Rare Diseases

Orphan drugs are not driving health care spending so let’s invest in them

Orphan drugs are not driving health care spending so let's invest in them
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As the debate over health care costs and drug prices intensifies, one of the issues that has been raised relates to the cost of orphan drugs — drugs for patients with rare diseases. Such drugs are often identified by critics as being more costly than drugs for more common diseases, despite the counterpoint that the volume of such sales is, by definition, low.

Thus, we often hear that orphan drugs are significant contributors to rising health-care costs in the U.S., out of proportion to their contributions to health care.

The National Organization for Rare Disorders (NORD), which I head, decided earlier this year to see if the critics are right. We commissioned the Quintiles/IMS Institute to study whether orphan drugs really are a significant driver of health care costs.

The results, a report entitled “Orphan Drugs in the United States: Providing Context for Use and Cost,” is the first study to quantify the cost of orphan drugs within the much larger health care system.

The inescapable conclusion of the study is that orphan drugs are not a major factor in health-care spending. Of total drug sales of $450 billion in the U.S. in 2016, only 7.9 percent was for orphan drugs.

The reason that this study is especially important to the rare disease patient community is that the false assertions about the high cost of orphan drugs to the health care system has led some to call for major undermining of the Orphan Drug Act (ODA).

This is the law…