Drug Shortages: Blame Policies, Not Agencies

A recent article in the Washington Post by Rob Stein pointed out shortages of 211 medications in 2010, including lifesaving drugs used in emergency rooms and oncology wards ("Shortages of key drugs endanger patients"). What is causing this shortage? "Experts cite a confluence of factors," writes Stein:

Consolidation in the pharmaceutical industry has left only a few manufacturers for many older, less profitable products, meaning that when raw material runs short, equipment breaks down or government regulators crack down, the snags can quickly spiral into shortages.

Stein points out that there are especially acute shortages of generic medications (which aren't very profitable for manufacturers), especially sterile injectable medications (whose manufacturing processes are complicated and error-prone). There are also shortages of raw materials (often imported from abroad).

Stein cites the shortage of the leukemia and lymphoma drug cytarabine due to problems obtaining raw materials and manufacturing the drug. Cytarabine is a vital cancer drug that many hospitals have been forced to ration to patients. Inadequate medication substitutions have also lead to patient deaths, he said.

It's easy to blame the FDA for some of the problems with the drug supply. As Stein explains, "some industry representatives blame part of the problem on increased oversight by the FDA, which has made drug safety a higher priority after coming under intense criticism for being too lax." If the FDA would just skip a few manufacturing facility inspections, the supply pipeline would be smoother?

Drug supply problems don't originate with the FDA, however. Pharmaceutical manufacturers, eager to turn a healthy profit for their investors, would rather chase the next blockbuster drug (earning $1 billion or more in profits yearly) than thanklessly churn out low-profit items such as generic drugs and vaccines. Increasingly, venture capital firms that might fund new drug development would rather fund profitable new technologies than invest in better treatments for diseases.

The financial market, while it plays a role in drug development, should not drive public health decisions. The antidote to this problem is thoughtful legislation. Laws such as the Orphan Drug Act, which I've written about before, have successfully helped pharmaceutical companies refocus some of their energies on patient needs rather than profits.

Maybe the FDA could require that a company whose FDA-approved drug reached blockbuster status must ramp up its generic manufacturing to a certain level - building more manufacturing plants for a needed drug, or adding a popular vaccine to its roster -  before any more drugs are approved. Why not? Stronger regulations and incentives can encourage pharmaceutical manufacturers to diversify their assets and create a safer and more stable supply of drugs for everyone who might need them some day. 

Big Bucks and Orphan Drugs

I had the opportunity a while ago to cover a pharmaceutical company’s drug development meeting. The meeting took place at a lovely and incredibly expensive hotel, and the salmon and petit fours for lunch were a far cry from my usual refrigerator-browsing when I work at home. I knew that the pharmaceutical industry was exceedingly wealthy, but I hadn’t seen it up close before.

The people at the meeting included deferential twenty-something sales representatives and assistants; opinionated mid-career researchers and doctors, who occasionally cracked incomprehensible jokes involving biochemical formulas; and a polite and self-deprecating high-level executive. I’m sure there were plenty of office politics and power plays behind the scenes, but as an observer what I saw was a well-oiled machine. The real politics were external: a corporation versus the FDA (“the Agency,” they called it). The pharmaceutical company was starting human trials of a new drug that they hoped to bring to market if all went well.

For all the people at the meeting, and the expense and effort of bringing everyone together (many from out of town), the new drug was not a potential blockbuster. It was an orphan drug. Orphan drugs are developed to treat rare (orphan) diseases, defined as: diseases that affect 200,000 or fewer Americans, and/or diseases that are uncommon in the developed world (although they may be common elsewhere).

In the past, orphan drugs were too unprofitable for pharmaceutical companies to develop. In 1983, however, the federal government passed the Orphan Drug Act (ODA) to provide tax incentives, research grant money, and marketing benefits to companies that develop orphan drugs. The ODA has had a huge impact on orphan drug development. According to the FDA, from 1973 to 1983, about one orphan drug was brought to market per year. Since 1983, about 250 orphan drugs and products have been brought to market – averaging about ten each year.

The drug in question at the pharmaceutical meeting was designed to replace the current drug treatment, which has several miserable side effects that inhibit patient compliance, for a disease so rare that there might be more people involved in developing and regulating this new drug than there are potential patients for it. But if it survives the trials and the FDA and is brought to market, the drug could vastly improve the quality of life of these patients. It was good to see such altruism from a pharmaceutical company, even if it was prompted by a big carrot from the federal government.