We Need Incentives for New Anticancer Drugs We need to address incentives that will lead to new anticancer drugs for rare cancers. The human stakes are about a million person years lost in the U.S. for lack of effective chemotherapy agents.
http://www.wsj.com/articles/we-need-incentives-for-new-anticancer-drugs-1451672879
Marty Makary’s “One Pharm Fix: Limit the ‘Orphan Drug’ Incentives” (op-ed, Dec. 21) addresses shortcomings of the Orphan Drug Act that lead to increased costs to consumers and insurers. While better controls of financial oversight of orphan drugs might lead to lower medical costs and reduce patient expectations for some of the unsupported off-label claims, I would argue that the Orphan Drug Act does not provide enough incentive for the development of drugs to treat low- and mid-grade cancers such as primary brain gliomas and medulloblastoma tumors that I have treated for 43 years.
The drugs needed may take many preclinical years to develop and 12 years to do the clinical trial required by the FDA. In addition, one drug is likely to be insufficient for tumor control and two to three drugs targeted to specific pathways may be needed. Complicating this is the likelihood that one drug may provide limited antitumor efficacy and two to three drugs together may be needed to control tumor growth and future transformation to a more malignant glioblastoma. In this case, Orphan Drug 7-year exclusivity is an inadequate incentive as the drugs may easily take 15-17 years to develop and test and hundreds of millions of dollars in cost, leaving insufficient time to recover costs associated with this risky undertaking to develop chemotherapy for these rare tumors. This argument is also true for many other low- and mid-grade solid cancers.
We need to address incentives that will lead to new anticancer drugs for these rare cancers. The human stakes are about a million person years lost in the U.S. for lack of effective chemotherapy agents.
Victor A. Levin
Larkspur, Calif.
Dr. Makary urges fundamental changes in the Orphan Drug Act, alleging that some drugs developed for rare diseases in fact have become blockbusters, treating other diseases.
We within the rare-disease patient community regard this 1983 legislation as one of the most successful laws ever enacted to support our community. It provides incentives for the development of new drugs for patients with rare diseases. And it has been successful.
The commentary surprisingly does not even use the word “patient” until the very last sentence. Our view is that the focus of any discussion about drug development should be on patients—in the case of rare diseases, the one in 10 Americans who has or will develop a rare disease, and then encounter a 95% likelihood of zero treatment. This is the public health issue that the Orphan Drug Act addresses.
The author cites his own study that relied on sales figures to indicate how widely a drug product is used. The more appropriate measure of drug use is how many patients receive it. Many recently approved drugs are expensive, especially biologics, and so the sales figures for them can be high. Thus sales figures are not an indicator of actual product use or value.
We all would like drug prices to be lower. But it takes an average of 12.5 years and $1.5 billion (in 2014 dollars) to bring a new drug from the preclinical stage through FDA approval. And each drug development project is highly risky. Most fail. The costs and risks increase when you factor in the challenges of working with small, dispersed patient populations in the rare-disease community.
It should be seen as welcome news that therapies developed for a limited patient population can and do provide value to others. As drug developers invest in possible new uses of their products, they seek FDA approval when the data support it. We need to continue to trust that the FDA will implement the Orphan Drug Act with discretion and make sound judgments in determining which drugs qualify for the incentives provided for in the law.
Peter L. Saltonstall
President and CEO
National Organization
for Rare Disorders (NORD)
Danbury, Conn.
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