The Orphan Drug Act has been a tremendous success and addressed a market failure by providing incentives to bring new treatments to those suffering with rare diseases.
That’s why I was deeply troubled by Friday’s unfair article in The Washington Post targeting the Orphan Drug Act through selective and unsubstantiated attacks that seem designed to undermine this bipartisan and hugely successful regime that has helped so many patients with rare diseases live longer and healthier lives.
While each rare disease only affects a small number of patients, the approximately 7,000 known rare diseases collectively impact over 30 million Americans and about half of all rare diseases affect children.
Since the passage of the law, more than 400 new orphan drugs have been approved by the FDA—compared to just 34 approvals before enactment.
Yet even with this success, 95 percent of rare diseases currently have no treatment, thus maintaining the incentives of the Orphan Drug Act remains as critical as ever.
BIO—in conjunction with the National Organization for Rare Disorders (NORD) and Ernst & Young—recently published a study that showed the tremendous real-world impact this law and these drugs have had on patients suffering from rare and debilitating diseases for which there were no good medical options.
According to the study, there would have been one-third fewer orphan drugs on the market treating patients with rare diseases over the last 30 years without the incentives found in the Orphan Drug Act.
That’s people who thought their disease were forgotten.
That’s families with renewed hope.
That’s patients able to live their life to the fullest.
But unfortunately, the article takes none of this into account. And it simply fails to provide appropriate context for the broader claims it makes.
For example, the article speculates that orphan drugs “could drive up insurance costs for everyone.” Yet, it provides absolutely no evidence of this. In fact, it even contradicts itself when it states, “insurance companies often have so few rare-disease patients on their books that even a very high-priced drug gets little attention. And pharmaceutical companies fund assistance programs that ensure patients do not forgo a treatment because of its price.”
The fact is that drug costs are a small fraction of overall healthcare costs for insurance companies, and orphan drugs are a small fraction of overall drug costs.
The article also attempts to take aim at the higher costs associated with developing existing drugs for orphan indications. It fails to mention that the higher costs are necessary to support the studies that are rightly required by the FDA to evidence the drug’s new orphan designation. These rigorous safety protocols ensure the new orphan drug works safely and effectively.
We should be supporting smart policies, like the Orphan Drug Act, that spur innovation and achieve real results for patients whose lives have been transformed by them.
By any measure, the Orphan Drug Act has been a resounding success and given hope to millions of families.
Let’s not harm the system that has made this success possible.