What is the FDA’s Orphan Drug Designation program?
Supporting the development and evaluation of new treatments for rare diseases is a key priority for the FDA. The FDA has authority to grant orphan drug designation to a drug or biological product to prevent, diagnose or treat a rare disease or condition. Orphan drug designation qualifies sponsors for incentives including:
What is an orphan disease?
The conditions are referred to as orphan diseases . The assignment of orphan status to a disease and to drugs developed to treat it is a matter of public policy in many countries and has yielded medical breakthroughs that might not otherwise have been achieved, due to the economics of drug research and development.
What are orphan drugs and how do they work?
In simple words, orphan drugs are those that companies don't expect to make much money from, and instead direct their efforts at drugs which will bring in money. Pharmaceutical (drug) and biotech companies are constantly researching and developing new medications to treat medical conditions, and new drugs come on the market frequently.
What is the Orphan Drug Act of 1983?
The Orphan Drug Act (ODA) of January 1983, passed in the United States, with lobbying from the National Organization for Rare Disorders and many other organizations, is meant to encourage pharmaceutical companies to develop drugs for diseases that have a small market.
What is an orphan drug?
Jump to navigation Jump to search. Regulatory class of pharmaceutical drug. An orphan drug is a pharmaceutical agent developed to treat medical conditions which, because they are so rare, would not be profitable to produce without government assistance. The conditions are referred to as orphan diseases .
What does "orphan" mean in medicine?
In the European Union (EU), the European Medicines Agency (EMA) defines a drug as "orphan" if it is intended for the diagnosis, prevention or treatment of a life-threatening or chronically and seriously debilitating condition affecting not more than 5 in 10,000 EU people. EMA also qualifies a drug as orphan if – without incentives – it would be ...
Why are orphan drugs so lucrative?
According to a 2014 report, the orphan drug market has become increasingly lucrative for a number of reasons. The cost of clinical trials for orphan drugs is substantially lower than for other diseases because trial sizes are naturally much smaller than for more diseases with larger numbers of patients. Small clinical trials and minimal competition place orphan agents at an advantage in regulatory review.
Why is there an increase in orphan drug research?
According to Thomson Reuters in their 2012 publication "The Economic Power of Orphan Drugs", there has been increased investment in orphan drug research and development, partly due to the U.S. Orphan Drug Act of 1983 (ODA) and similar acts in other regions of the world driven by "high-profile philanthropic funding".
Why is it easier to get approval for an orphan drug?
In the U.S. and the EU, it is easier to gain marketing approval for an orphan drug. There may be other financial incentives, such as an extended period of exclusivity, during which the producer has sole rights to market the drug. All are intended to encourage development of drugs which would otherwise lack sufficient profit motive to attract corporate research budgets and personnel.
How much will orphan drugs be sold in 2020?
The report projected a total of US$176 billion by 2020.
What was the most productive period in the history of orphan drug development?
According to Drug Discovery Today, the years 2001 to 2011 were the "most productive period in the history of orphan drug development, in terms of average annual orphan drug designations and orphan drug approvals".
What is considered an orphan drug?
For the purpose of Orphan Drug Designation, what FDA considers the disease or condition to be: Ovarian, Fallopian Tube, and Primary Peritoneal Cancer. FDA considers ovarian cancer, fallopian tube cancer, and primary peritoneal cancer to be one distinct disease or condition. Metastatic Brain Cancer.
What classifications does the FDA recognize for lymphoma?
FDA recognizes the WHO classifications of lymphoma as distinct diseases or conditions. Familial Adenomatous Polyposis. FDA recognizes Familial Adenomatous Polyposis as a distinct disease or condition from sporadic adenomatous polyps. Medication-induced Dyskinesia in Parkinson’s Disease.
Is levodopa a medication induced dyskinesia?
FDA recognizes Medication-induced Dyskinesia in Parkinson’s Disease (PD) as the disease or condition. Levodopa-induced dyskinesia in PD is considered to be a subset of Medication induced Dyskinesia in PD.
What is an orphan drug?
An orphan drug (OD) is a medication used for the diagnosis, prevention, or treatment of a rare disease — a medical condition that isn’t common but can be serious or life-threatening.
Who gives out orphan drug status?
The FDA is an organization with many offices. Each office has different responsibilities — for example, the FDA Center for Devices and Radiological Health has the responsibility of making sure that people have access to high-quality medical devices and radiation-releasing products that are safe and effective.
What are the benefits of being awarded orphan drug status?
There are many benefits to having ODS. In addition to eligibility for grant funding, regular input from the OOPD, and qualification for rapid FDA approval processes, other benefits include:
How expensive are orphan drugs for patients?
ODs can be expensive because manufacturers have control over prices, and individuals are often willing to pay for a treatment option for their rare medical conditions. Some examples of costly ODs include:
What counts as a ‘rare disease’?
