A report, commissioned by the National Organization for Rare Disorders and released by IQVIA, has revealed that the majority of orphan drugs, 79%, treat only rare diseases. Despite this fact, orphan conditions make up just 11% of drug spending in the United States. Fortunately, the report also revealed that progress made in the past few years has increased access to treatments for rare disease patients.
About the Report
The study covered a lot of information, focusing on multiple aspects of rare disease drug development and care. It utilized data from 2019, finding that 838 rare diseases saw 564 orphan treatments approved by the FDA, giving many patients access to necessary therapies.
In fact, this statistic represents a larger trend; we are seeing more drugs that hold more than one use. Autoimmune conditions and various forms of cancer have seen benefits from this trend, as many treatments have been indicated for them. Research into biological pathways has allowed a better understanding, which in turn results in various medications able to treat multiple conditions, both rare and common.
Another point made in the report focuses on generic drugs, biosimilars, and the benefits they provide. Because there are multiple versions of one treatment, rare disease patients see increased access to the therapies they need.
Issues Addressed in the Report
The pricing of rare disease treatments has been an issue for a long time; treatments for rare diseases like spinal muscular atrophy can cost millions of dollars. The fact that treatments for orphan conditions make up just 11% of drug spending in this country is an issue; many rare disease patients face a severe unmet medical need.
Additionally, the report found that specialty drug spending accounts for nearly 50% of medical invoice spending and has increased by 22 percentage points. This can be compared to orphan drug spending, which has only increased by 5 percentage points. The majority of specialty drug funding, 77%, goes towards treatments of common conditions. This means rare disease treatments are facing less growth and spending.
These issues must be addressed in order to help rare disease patients. To read more about the subject, read the source article.