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Commissioner's Address

Margaret A. Hamburg, M.D.
Commissioner of Food and Drugs
Annual Meeting of the American Society of Clinical Oncology
Chicago

June 2, 2013


Thank you, Dr. Swain, and good afternoon everyone.

It is an honor to join you today.  This organization and the FDA have forged close ties over the years, and to paraphrase the theme of your annual meeting, we have built a strong bridge that connects us in our shared quest to conquer cancer.

That connection has served patients well, most recently in ASCO’s robust support for FDA’s crusade to do everything possible to prevent drug shortages—and to mitigate them when they occur. 

But our connection goes back a long way.   ASCO has co-hosted workshops with FDA; we have worked together to design programs for community oncologists, and we have been able to provide—in real time—important FDA updates to your members, including a jointly developed email notification program to ASCO members that rapidly informs them about FDA drug approvals and other important developments. 

Let me mention one special area of cooperation.  For several years, FDA has been privileged to host fellows participating in ASCO’s Leadership Development Program.  I understand that the fellows consider their visit to the FDA to be one of the highlights of their program—I certainly hope this is true.

This afternoon, I’m delighted that I will be able to join you in honoring my colleague Dr. Rick Pazdur, but before that I’d like to outline some of the progress we’ve made in oncology and also provide an update on three issues that I think will likely be of special interest to oncologists:  the new expedited program for breakthrough therapies, the development of cancer drugs with companion diagnostics, and laboratory-developed tests.

This is an exciting time for the oncology community.

I’m sure you would agree with me that the last decade has witnessed significant improvements in cancer drug development.  Just consider the therapies you had to work with just a decade or so ago and what you have available today. 

Patients facing diseases—such as CML, renal cell cancer, and multiple myeloma—have multiple treatment options that improve and prolong life. And oncology diseases are being redefined based on their molecular pathways.

Many of these encouraging developments have taken place on Rick Pazdur’s watch as the director of FDA’s office of hematology and oncology drug products.

Rick, who joined FDA in 1999, has never been a status quo kind of guy. His sense of urgency and commitment to progress pervades all that he does. And he has never taken a one-size-fits-all approach to cancer drug development. He recognizes that the appropriate balance between benefits and risks depends on the product and the disease being treated. While respecting the complexity of the process, and the integrity of the science, he has excelled in using our expedited approval pathways to bring promising products to market as quickly as possible.

Under his visionary leadership, new oncology treatments have been consistently available in the United States before they were available in Europe.  But that doesn’t mean we view our regulatory counterparts abroad as rivals.  In fact, we work closely with them to understand potentially differing regulatory viewpoints and make promising new therapies available sooner. For example, with Rick’s leadership, we hold monthly telephone conferences with our colleagues at the European Medicines Agency and Health Canada to coordinate our work, both in the review of specific applications and to discuss the advancing science that is the basis for our regulatory decisions.

And the rapid pace to meet the urgent need for oncology drugs has continued. In 2012, nearly 40 percent of the new molecular entities the FDA's Center for Drug Evaluation and Research approved were for oncology indications.

And I want to tell you about a new tool that should help bring desperately needed therapies to cancer patients even sooner.  Thanks to the passage last July of the Food and Drug Administration Safety and Innovation Act—or FDASIA, as we call it—cancer drug developers have another expedited program available: breakthrough therapy designation. It can be used when preliminary clinical data suggest that a new drug holds real promise of substantial improvements over available therapies to treat a serious condition.

In addition to all of the features of fast track designation, a sponsor that receives a breakthrough designation will usually receive more intensive guidance on an efficient drug development program, beginning as early as phase 1, as well as the involvement of senior FDA management.

As of May 23rd, FDA’s Center for Drug Evaluation and Research had received a total of 51 breakthrough therapy designation requests, of which 20 have been granted.  Eight of the 20 breakthrough designations have been for oncology drugs. 

