An op-ed by Dean J. Paranicas, HINJ President and Chief Executive Officer
About 1 in 11 Americans, and 350 million people worldwide, suffer from a rare disease, defined as a disease affecting less than 200,000 people in the United States. In many instances, the number of patients afflicted with a particular rare disease total just a few thousand.
There are more than 7,000 known rare diseases, including ALS (Amyotrophic Lateral Sclerosis), Fabry Disease, Scleroderma, SMA (spinal muscular atrophy) and all pediatric cancers. Ninety percent of rare diseases have no FDA-approved treatment. As the National Organization for Rare Disorders (NORD) states, “many rare diseases may result in the premature death of infants or can be fatal in early childhood.”
Around the world, the last day of February every year is recognized as Rare Disease Day, when the rare disease community comes together to raise awareness of rare diseases and the daunting challenges many of these patients and their caregivers face on a daily basis. Nevertheless, their spirit and optimism that biomedical research will find a treatment or cure for their condition remain undiminished.
Biopharmaceutical research, however, is a time-intensive, high-risk and costly endeavor irrespective of the size of the affected patient population. On average, it takes 10-15 years and costs $2.6 billion to bring one new medicine to market. Only one out of every 5,000 compounds researched enters a clinical trial, only 12% of compounds in clinical trials are approved by the FDA, and only 20% of all new medicines will recoup the average R&D cost.
The U.S. research-based biopharmaceutical industry spends more than $100 billion annually on research, with smaller companies and start-ups relying on private investment to support their research, even though these investors know the odds of success are so long. In the smaller rare disease universe, it is even more likely that a new medicine will not recapture its R&D cost before the innovator company loses its patent on the drug and it becomes available as a generic or biosimilar.
So why invest in rare disease research, development and commercialization? Because it is the life sciences industry’s ongoing noble mission to improve human health regardless of the size of the affected patient population. Another benefit is that research can build on itself – a breakthrough on one disease can lead to progress on others.
On this Rare Disease Day, let’s recommit ourselves to encouraging and incentivizing biomedical research and reward the hope of our rare disease patients and community. They deserve no less.