Can patients afford orphan drugs?

Can patients afford the high cost of orphan drugs?

Most people have a great deal of sympathy for orphans. They have lost their parents and often must depend upon the kindness of strangers to overcome the significant challenges life throws at them.

When it comes to orphan drugs, however, the pharmaceutical industry has discovered that this classification can represent huge profits. It wasn’t supposed to be that way.

In 1983, Congress passed and President Ronald Reagan signed the Orphan Drug Act. This legislation was intended to encourage the pharmaceutical industry to research and develop medicines for rare conditions. It was assumed that such drugs would not be profitable because so few patients would take them.

The Food and Drug Administration was very cooperative in advancing this program. It referred to these medications as “significant drugs of limited commercial value.”

To incentivize drug companies to invest in this kind of research, Congress offered tax breaks. Market exclusivity and patent extension were added benefits. In those days, neither the FDA nor Congress imagined that pharmaceutical companies would charge tens of thousands of dollars for orphan drugs.

Fast-forward to today’s marketplace. The humanitarian Orphan Drug Act that was supposed to create affordable medicines for rare conditions has become a windfall for the pharmaceutical industry. It has challenged many patients and their families who may not be able to afford the medicines they so desperately need.

Some of the most expensive drugs in the pharmacy were developed under the orphan drug umbrella. One example is interferon gamma-1B (Actimmune). Injections of this drug treat a rare immunodeficiency disease called chronic granulomatous disease. A genetic defect causes people with this condition to develop frequent fungal and bacterial infections. The drug also is used to treat severe malignant osteopetrosis. A month’s supply of Actimmune could cost as much as $55,000. Over a year, that could add up to $600,000 or more.

Another orphan drug is a C1 esterase inhibitor called Cinryze. It is prescribed for a rare condition called hereditary angioedema. Without warning, the face may swell. The lips and tongue also can balloon and block breathing. The lifesaving drug that prevents the attack can cost more than $46,000 a month.

Cancer drugs also are notoriously expensive. The multiple myeloma drug lenalidomide (Revlimid) is a lifesaving medication. A month’s supply can run more than $14,000. Even people with insurance may find that their copay and deductible payments are out of reach.

Other very expensive cancer treatments include drugs like pembrolizumab (Keytruda), nivolumab (Opdivo) and ipilimumab (Yervoy). A new generation of one-time cancer treatment called CAR-T is projected to cost anywhere from $373,000 to $475,000. That doesn’t include the cost of hospitalization. An article in The Wall Street Journal (April 28, 2018) points out that the entire treatment could add up to $1 million.

It’s hard to imagine how any individual could afford such treatment. Even insurance companies are likely to find the payments busting their budgets. Premiums will rise. That will impact everyone.

Perhaps it is time for Congress to revisit the Orphan Drug Act. What was intended as an act of kindness has become a pathway for the pharmaceutical industry to charge exorbitant prices.

Joe Graedon is a pharmacologist. Teresa Graedon holds a doctorate in medical anthropology and is a nutrition expert. In their column, the Graedons answer letters from readers. Write to them in care of King Features, 628 Virginia Drive, Orlando, FL 32803, or email them via their website:

2018 King Features Syndicate

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