We’re currently developing a guidance document that will describe this novel program and explain the various criteria—including what we mean by such concepts as "available therapy," "unmet medical need," and "serious condition."

I noted that oncology diseases have been redefined based on our growing knowledge of molecular pathways. This has led to the use of companion diagnostics to identify the right therapies for the right patients.  An early example of this strategy was DAKO’s HercepTest, which we approved back in 1998, to target patients with the Her2 gene for treatment with Herceptin.  

There have been other examples, including Zelboraf for late-stage melanoma and Xalkori for late-stage lung cancer in 2011, each of which was approved with a companion diagnostic.

More recently, on May 14, we approved the cobas EGFR Mutation Test, a companion diagnostic for the cancer drug Tarceva (or erlotinib). This is the first FDA-approved companion diagnostic that detects epidermal growth factor receptor (EGFR) gene mutations, which are present in approximately ten percent of non-small cell lung cancers.

And just last week, on May 29, FDA approved Tafinlar (dabrafenib) and Mekinist (trametinib) for patients with advanced or unresectable melanoma. Tafinlar is intended for patients whose tumors express a single BRAF gene mutation, V600E; Mekinist is intended for patients who express that mutation or the V600K mutation.

These drugs were approved with a companion diagnostic that will help determine if a patient’s melanoma cells have one of the two BRAF mutations.

Advanced diagnostics such as these are the cornerstone of personalized medicine, and their development can only foreshadow the many advances on the horizon.  This is truly an exciting time in the history of cancer therapies and their companion diagnostics. 

Unfortunately, not all complex diagnostics used in cancer diagnosis or treatment have been developed to perform at the same demonstrated standards.

There is a category of diagnostics called laboratory-developed tests which are produced in and offered by laboratories for use in their own facilities. LDTs are currently marketed without FDA premarket review to determine whether they are safe and effective – whether they are accurate and clinically valid.

And that can be a problem. As many of you may recall, in 2008, "OvaSure," an early-stage ovarian cancer screening test, came onto the market.  For high-risk patients and their doctors, this simple blood test appeared to offer great promise for combating the disease and providing peace of mind.  Although FDA did not consider OvaSure to be a laboratory-developed test, it was offered as an LDT and thus did not undergo adequate clinical validation before the test was used across the country.

Thanks in large part to the involvement of the oncology community, together with FDA, this flawed test was eventually withdrawn from the market, some four months after it had been launched. 

Historically, FDA exercised enforcement discretion—that is, it did not generally enforce applicable regulatory requirements for these devices, because they were relatively simple, low-risk tests performed on a few patients being evaluated by physicians at the same facility as the lab. 

But LDT’s have become more sophisticated and complex. Results from these tests are rapidly becoming a staple of medical decision-making, particularly for cancer.  And some people with a family history of cancer are using these tests to decide whether to take preventive action.

FDA does not know how many women may have received erroneous results from the OvaSure test, or how many may have used that flawed information to make critical medical decisions.  But relying on advanced diagnostics to make critical, life-altering treatment decisions exposes patients to obvious risks if these tests do not perform as expected. False results put patients at risk of a missed diagnosis or a wrong diagnosis that could result in either inappropriate treat or no treatment at all.

The Agency is working to make sure that the accuracy and clinical validity of high-risk tests are established before they come to market.

The risk-based framework we have under development will ensure that diagnostics used in cancer treatment will provide medical professionals with a critical baseline for confidence in the tests they order for their patients.

Our intent in considering what to do about LDTs is to provide for safe and effective diagnostics while promoting innovation and patient access.

In fact, what motivates us every day is getting safe and effective innovative medical products to patients who need them.   And this brings me back to Rick Pazdur.

There’s a lot more I could say about Rick’s ceaseless and creative work to conquer cancer.  But we’re on a tight schedule, so I’ll stop here.  Let me just add that I am absolutely thrilled to join  Dr. Link in presenting Rick with this award that he so richly deserves. 

Thank you.