Opinion requested: Should new drugs always be compared not just with placebos but with old drugs for the same conditions?

While I agree this would make determining drugs more effective for treating patients, this process will likely never happen. As it stands now, most clinical trials for investigational drugs are not designed to evaluate if the new drug is going to be more effective or safer than a previous drug, but only to prove that the agent being tested will work for the disease/condition the company is applying for. It’s real purpose is making sure any adverse effects or reactions are not severe enough that it would overstep the risk vs benefit comparison. We have to keep in mind that the FDA drug approval process is really nothing more than the government stepping in to say, “Hey, does this drug do what it says? But above all, can it harm or kill people?” The FDA is essentially a reactionary regulating body. Prior to the establishment of the Food Drug and Cosmetics Act (FDCA), which gave the FDA the power it has now, they had little to no regulatory power. The passage of the FDCA itself was largely due to the sulfanilomide disaster during that time.

Also, let’s look at who is actually paying for these clinical trials. There are grants and foundations here and there that help with mitigating some costs, but largely they are funded by the pharmaceutical companies. Costs for running clinical trials generally range from millions to tens of millions, and since the companies have a vested stake in the result of their product being successful, the data they want has to be as convincing as possible that their agent works. That’s the reason that most of the studies are going to compare to placebo, and not an API. There are definitely some trials that do compare active ingredients, called active-treatment concurrent control studies, but if you think about it from a financial standpoint, a pharma company will generally avoid those sorts of trials unless pre-clinical or Phase I testing has shown great promise. To be asking those companies to compare a potential pipeline agent is like asking a trader to only invest in high risk stocks.

This current system isn’t exactly efficient, nor does it really benefit the general population. The industry landscape is changing however, since we are moving into the direction of outcomes based medicine. With the fall of the patent cliff and fewer drugs in the pipelines, pharmaceutical companies are hard pressed to recover that lost profit. One way we can combat the rising power of third-party payer is to prove that their current brands or pipeline drugs are more effective than existing therapies. If we can replace the existing “gold standard” with a a newer agent, then everyone wins. However, for a pharmaceutical company to shift focus from their existing drug development phase to this new method requires substantial risk. In my opinion, if an industry giant were to take this leap of faith, and others follow, we may be able to both improve the quality of care for patients, while also create a potential new revenue stream. There are two ways to deal with the rising power of third-party payers: fight them and delay the inevitable, or find a way to use that changing landscape towards your benefit by changing your existing structure to work with the new dynamic.