On October 1, the first-ever Rare Cancer Day, an awareness day highlighting the challenges people living with rare cancers face and emphasizing the importance of early diagnosis, was marked by thousands nationwide. Cancer is not a singular disease but, rather, many diseases, of which several are rare. One in 5 people living with cancer in the U.S. have been diagnosed with a rare cancer, and, for those individuals, the 5-year survival rate is lower than that of individuals diagnosed with more common cancers. It is clear that more needs to be done.

Some have asserted that pharmaceutical companies have shifted their focus to rare cancers and other rare diseases solely in an effort to command higher prices and take advantage of a regulatory regime that sets out an “easier” path to market. This view fundamentally mischaracterizes the law. The path to market is not easier; it is expedited only in cases where patients have a serious illness and there are no other alternatives.

Over 90% of the known 7,000 rare diseases still do not have an FDA-approved therapy. That means most rare disease patients must go without treatment or rely on trying a medicine originally designed for a different use in hopes it could work for them. Congress recognized the need for patients with diseases or conditions without FDA-approved treatments available to have access to medicines at the earliest scientifically feasible time and passed important legislation requiring FDA to meet this goal. The National Organization for Rare Disorders and Friends of Cancer Research have supported legislative efforts to ensure that our patients have access to therapies that meet the same rigorous FDA standards as therapies for serious non-rare conditions.

The resulting laws have created regulatory flexibility at FDA while maintaining the Agency’s gold standard for safety and efficacy. Innovative treatments are being expedited because of a variety of review and approval pathways that allow FDA to consult with companies earlier and more often, resulting in a more efficient drug development process. To the extent there has been an increased effort by the pharmaceutical industry to focus on rare cancers and other conditions, it is a reflection of the advancing state of science that has allowed for a better understanding of the cause and progression of disease.

As part of this advancement in science, there have also been efforts to refine uses of drugs that are already on the market. Contrary to what some have claimed, this is a positive development and should not be viewed as easier or less important. Adding an indication to a drug already on the market still involves conducting clinical trials to demonstrate the safety and effectiveness of that particular use. We believe it is critical that incentives for the pharmaceutical industry to conduct these studies necessary to obtain FDA approval for new uses of older drugs remain in place. Having an FDA-approved use of a drug to treat a condition can help ensure access and patient safety.

Further, the increasing focus on smaller populations reflects an effort to advance “precision medicine.” The intention of precision medicine is to find the most effective, safe, and durable treatments and cures for a particular person, which inherently lends itself initially to smaller groups of patients. If this endeavor succeeds, it will ultimately benefit all of society, not just patients with rare disease, because the logic and science underlying these therapies can be applied to other common diseases. More personalized treatments by definition means better outcomes for patients.

For those with rare cancers and other rare diseases without FDA approved treatments, the increasing number of orphan therapies creates hope where previously there was none. As a society, we need to support these vital efforts, every day of the year.

Peter L. Saltonstall
President & CEO,
National Organization for Rare Disorders (NORD)

Jeff Allen
President & CEO,
Friends of Cancer Research