While no one argues that orphan drugs that treat rare diseases can be expensive, the drugs’ reputation for being budget-busters is not borne out by a data presented in a recent report by Quintiles IMS Institute.
While no one argues that orphan drugs that treat rare diseases can be expensive, the drugs’ reputation for being budget-busters is not borne out by a data presented in a recent report by Quintiles IMS Institute, “Orphan Drugs in the United States: Providing Context for Use and Cost.”
Between 25 million and 30 million Americans suffer from rare diseases—serious, chronic diseases affecting fewer than 200,000 people, which can become progressively disabling and limit life expectancy. There are approximately 7000 rare diseases, and children make up more than half of affected patients. A challenge faced by patients and caregivers in the rare disease community is the lack of treatments available: treatments exist for just 5% of an estimated 7000 rare diseases.
The Orphan Drug Act (ODA) of 1983 greatly advanced efforts to introduce more treatments for rare diseases. The ODA provides tax incentives and research grants for drug makers to develop therapies for these diseases, extends marketing exclusivity to patentable as well as unpatentable drugs, and exempts orphan drugs and biologics from submitting a new drug application (NDA) or paying user fees charged by FDA. The 449 approved orphan therapies for 549 orphan indications has led to small-molecule drugs and biologics to treat rare diseases across numerous therapeutic categories.
Of the 449 approved orphan drugs, 351 have only orphan indications, while 98 have both orphan and non-orphan indications. An example of a drug with both orphan and non-orphan approved indications is the biologic Humira (adalimumab), which was approved in 2002 for rheumatoid arthritis and then received 4 approvals for orphan-designated indications. Use in these indications accounts for only a small fraction of overall spending on Humira: use in rare diseases accounted for just 3.8% of the total US adalimumab use in 2016.
In fact, the report found that, in 2016, spending on orphan drugs and orphan indications represented only 7.9% ($36 billion) of the $450 billion spent on pharmaceuticals. Almost 60% of spending was for non-orphan traditional drugs and one-third was spent on non-orphan specialty drugs.
Additionally, though the number of orphan drugs approved in the last 5 years increased from 315 to 449, the share of spending on orphan drugs increased moderately:
AMCP Posters Tackle Interchangeability and Medicaid, Factors Driving Biosimilar Access
April 24th 2024Two posters from the Academy of Managed Care Pharmacy (AMCP) annual meeting explore how an interchangeable insulin glargine biosimilar plays into Medicaid budgets and the top factors driving access to biosimilars.
Exploring the Biosimilar Horizon: Julie Reed's Predictions for 2024
February 18th 2024On this episode of Not So Different, Julie Reed, executive director of the Biosimilars Forum, returns to discuss her predictions for the biosimilar industry for 2024 and beyond as well as the impact that the Forum's 4 new members will have on the organization's mission.
A New Chapter: How 2023 Will Shape the US Biosimilar Space for 2024 and Beyond
December 31st 2023On this episode of Not So Different, Cencora's Brian Biehn and Corey Ford take a look back at major policy and regulatory advancements in 2023 and how these changes will alter the space going forward.
The 6 Key Policy Factors to Ensure Biosimilar Market Sustainability
April 16th 2024Magnus Bodin, senior director and head of international access and policy at Biogen, presented warning signs for unsustainable biosimilar markets as well as key factors needed to create effective policies and future-proof biosimilar markets globally.
BioRationality: Removing the Misconceptions Surrounding Interchangeability
April 15th 2024Sarfaraz K. Niazi, PhD, outlines the current state of interchangeable biosimilars in the US and policy changes needed to clear up misconceptions surrounding the meaning behind interchangeability designations.
AMCP Posters Tackle Interchangeability and Medicaid, Factors Driving Biosimilar Access
April 24th 2024Two posters from the Academy of Managed Care Pharmacy (AMCP) annual meeting explore how an interchangeable insulin glargine biosimilar plays into Medicaid budgets and the top factors driving access to biosimilars.
Exploring the Biosimilar Horizon: Julie Reed's Predictions for 2024
February 18th 2024On this episode of Not So Different, Julie Reed, executive director of the Biosimilars Forum, returns to discuss her predictions for the biosimilar industry for 2024 and beyond as well as the impact that the Forum's 4 new members will have on the organization's mission.
A New Chapter: How 2023 Will Shape the US Biosimilar Space for 2024 and Beyond
December 31st 2023On this episode of Not So Different, Cencora's Brian Biehn and Corey Ford take a look back at major policy and regulatory advancements in 2023 and how these changes will alter the space going forward.
The 6 Key Policy Factors to Ensure Biosimilar Market Sustainability
April 16th 2024Magnus Bodin, senior director and head of international access and policy at Biogen, presented warning signs for unsustainable biosimilar markets as well as key factors needed to create effective policies and future-proof biosimilar markets globally.
BioRationality: Removing the Misconceptions Surrounding Interchangeability
April 15th 2024Sarfaraz K. Niazi, PhD, outlines the current state of interchangeable biosimilars in the US and policy changes needed to clear up misconceptions surrounding the meaning behind interchangeability designations.
2 Commerce Drive
Cranbury, NJ 08512