Are Consumers Fully Informed About Cancer Drugs Given Accelerated Approval?

These remarks were made during the public comment period at the FDA's Oncologic Drugs Advisory Committee Meeting, March 12, 2003

I am Maryann Napoli, the associate director of the Center for Medical Consumers in New York. We are a non-profit advocacy organization that has never taken pharmaceutical industry funding.

Because our center was founded to promote informed medical decision-making, I have spent a lot of time listening to people and helping them make cancer treatment decisions. In 25 of the 27 years of our Center's existence, we have had a medical library that is open to the public. The people we attract are those who weigh and consider the evidence before going on a cancer drug regimen. In the years I've spent listening to people, I have been struck by the disconnect between what the oncologists say to their patients and what the patients hear. For example, oncologists, when asked about efficacy, often use the term response rate, but the patient inevitably hears survival rate.

I think that most people would be shocked to know how unreliable tumor response is as an endpoint and that it was the basis for the accelerated approval (AA) of ten out of 11 cancer drugs and the sole basis for 10 of the 55 drugs given regular approval between 1990 and 20011. Consider what most cancer patients want from a drug-a significantly prolonged life without side effects that are too horrendous. I applaud current efforts to make clinical benefit the required endpoint. And I'm glad that ODAC is rethinking the AA process because it allows expensive, minimally tested drugs on the market to enjoy a long period of unearned hope and acceptance. AA drugs have never been compared to the standard care to determine whether they are better or worse.

No matter what you decide to do as a committee, cancer patients must have a way of understanding the basis for approval, be it regular or accelerated. I have looked at the label for each of the drugs to be discussed at this meeting and concluded that the average intelligent consumer could easily miss their AA status when reading the Physicians' Desk Reference. Sure, you can read the label and see descriptions of Phase II trials that show complete or partial responses. But what does that mean to consumers? And yes, people can go to the FDA web site where they can see the list of drugs given AA. But the explanation of AA is not readily understandable, nor does it explain tumor response and how debatable it is as a good surrogate for prolonged survival or even symptom improvement.

Most manufacturers of drugs given AA haven't completed the required phase IV trials, but you'd be hard pressed to find that out-unless you read the Wall Street Journal.2 The FDA web site lists each drug's date of approval, but not the status of those required phase IV trials. We advocates who write, "translate," and assimilate information for people with cancer need to know this information. We need to know whether companies are complying with this regulation and how long it is taking them to do so.

All cancer drugs should come with written information that is understandable for the average intelligent consumer who needs a summary of the supporting scientific evidence. In fact, there should be something like a black box warning to alert the consumer of a drug's AA status.

For additional information see "How Well Tested Are the New Cancer Drugs?"

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1 Telephone interview with Richard Pazdur, MD. Director of Oncologic Drug Products, FDA, August 28, 2002 in preparation for article entitled "How Well Tested are the Newest Cancer Drugs?" September 2002 issue of HealthFacts newsletter, published by the Center for Medical Consumers.
2 Chris Adams. "Test Data for Some Drugs Long Overdue at the FDA." Wall Street Journal, January 28, 2003.