There was a time not long ago when patients suffering from rare diseases had little hope of finding a medicine that could cure their ills. But in the 30 years since Congress passed the Orphan Drug Act, research into “orphan drugs” — defined as those treating diseases that affect fewer than 200,000 people — has become increasingly common.
All told, orphan diseases afflict about 25 million people in the United States. But before 1983, there were just 38 orphan drugs approved for use in the United States.
Since then, however, 420 such medicines have been approved to treat more than 2,700 diseases. These drugs make up an increasingly large share of what’s becoming available to patients. Last year, a third of new drug approvals were for orphan designations.
And there are many more on the way. A new report by the Analysis Group, an independent consulting firm, found that there are more than 5,400 new drugs in various stages of development and about 8,000 total projects underway, as some drugs may end up targeting multiple diseases. At the end of 2011, 1,795 of those projects were aimed at orphan conditions.
The current level of drug research and development offers hope for a wealth of new treatments that can mend and extend the lives of millions of people. But actually putting those treatments into the hands of patients and doctors won’t be easy. And Obamacare’s many taxes and fees will make innovation even harder.
New orphan drugs are just one of the reasons that individuals with hard-to-treat diseases have reason to hope. The Analysis Group also reports that about 78 percent of the projects in the pipeline are aimed at developing “first-in-class” medicines — drugs that attack a disease in a way that’s unique compared to any existing medicine.
Many of those potential first-in-class treatments target some of today’s most prevalent and deadly diseases. Eighty percent of cancer treatments in the pipeline are potentially “first-in –class,” as are 79 percent of diabetes drugs and 84 percent of neurology medicines.
Other types of novel treatments are also heavily represented in the drug pipeline. The report counted 155 personalized medicine trials — which target patients by using their unique genetic biomarkers — started by January 2009.
Those are already beginning to pay off. Two different personalized medicines were approved for use last year, including a new treatment for late-stage breast cancer.
At least 400 different projects are currently in the works for diseases that haven’t seen a new treatment in more than a decade, like lupus, which currently affects 1.5 million people.
Ovarian cancer, which hits about 22,000 women a year and kills about 15,500 annually, is another disease that is getting a lot of attention. There hasn’t been a new therapy for ovarian cancer since 1996. But at the end of 2011, there were more than 120 potential new treatments, including 16 in the final stage of clinical trials.
But innovation like this takes time, patience, and money.