Many rare diseases lack effective treatments, despite the over 500 orphan drug therapies that have been approved in the US over the past couple decades. Between 25 million and 30 million people in the US are affected by 7,000 rare diseases. Thanks to an increased focus on these diseases on the part of scientists, researchers and the US FDA, there has been a large increase in new therapeutic options, especially in 2017 and 2018, with the FDA approving 80 new orphan indications in 2017 and 57 in the first 8 months of 2018. These approvals mark the highest annual numbers of approved orphan indications since the Orphan Drug Act was established in 1983.

Advances in genetics, biomarker discovery and next generation therapeutic approaches to treatment have fast-tracked precision medicine and, therefore, the number of potentially life saving orphan drugs. Since August, 503 orphan drugs have been approved in the US, with 78% having solely orphan indications and 22% having both orphan and non-orphan indications.

Total 2017 drug spending in the US was $451 billion, with 56% of the total for non-orphan traditional drugs, 35% for non-orphan specialty drugs and 10% for orphan indications of approved orphan drugs. While these drugs account for a small portion of the overall budget for medicine, orphan indication spending has slightly increased as a share of overall spending within the US pharmaceutical industry.

Source: IQVIA