Provenge wins favorable vote at CMS hearing

Physicians and researchers on a panel advising the Centers for Medicare & Medicaid Services (CMS) gave Provenge® a favorable vote at a “National Coverage Analysis” hearing yesterday in Washington, DC. You could almost hear the world of prostate cancer survivors, their physicians, Dendreon investors, and other drug development companies breathe a sigh of relief!

Provenge, technically known as sipuleucel-T, is the first autologous immunotherapy drug to be approved for any cancer by the U.S. Food & Drug Administration (FDA). It was approved in April 2010 for use in men with metastatic, androgen-independent prostate cancer who did not have symptoms or had only minimal symptoms. The key evidence came from a large clinical trial that demonstrated a survival advantage of 4.1 months (25.8 months median survival in the Provenge arm, versus 21.7 months in the “placebo” arm, which was actually a delayed, frozen version of Provenge given when disease progressed). That 4.1-month advantage was better than results for all therapies for metastatic cancer except three over the past 15 years. Another encouraging result was that 33 percent of patients in the Provenge arm of the trial were alive at the 3-year point, versus just 11 percent in the placebo arm. With survival results at least as good as those for current chemotherapy drugs, many eligible patients were attracted to Provenge’s strong safety profile and mild side-effect record, a marked contrast with the well-known rough side effect consequences of chemotherapy drugs.

After approval, Dendreon (the manufacturer) announced that the price for the three-treatment course would be $93,000, and subsequently CMS called for the National Coverage Analysis to determine whether Medicare would pay for the therapy. That was an unprecedented step, as CMS had previously automatically covered drugs once they had been approved by the FDA. While the law stated that Medicare was not to consider cost of therapies in its coverage decisions, many commentators and knowledgeable sources said that cost was clearly a concern.

A number of sources looked at CMS’s special review of Provenge coverage as a harbinger of future struggles for other newly approved drugs as the nation works its way through financial consequences of coverage for expensive health care in an era of budget austerity. It was evident that some in the public, including physicians used as authorities by reporters, did not understand the significance of a survival advantage of a few months for a drug for metastatic cancer in a clinical trial for FDA approval, nor did they understand the large investment behind a successful drug. (Dendreon reported spending about a billion dollars to get Provenge through FDA approval, and it estimated it would spend about a half-billion more to go into full production.)

The key step in the National Coverage Analysis was the hearing held yesterday. When the dust settled, the 14 physicians and researchers on the Medicare Evidence Development & Coverage Advisory Committee (MEDCAC) had voted an average score of 3.7 on a 1 to 5 scale, (with 5 as the best score) for coverage of Provenge for the patients falling within the approved indication of the drug as specified in the FDA labeling. Essentially, experienced observers considered this intermediate level of confidence a firm vote in favor of Medicare coverage for “on-label” use of Provenge. However, the MEDCAC advisors also disapproved of covering “off-label” use, which is use beyond the scope of the FDA approval. For example, a man with metastatic, androgen-independent prostate cancer but with intense symptoms would be outside the approved purpose of the drug. While his physician could prescribe the drug after explaining the benefits and risks, such uncovered off-label use would mean that Medicare would not pay for his treatment. Detailed score sheets from the MEDCAC meeting are available on the CMS web site.

As at the initial FDA hearing on Provenge, prostate cancer advocates and physicians with experience in the use of Provenge testified at the hearing and provided a supportive presence.

The next step will come in March 2011 with release of a draft coverage policy for public comment, and the last step is expected by late June with publication of the official policy. Meanwhile, 14 of the15 regional Medicare units are already covering eligible patients or have published coverage policies, so it appears that Provenge will be covered by Medicare for it’s on-label use, pending the final decision.

2 Responses

  1. Unfortunately I was at the meeting and commented. In Virginia we have a man whose doctor referred him for Provenge treatment. Virginia is approved on a case by case basis and since there is no approval he is being asked for a $20,000.00 deposit. Nothing has happened that will change that. Also patients in Mississippi, Alabama and Louisiana, states with high Black populations, will not be covered for this treatment. We still have problems that need to be overcome so that men who want and need treatment with Provenge can get it.

    Another aspect of the meeting that I found potentially troubling was that they discussed how it was necessary to do additional trials for treatments to get approval. They discussed the necessity for tissue banks so that the drug can be given to appropriate men. They also discussed the need for marker development so that we can identify who is appropriate for the treatment. Theoretically this is wonderful but if they in the future ask for this from the companies it could potentially delay new treatments and raise development costs. Medicare and the FDA have signed a memorandum of understanding that would potentially stop one agency asking for one thing and another asking for another. Trial planning and design is very important. They seemed to miss the fact that the landscape of prostate cancer treatment changes such that what is known or approved may not be the same at the beginning of the trial as it is at the end. One of the issues that was also brought up was that men with prostate cancer have a very poor clinical trial accrual rate and this also raises the cost of trials. The solution mentioned was that advocacy groups must work harder to educate men about clinical trials and work to get more men involved.

    While I support all these things, with potential cuts in funding I wonder where the money is going to come from to accomplish all these things.

    Unless more men get involved in advocacy, I do not know how we can accomplish these goals.

  2. That is too much money to heal someone. This makes no sense whatsoever. No wonder nothing ever gets cured. It is all just for money.

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