congressional briefing titled Expediting New Treatments to Patients: FDA Approval Mechanisms Breakthrough Therapy designation received public endorsement from FDA, industry and academia. In the spring of 2012, the bipartisan Advancing Breakthrough Therapies for Patients Act was introduced by Senators Michael Bennet (D-CO), Orrin Hatch (R-UT) and Richard Burr (R-NC) and Representatives Diana DeGette (D-CO) and Brian Bilbray (R-CA).

8 Concept Scientific Whitepaper Bipartisan Legislative Solution Tool in use by FDA to expedite the approval of multiple drugs in 13 months

9 While people like to talk about polarization and gridlock in Washington, this bill is a victory for both bipartisanship and for the millions of American who rely on medicines and medical devices. Sen. Tom Harkin (D-IA) and Sen. Michael Enzi (R-WY)

10 7 Breakthrough Designations Announced By Companies So Far Vertex- 2 cystic fibrosis designations Ivacaftor in combination with VX-809 in patients with two copies of the F508del mutation Ivacaftor alone in other CFTR mutations Ibrutinib - (J&J/Pharmacyclics) - 3 designations: mantle cell lymphoma, chronic lymphocytic leukemia, waldenstrom macroglobulinemia LDK378 - (Novartis) ALK+ NSCLC resistant to crizotinib palbociclib - (Pfizer) - treatment of post-menopausal patients with ER+, HER2- locally advanced or metastatic breast cancer

11 Courtesy of AACR Annual Meeting 2013

12 We re also now just implementing the biosimilar statute, which is lengthy and it s a nightmare because we don t know what Congress meant when they say in any given patient. Statutes stand for a long time and sometimes are worded by nontechnical folks, words are a negotiation. This is worded extremely well. It sets a standard and then it allows us to grapple with the science. Our gratitude for the careful crafting of this. Dr. Rachel Sherman, FDA Associate Director for Medical Policy at 2013 AACR Annual Meeting