About the report
Significant and transformative innovations for patients with rare disease have become available in 2019, highlighting the commitment made by manufacturers and regulators to these patients. The significant increases in the numbers of orphan approvals has raised orphan share of invoice spending from 2% in 1992 to 11% in 2019. However, rare disease patients continue to face hurdles in receiving treatment despite the increase in approvals and use of orphan medicines. In addition, access to rare disease medicines have been confounded by the COVID-19 pandemic, as the challenges rare disease patients face in starting new treatments have been exacerbated by widespread health system disruptions. This report discusses the current innovation in rare disease treatments, trends in overall drug spending levels and growth, and the effect of COVID-19 on patients.
Report Summary
In 2019, there were significant advances in treatments for rare diseases, underscoring the commitment to patients by manufacturers and regulators. The report finds that orphan indications have now reached 838 in total since the passage of the Orphan Drug Act and have been granted to 564 distinct drugs, with transformative innovations including cutting-edge gene and nucleotide therapies. In addition to novel treatments, continued investment and commitment by manufacturers to pursue multiple orphan indications has led to increases in the number of patients eligible for treatment. For example, as of 2019, 90% of cystic fibrosis patients are eligible for targeted treatment, compared to only 4% in 2012.
In addition to innovation, the report also examines overall levels of orphan medicine spending and cost and finds that invoice spending on orphan indications accounted for 11% of the United States’ $518 billion in invoice spending in 2019. Another significant finding is that approximately 39% of drugs with orphan indications cost more than $100,000 annually, but the average annual cost of an orphan treatment was well below, at $32,000. The ongoing COVID-19 pandemic has negatively affected new orphan prescription starts, which are cumulatively down 21% since the beginning of the pandemic. This means that to-date, almost 48,000 new therapy starts have not taken place, a substantial impact to rare disease patients.