Medicines and Vaccines Part 1: The Pharmaceutical Pipeline

I often hear people express concern that pharmaceutical companies (Big Pharma) are making a killing on medications and vaccines and fear that we can’t trust them because of it. I also feel a lot of discomfort with pharmaceutical companies skyrocketing the prices for products like daraprim and epi pens. Drugs are really, really, really expensive to make and somebody has to foot the cost for developing them, but we also have people dying because they can’t afford the basic medications they need to live. There has to be a better way, but it’s hard to know what to think when the process is so mysterious. This piece is the first in a 3 part series on how medicines and vaccines are made and what they do. We won’t be able to cover every aspect of it because 1. that’s the stuff of dissertations and 2. I honestly don’t know all of it, but we’ll go through an overview of how the very basics.

To bring a new pharmaceutical drug to market costs an average of $5 billion and fifteen to twenty years, with more than nine out of every ten candidates being being disqualified along the path to FDA approval. For high-cost drugs to treat chronic conditions in wealthy nations (like erectile-disfunction drugs), this cost is easily made up in the time that the drug is on the market–particularly during the exclusivity period (the time when the drug’s original manufacturer gets to be the only manufacturer). This high cost can’t be completely recuperated, however, for low-cost, short-term pharmaceuticals like antibiotics and vaccinations. I sometimes struggle to conceptualise the process (so many large numbers!), but imagining the path from idea to market as a funnel into which as many as 10,000 compounds go in, but only a single safe and effective product comes out helps me.

In some cases, the process is random and begins with indiscriminate testing of thousands of compounds at a time to find a viable candidate; in others existing compounds are repurposed with the hope that they can be re-marketed. This was the case with the first drug used to effectively treat HIV—azidothymidine—which had originally been marketed as a cancer treatment. [1] This method is less costly for manufacturers as the compounds are already known to be safe for human use, though efficacy must still be proven. The type of drug being developed and the size of the researching institution or company’s reactant catalogue are the primary drivers in deciding which method will be utilized.

The time it takes a pharmaceutical to go from conception to market is a lengthy one, with a large portion of it covering the trial phase to gain FDA approval. The approval process for both standard and “orphan” drugs (ones that only work for single, typically rare conditions) comprises three clinical trial stages, using increasing numbers of patients at each stage and eventually and comparing patient who receive (“treatment”) and do not receive (“control”) the drug.

At each stage of the process, the pharmaceutical company is trying to decide if the efficacy of the product (how well it works) is good enough to warrant continued investment in it, since the stages get more expensive with the expanding number of human test subjects and fees levied directly by the FDA. The table above from the Manhattan Institute for Policy Research outlines the cost at each phase of clinical trials for FDA approval. The preclinical/prehuman stage is vey expensive on its own and comprises the first two portions of the pipeline above, meaning it is almost half the time it takes to bring a single product to market.

In our next piece, I’ll talk about how pharmaceutical companies and nonprofits partner to tackle the high cost of bringing “essential medicines” like vaccines and antibiotics to market. We’ll talk about groups like the Eli Lilly, Merk, IDRI, the Gates Foundation, and PATH. As with this section, we won’t be able to dig into every aspect of the process, but we can shed some light on how we get vaccines for diseases like Rotavirus.

Garfield, S. The rise and fall of AZT: It was the drug that had to work. It brought hope to people with HIV and Aids, and millions for the company that developed it. It had to work. There was nothing else. But for many who used AZT - it didn’t. The Independent. 2 May 1993. http://www.independent.co.uk/arts-entertainment/the-rise-and-fall-of-azt-it-was-the-drug-that-had-to-work-it-brought-hope-to-people-with-hiv-and-aids-and-millions-for-the-company-that-developed-it-it-had-to-work-there-was-nothing-else-but-for-many-who-used-azt--it-didnt-2320491.html

By: Amanda