According to a report by Evaluate Pharma, sales of orphan drugs are expected to reach $224 billion by 2024 growing at a 12% CAGR through the year." Growth rates approximately double those of the overall prescription drugs market and the mean cost per patient per year of the top 100 orphan products
in the U.S.
Since its creation in 1983, the Orphan Products
Grants Program has provided more than $400 million to fund more than 600 new clinical studies, said Debra Lewis, O.D., acting director of the FDAs Office of Orphan Products
The FDA's Office of Orphan Products
Development is dedicated to supporting the development of products that are promising for treatment and diagnoses of diseases that affect less than 200,000 Americans annually.
The Rare Diseases Orphan Product
Development Act of 2002 increased the funding for FDA's Orphan Products
Research Grant Program to $25 million, putting more money toward development of therapies for rare diseases.
The grants, which will be issued by the agency's Office of Orphan Products
Development, will go to "consortia whose business model and approach to device development will either result in, or substantially contribute to, market approval of medical devices designed specifically for use in children." However, the program won't support single-device projects, the agency said.
To accelerate the development of treatments for rare and neglected diseases that will complement and advance the objectives of ODA and facilitate the availability of the next generation of orphan products
for children, he recommended:
Both regulatory agencies require the submission of an annual report for orphan products
For the past 15 years, funding for the grants program at FDA's Office of Orphan Products
Development has remained stagnant at $14 million.
Advanced Biotherapy, Inc., Los Angeles, CA, announced the FDA's Office of Orphan Products
Development (OPD) has granted the company Orphan Drug Designation for its investigational antibodies to interferon-gamma to treat immunologic corneal allograft rejection.
Medicines developed and approved under the EU's three-year-old scheme to support orphan products
- many of them based on biotechnology - are still not widely available to patients in need of them, EuropaBio pointed out.
In addition, updated epidemiologic information from experts in the field of emerging diseases; increased disease awareness among health professionals, patients, and the general public; a list of priority vaccines; emergence of a dedicated organization with strong leadership; and the long-term pharmacoeconomic viability of orphan products
will be key factors in overcoming the complexity of orphan status and the limited need for vaccine.
Senators Edward Kennedy (D-Mass.) and Orrin Hatch (R-Utah) claim that manufacturers are abusing the provisions by charging high prices for their orphan products
. They have asked that the FDA impose a moratorium on orphan approvals until June 30, 1989, so that Congress can review the Act and consider amendments.
First, the manufacturers are dealing with products that can be classified as "orphan products
." This means that the potential product market is quite small and may require that the product be sold at a high unit price in order to recover development and production costs.
FDA Office of Orphan Products
for the treatment of glioma, medulloblastoma and ovarian cancer, and in Europe for the treatment of malignant gliomas.
The US FDA Office of Orphan Products
Development grants orphan drug designation to investigational drugs and biologics that are intended for the treatment of rare diseases that affect fewer than 200,000 people in the US, according to the company.