Traditionally, the FDA makes its regulatory decisions based solely on demonstrated safety and provable increases in meaningful survival data. The FDA asks, Will a new treatment that is being considered for approval be able to extend our life longer than the current standard of care? They also ask if it will extend our life is it safe for us to take?
In cancers like prostate cancer these survival statistics take time to mature. In a way this is great news as it means that we tend to live for a good period of time with our cancer. On the flip side this also means that new drugs in clinical trials take excessive time to prove that they can extend our survival time.
Does this FDA policy work against us by allowing good drugs to linger longer than they should in the approval process, thus denying the treatment to men in immediate need? This question is especially relevant in the slower progressing cancers like prostate cancer. Does this process mean we have less approved drugs and does this process mean that our drugs, when approved, are more expensive because we are forcing longer and more expensive clinical trials?
Consumer groups and Congress have asked why is the FDA slowing down the approval process and forcing drug prices to rise? Increasingly, the FDA, in response to what is sometimes intense pressure, especially for cancer medications and other treatments for life-threating diseases, uses a program known as “Accelerated Approval” to approve drugs. There is a growing number of treatments using surrogates markers (i.e. a shrinking a tumor, slowing of disease progression and palliation) in place of hard evidence of increased survival to obtain regulatory approval. Often approval is made if such a measure is “reasonably likely” to predict a clinical benefit, such as increased survival or an improved quality of life.
Besides the obvious benefit patients receive there is an economic benefit for the pharmaceutical companies. Using surrogate endpoints allows drug manufactures to run shorter, smaller and, therefore, much cheaper clinical trials.
In theory this should translate into less expensive drugs for patients. But, has it? It doesn’t seem so. Of the five most expensive cancer drugs approved, four approvals were based on surrogate measures, yet these drugs are the most expensive. One could conclude that cheaper surrogate driven trials yield higher cost drugs!
What is the rational for the very high price? Simple, drug pricing often does not relate to cost. Not to development cost (as often cited by drug companies) and not to manufacturing costs. Drug pricing is set by what the pharmaceutical companies determine the market will bear. The real cost of development and manufacturing is now just an item in the decision making when determining end user pricing.
If drugs can be approved with less rigorous standards that save billions of development costs these savings should be passed back to society by making these drugs available at lower costs. We could argue that these drugs, which have received their approval with the use of surrogate markers, might be inferior because they have not been measured with the best bar, actual survival statistics. So, why should we pay a higher price and not an inferior (lower) price for an inferior product?
Society as paid the price of allowing less proven drugs into the market, possibly at the risk of patients, so there has to be an equitable pay back to consumers and to society.
Perhaps we have reached a time to put into place a limited price control system. We consumers are not dealing in a fair and open market when our life is the price, and neither are the pharmaceutical companies dealing in an fair and open market. Lets take this into consideration and level the playing field so that we can have the benefits of the drugs and the pharmaceutical companies can make a fair and reasonable return on their investments.
Joel T Nowak, M.A., M.S.W.
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