A Plea to Save Orphan DrugsUntil you or a family member...


May 30, 1992

A Plea to Save Orphan Drugs

Until you or a family member suffer from an incurable rare

disease, altering the Orphan Drug Act of 1983 may seem like a good idea. For those of us who witness the pain and anguish of these diseases firsthand, changing this law poses terrible and unacceptable risks.

My teen-age sons, Dean and Damien Storm, have cystic fibrosis, a genetic disease that affects mainly their lungs and digestive systems. Ultimately, they learn to breathe on less and less. Cystic fibrosis causes a slow and painful build-up of thick, sticky mucus in the lungs, which eventually suffocates the victim, leading to death.

Only about 50 percent of our cystic fibrosis children and young adults reach their mid-twenties. So far there is no cure.

Over the years the boys have been exposed daily to numerous antibiotics, both orally and intravenously. They also take 30 capsules each for digestion and need chest physical therapy on a daily basis.

Despite their illness, however, they are experiencing life to the fullest. Over the years, both boys have benefited from a chronic health-impaired program through the Upton School, which has given them the opportunity to be taught during their illnesses and not marked absent.

The boys have been in the German exchange program and on the soccer team, although their physical endurance is somewhat limited. Dean and Damien are 11th and 12th-grade honor students at Baltimore City College. Dean is graduating this June and plans to attend Towson State on a full scholarship. His goal is the journalism field.

The costs involved for treating this disease are tremendous. Last year, their prescription drugs were approximately $3,000 each for the year, not including the additional thousands when treated with intravenous drugs for two-week to three-week periods, frequent outpatient visits to the hospital, cases of Ensure Plus to help them gain weight, fat-soluble vitamins, etc.

In spite of their increased lung problems and daily challenges, the boys could not be more hopeful. They are currently involved in a DNase study (DNase is a synthetic enzyme) in the third stage of clinical trials, where the drug has been known to improve lung function 15 to 20 percent. If it's approved by the FDA, all of our cystic fibrosis children could benefit.

The scientific breakthroughs are tremendous. We are within a few years of a cure. All of this is possible through the research efforts that are finally paying off after so many years, so many lost lives.

Congress passed the Orphan Drug Act in 1983 to provide incentives for drug companies to develop drugs for orphan diseases, those afflicting fewer than 200,000 people each. The reasoning was that, since it can cost hundreds of millions of dollars to research and develop a new drug, it just didn't pay to develop drugs with very limited markets.

This was especially true for biotechnology breakthroughs and other products, which are not otherwise eligible for the regular 17-year patent protection available to most new drug discoveries. The most important feature of the law is a seven-year period of elusive marketing rights, which acts like a patent for these medicines.

In the nine years since this law has been in effect, 64 orphan drugs have been developed to treat 74 rare diseases, including hemophilia, leprosy, sleeping sickness and severe combined immunodeficiency disease -- the "bubble boy" disease.

Despite this progress, there are still some 10 million to 20 million Americans who suffer from the 5,000 rare diseases for which there is no effective treatment. Those of us who suffer from these diseases, or have close relatives who do, don't want the wheels of progress to stop before it's our turn. But now, misguided legislative efforts threaten to cripple this provision.

Under a proposed amendment, Senate bill S.2060, orphan drugs would lose their patent-like exclusivity once sales reach an arbitrary "sales cap" of $200 million.

People promoting this change say it will make orphan drugs more affordable. But there is no proof of that at all. Logic suggests that because of the limited market size of orphan disease populations, drug costs will increase because manufacturers will have smaller, more fragmented markets from which to recover their investments after losing exclusivity because of the S.2060 sales cap.

Everyone wants affordable drugs. But if the drugs are never discovered, the question of cost is academic. And the fact is that most drug companies have pledged -- in writing -- to provide orphan drugs free to patients who are uninsured or who cannot otherwise pay for the medicine they need.

The Orphan Drug Act works and should be left alone. If the prevailing amorphous Washington feeling pervades, that "something must be done," then I strongly advise that the Orphan Drug Act should be strengthened, so that individuals like my sons with cystic fibrosis can keep their hopes alive for a cure.

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