Conference Coverage
ALEXANDRIA, VIRGINIA—When Representative Henry A. Waxman (D–California) held the first hearing in Congress on rare diseases in 1980, 10 drugs developed by US pharmaceutical companies to treat rare diseases were on the market. Today, three decades after the 1983 passage of the Orphan Drug Act, which Rep. Waxman championed, the FDA has approved more than 450 orphan drugs to treat the more than 25 million Americans living with rare diseases.
To ensure that the accelerated growth of such treatments continues, Congress must increase funding for the NIH, retool current legislation designed to encourage the pharmaceutical industry to undertake more rare disease research of its own, and—above all, in Rep. Waxman’s estimation—come to terms with the high cost of orphan drugs.
“We can celebrate that we’ve accomplished a lot, but there are thousands of rare diseases without treatments,” he said in a keynote address at the National Organization for Rare Disorders (NORD) Rare Diseases and Orphan Products Breakthrough Summit. “The cost of many orphan drugs is still far too high. We need to continue our quest to ensure that the rapid pace of development of orphan drugs continues while preserving patients’ access to these drugs by keeping the price tag at a reasonable level.”
The Hard-Knock Realities of Orphan Disease Drug Development
Excessively high drug prices, including those for orphan drugs, are unsustainable for the US healthcare system, according to Rep. Waxman. Among the examples he cited was Kalydeco (ivacaftor), which the FDA approved in 2012 for the treatment of a rare form of cystic fibrosis and which the Congressman reported costs more than $300,000 per year. Nearly one-third of orphan drugs net more than $1 billion in annual sales, he noted.
“In 2012, the leukemia drug Rituxan [rituximab] had sales of over $7 billion,” Rep. Waxman said. “Another blood cancer drug, Revlimid [lenalidomide], had sales of over $3.7 billion. And the multiple sclerosis drug Copaxone [glatiramer acetate injection] had sales of over $4 billion. Perhaps these drugs would be justified if we looked at the cost of the development of the drugs, but they don’t really compare…. We’re seeing extraordinarily priced drugs bearing no relationship to the cost of their development.”
Driving up the cost of any new drug is the development cost to the manufacturer of previous drugs that did not succeed, which Rep. Waxman believes should be weighed against the savings gained when new drugs are effective. Without these savings, the costs of medical care for patients with orphan diseases would skyrocket.
“I don’t know what the answer is to this problem, but there should be a more rational relationship between the cost of the drug’s development and its price, and this is, of course, not a problem specific to orphan drugs,” he said. “From the perspective of the sustainability of our healthcare system, it’s becoming an even bigger problem with new specialty drugs intended to treat millions of patients…. The poster child of such drugs is Sovaldi [sofosbuvir], which, at $1,000 a pill costs $84,000 for a course of treatment for hepatitis C.”
Although Sovaldi’s sales for the first half of this year reached $6 billion, Rep. Waxman pointed out that it is likely to be superseded by a new combination drug recently approved by the FDA. Harvoni (ledipasvir and sofosbuvir), made by the manufacturer of Sovaldi—Gilead Sciences—will cost around $94,000 for a full course of treatment.
“These are great drugs, offering a complete cure for people with the most common form of hepatitis C,” he said. “However, when you look at two to three million Americans with this form of hepatitis C, insurers, patients, and Medicaid programs are wondering where they will get the money necessary to pay for the drugs. If all the people currently eligible for the treatment were to get it, it could cost the US healthcare system well over $100 billion just for this one disease alone, and clearly this is not sustainable.”
The High Cost of Not Adequately Funding the NIH
Although Rep. Waxman lauds current efforts in Congress to help facilitate the discovery, development, and marketing of new medical therapies as part of the 21st Century Cures Initiative, he emphasized that the most important and consistent recommendation emerging from every one of the hearings related to this issue is that Congress increase its funding of the NIH.
“Everybody agrees with that,” he said, citing NIH Director Francis Collins’s observation that the agency is able to fund one in six research proposals, versus one in three in the past. “We shouldn’t have been proud of one in three. But we should be ashamed that we’re going backward, and it’s now one in six.