By Clinical Informatics News Staff 

July 29, 2015 | Following a 26-week study of 1,311 patients, Pfizer today announced that it is dropping efforts to have its cholesterol drug Lipitor cleared for over-the-counter sales. Pfizer had hoped the blockbuster statin, whose patent expired in 2011, would be a good candidate for an over-the-counter designation because it has simple instructions for use and a relatively good safety profile. In today’s quarterly earnings report, however, the company revealed that the FDA was not satisfied with the study results, ending an effort to regain a competitive advantage over generic versions of the drug. (At Forbes, John LaMattina, formerly head of R&D at Pfizer, writes more on the history and commercial implications of this Lipitor study.)

The Lipitor trial presented an interesting test for a changing healthcare system. Statins are an enormously widespread form of preventative medicine in this country, with more than a quarter of Americans aged 40 and over taking the drugs on a prescription basis to lower their levels of LDL cholesterol and their risks for heart attack and stroke. Shifting one of these drugs to over-the-counter might have improved access to a staple of modern care, while placing more responsibility for prevention in patients’ hands.

However, the FDA is extremely cautious about over-the-counter products ― particularly whether patients will use them appropriately without guidance from healthcare professionals. (For an example of how this has worked out in another field, see “What Comes Next for Direct-to-Consumer Genetics?” in our sister magazine Bio-IT World.) The trial the agency arranged with Pfizer simulated real-world over-the-counter sales by allowing subjects to pick up Lipitor doses at local pharmacies, with instructions to have their LDL cholesterol levels tested after starting the drug. Pfizer tracked whether patients followed those instructions, and whether they changed their behaviors appropriately based on the results.

It now seems the trial missed both endpoints, and conversations with the FDA did not produce any way to salvage the regulatory submission. Importantly, what matters to the FDA is not that patients can’t use Lipitor correctly on their own, but that in practice they won’t. That’s no great shock with a drug that must be taken daily, on a long-term basis, to guard against unfelt chronic symptoms; the perceived incentive to visit a doctor and get a cholesterol test would have been small. Still, the results are a letdown not only for Pfizer and its shareholders, but also for the many health advocates who hope to encourage patients to take a more active role in their own care before major illnesses set in ― confirming once again that they are up against deep-seated behavioral biases.

In the end, this trial may have been ahead of its time. Many healthcare providers and independent developers are working on mobile applications and care portals to help patients keep on top of prescriptions, contact their doctors, and form and stick to health management plans. It’s not hard to imagine that active users of these tools might have an easier time using drugs for chronic conditions over the counter, without neglecting basic follow-up measures. If enough healthcare systems adopted these tools and convinced patients to use them, or if pharma companies could build their own applications and coordinate their use with patients and providers, a trial like this might turn out differently.

For now, though, these kinds of technological solutions to the problems of healthcare are rare in the real world, where adherence counts.