Orphan Drug Act delivers cures for rare disease patients. Now, it’s under attack.

Speculation season is practically a year-round practice in Washington, and the possibilities of the 2018 election are no exception. A prominent one is whether Utah’s Orrin Hatch — the longest-serving Republican in the Senate — will seek an eighth term next year. So far, he’s undecided.

Hatch has been a committee chairman or prime mover of essentially every major government policy at one time or another: Taxes and banking, national security, foreign relations, education, the judiciary. All of it.

And yet, his legacy that touches the most lives may be in healthcare, specifically, the 1983 Orphan Drug Act.

What diseases afflict more Americans every day than cancer, diabetes or even heart disease? Rare diseases.

By definition, a rare disease accounts for no more than 200,000 patients but taken together, those conditions designated by the FDA afflict more than 30 million people in this country — almost one in 10 of all Americans.

And while they afflict smaller populations, some of these conditions have emerged to become household names: Tourette’s syndrome, muscular dystrophy, cystic fibrosis, spina bifida, ALS — as well as cancer patients with multiple myeloma.

This is because even these less common diseases have ready and innovative treatments — mainly thanks to the Orphan Drug Act, which incentivized the development of cures for smaller populations. It may be hard to believe, but just 35 years ago, every patient suffering from essentially every disease mentioned above had no hope and even fewer cures.

Today, we live in a vastly different and better world. Bloomberg’s Joshua Green explains it this way, “The FDA has approved more than 300 orphan drugs, with 1,100 more under development. One of the first developed under the law was AZT, the early AIDS treatment. Two years later, Congress expanded the law to include biological and chemical drugs, which helped spur the biotech industry.”

Seems more a cause for celebration than consternation, right? Not so fast say increasingly loud and influential voices in healthcare, particularly those supporting the application of “value frameworks” — complicated mathematical formulas that seek to determine how much a drug should be priced to treat a specific disease — and how much to spend on you, should you get sick.

This is primarily the brainchild of a privately influential but publicly anonymous think tank called the Institute for Clinical and Economic Review (ICER). It is funded by several millions of dollars from interests that seek to strengthen the healthcare “system,” (like insurance companies and pharmacy benefit managers) rather than human beings in need of complex and often expensive medicines to live.

It is holding what it calls an “orphan drug assessment and pricing summit” in D.C. on Wednesday to get that point across to lawmakers. ICER opposes the Orphan Drug Act because spending billions of dollars developing treatments and cures for hundreds of thousands of people doesn’t make sense to them.

Unfair? This is ICER in its own words: “The opportunity cost of supporting the use of ultra-orphan drugs necessitates that patients with a more common disease, for which a cost-effective treatment is available, are denied treatment.”

Here’s a rebuttal: “From an economic perspective, groups that small do not now justify the kind of research expenditures that companies must make. The bill that I am signing today helps to cure that problem and consequently, we hope, some of the diseases as well … providing incentives for the private sector to develop drugs to treat these rare diseases.”

Those were the words of President Reagan, as he signed the Orphan Drug Act into law. How far we’ve come in 35 years — from hope to rare disease patients to harsh assessment of their purported economic drain on society.

The Orphan Drug Act has many supporters. But it has powerful enemies as well like ICER that are dedicated to the collectivist principle and coldly calculating who is too expensive to treat and who is too costly to save.

Whether he is running or retiring next year, Orrin Hatch should defend his legislation and set the record straight — not least for the millions of patients who have benefitted from this landmark legacy of 41-year (and counting) Senate career.

Jonathan Wilcox is the co-founder and policy director of Patients Rising, a leading national patient advocacy nonprofit. He is a fellow with the University of Southern California’s Unruh Institute of Politics. Wilcox was also a speechwriter for Republican California Gov. Pete Wilson and campaign consultant for Sen. Orrin Hatch (R-Utah).

This piece was originally published at The Hill. Read it here.

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