- Center on Health Equity and Access
- Clinical
- Health Care Cost
- Health Care Delivery
- Insurance
- Policy
- Technology
- Value-Based Care
Orphan Drug Market to Continue Steady Growth; Sales to Double Between 2016 and 2022
The orphan drug market continues its steady and inexorable growth even with increased scrutiny on the high prices of these lifesaving treatments.
The orphan drug market continues its steady and inexorable growth even with increased scrutiny on the high prices of these lifesaving treatments.
According to EvaluatePharma’s Orphan Drug Report 2017, insurers will continue to cover orphan drugs, which will drive continued growth of the market. Sales of orphan drugs are expected to double between 2016 and 2022. By 2022, worldwide orphan drug sales are expected to total $209 billion and orphan drugs are set to be 21.4% of brand-name prescription drugs.
“The image of the plucky small biotech striving to develop treatments for the rare diseases largely ignored by big pharma is long gone,” Lisa Urquhart, EP Vantage editor and report contributor, said in a statement. “Instead, this year we again find big pharma dominating the sector.”
The report estimates that the average cost per patient in 2016 for an orphan drug was $140,443 compared with $27,756 for a non-orphan drug. There is a clear correlation between revenue per patient and the number of patients treated. Soliris, which is approved to treat patients with paroxysmal nocturnal hemoglobinuria, is one of the most expensive drugs in the world and was the highest revenue orphan drug in 2016, but it treats a very small patient population.
EvaluatePharma predicts that Revlimid, approved to treat myelodysplastic syndrome, orphan indications of Non-Hodgkin’s lymphoma, and multiple myeloma, will be the top-selling orphan drug in the world in 2022. Celgene, which makes Revlimid, will have the most orphan drug sales worldwide.
However, Urquhart and report co-author Andreas Hadjivasiliou, an analyst at EvaluatePharma, warn that payers and politicians are taking note of the industry more than ever before.
“As such, if the orphan drug industry is to continue to thrive, it must continue to generate innovations that justify the huge cost of these life-transforming treatments,” they wrote.
NCCN Guidelines Update Adds Momelotinib Below Ruxolitinib for High-, Low-Risk Myelofibrosis
November 21st 2023Momelotinib was given category 2A and 2B status for patients with high- and low-risk myelofibrosis (MF) and MF with anemia. However, ruxolitinib retains a higher category of recommendation as a treatment for patients with MF.
Read More
Oncology Onward: A Conversation With Dr Shereef Elnahal, Under Secretary for Health
April 20th 2023Shereef Elnahal, MD, MBA, under secretary for health at the Veterans Health Administration (VHA), sat for a conversation with our hosts Emeline Aviki, MD, MBA, Memorial Sloan Kettering Cancer Center, and Stephen Schleicher, MD, MBA, Tennessee Oncology, that covered the cancer footprint of the VHA.
Listen
Odevixibat Safe for Alagille Syndrome Based on Hepatic Changes
November 8th 2023Pooled phase 3 data presented at North American Society for Pediatric Gastroenterology, Hepatology & Nutrition 2023 support the benefit-risk profile of the ileal bile acid transport inhibitor in treating the rare liver disease.
Read More
Exploring Payer Coverage Decisions Following FDA Novel Drug Approvals
May 3rd 2022On this episode of Managed Care Cast, Ari D. Panzer, BS, lead author and researcher, then at Tufts Medical Center—now at Duke University—discusses the findings from his team’s investigation into coverage decisions by health plan insurers of the 66 drugs approved by the FDA in 2018.
Listen
