Pharmaceutical innovation has led to the development of incredible new prescription drugs that can treat many diseases and ensure that consumers have a good quality of life. But rising drug prices are threatening this, especially for drugs that treat rare diseases. In a new article, the New York Times examines skyrocketing drug costs and their impact on people with these illnesses, and how they are often forced to extreme measures to get the medicine they need to stay alive.
While rare diseases affect a small number of people, there are a lot of rare diseases-over 7,000 of them. About 30 million Americans have a rare disease, almost one tenth of the population. In 2018 the FDA approved 59 new drugs in 2018, and 34 of them were for rare diseases. This is no accident; brand drug companies are facing increased competition from affordable generic drugs, and they are often investing in a few very expensive treatments in order to make greater profits.
The results are costly for almost everyone. Ohio resident Dawn Patterson has a rare and painful bone disease, and the drug Strensiq can stop the pain. But Strensiq is enormously expensive, costing about $16,133 for a supply of 5.4 milliliters. In 2018, her bill for this drug was almost $2 million! This threw the union that covers her health care into a crisis. Alexion, which manufactures the drug, did not respond to requests for comment, but it finally announced that it would cap annual costs for Strensig at $1.5 million.
This is unacceptable and not even close to a solution. $1.5 million for a lifesaving drug is far too expensive, and puts Strensig out of reach for all but the very wealthiest Americans. Even if consumers have excellent coverage, the costs of paying for that drug would harm health payors, and contribute to rising costs and waste in health care.
The New York Times wrote that "the impact is already being felt. Some small businesses, hit with just a single claim for a family like the Pattersons, have considered ending their employee health coverage. Others have drastically cut back coverage for drugs, and some employers are considering excluding coverage for expensive and novel treatments like gene therapy." And the number of cases involving incredibly expensive drugs is rising.
What are some solutions? To begin with, there should be complete transparency throughout the drug manufacturing, distribution, and supply chain so that consumers and policymakers can see where the money is going. Second, the federal government should have the power through Medicare and other programs to negotiate lower drug prices and reduce costs, therefore driving prices down. Third, pharmacy benefit managers (PBMs) that contribute to higher drug costs should be reined in. And finally, there should be laws to prevent price gouging and ensure that drugs for rare diseases are reasonably priced.
A $1.5 million price tag is not good or sustainable-not for consumers, not for payors, and not for our prescription drug and health care systems.