These medical conditions are usually serious or life-threatening. Currently, there are an estimated 7,000 rare diseases.
What is the PDUFA exemption?
Prescription Drug User Fee Act (PDUFA) exemption: In 1992, the U.S. Congress created the PDUFA, which allowed the FDA to collect fees from manufacturers. The fees are important in speeding up the medication approval process. However, if the manufacturer obtains ODS, the FDA will waive the NDA or BLA application fee, which can be over $2 million.
How does the FDA grant help manufacturers?
Manufacturers can receive funding through grants. The FDA provides grants through the Orphan Product Grants Program. The OOPD provides funding to help manufacturers conduct safety and effectiveness studies that have a high likelihood of resulting in FDA approval.
What Is an Orphan Drug?
An orphan drug is a medication (pharmaceutical) that remains underdeveloped due to the lack of a company to find the drug profitable. Often the reason that the drug is not profitable is that there are relatively few people who will purchase the drug when weighed against the research and development needed to manufacture the drug. In simple words, orphan drugs are those that companies don't expect to make much money from, and instead direct their efforts at drugs which will bring in money.
Why are some drugs considered orphan drugs?
Why Some Drugs Are "Orphan Drugs". Pharmaceutical (drug) and biotech companies are constantly researching and developing new medications to treat medical conditions, and new drugs come on the market frequently. People who suffer from rare diseases or disorders, on the other hand, do not see the same drug research attention for their illnesses.
What is the 1983 orphan drug act?
The Orphan Drug Act offers incentives to induce companies to develop drugs (and other medical products) for the small markets of individuals with rare disorders (in the U.S., 47 percent of rare disorders affect fewer than 25,000 people). These incentives include:
What is the role of the FDA in orphan drug development?
The U.S. Food and Drug Administration (FDA) is responsible for ensuring the safety and effectiveness of medications on the market in the United States. The FDA established the Office of Orphan Product Development (OOPD) to help with the development of orphan drugs ...
What are some examples of orphan drugs?
Example of Drugs Available Due to the Orphan Drug Act. Among the drugs which have approved include those such as: Tetrabenazine for treating the chorea which occurs in people with Huntington's disease. Enzyme replacement therapy for those with the glycogen storage disorder, Pompe's disease.
How many orphan drugs have been approved by the FDA?
Prior to the passage of the Orphan Drug Act, few orphan drugs were available to treat rare diseases. Since the Act, more than 200 orphan drugs have been approved by the FDA for marketing in the U.S.
Why are orphan drugs not profitable?
Often the reason that the drug is not profitable is that there are relatively few people who will purchase the drug when weighed against the research and development needed to manufacture the drug.
What was the Orphan Drug Act?
The Story Behind the Orphan Drug Act. Scientific developments throughout the 20th century led to the development of many medical products and therapeutic advances for patients. But around the late 1970s it became increasingly clear that many citizens were being left out of these advances.
What is the FDA's role in orphan status?
In addition, FDA was authorized to designate drugs and biologics for orphan status (the first step to getting orphan development incentives) provide grants for clinical testing of orphan products, and offer assistance in how to frame protocols for investigations.
What are the incentives for orphan drug development?
That law, the Orphan Drug Act, provided financial incentives to attract industry’s interest through a seven-year period of market exclusivity for a drug approved to treat an orphan disease, even if it were not under patent, and tax credits of up to 50 percent for research and development expenses. In addition, FDA was authorized to designate drugs and biologics for orphan status (the first step to getting orphan development incentives) provide grants for clinical testing of orphan products, and offer assistance in how to frame protocols for investigations. A subsequent amendment defined a rare disease as one affecting under 200,000, though a disease with more patients could qualify if the firm could not recover the costs of developing the drug.
How many orphan drugs were approved in 1990?
The 1983 Orphan Drug Act completely changed the face of therapeutics for rare disorders. By 1990 FDA had designated 370 products for orphan status, and of these 49 were approved for orphan indications.
How many orphans were there in 2002?
By 2002 the number of orphan designations grew to almost 1100, and approvals to 232, a number that provided treatment to an estimated 11 million patients. Much work of course remained to be done, considering how many suffered from rare disorders. But the Orphan Drug Act finally provided for many of those orphaned among blockbuster treatments ...
Overview
Effect on investment, sales and profit
According to Thomson Reuters in their 2012 publication "The Economic Power of Orphan Drugs", there has been increased investment in orphan drug research and development, partly due to the U.S. Orphan Drug Act of 1983 (ODA) and similar acts in other regions of the world driven by "high-profile philanthropic funding".
According to Drug Discovery Today, the years 2001 to 2011 were the "most productive period in t…
Definition
According to the US Food and Drug Administration (FDA), an orphan drug is defined as one "intended for the treatment, prevention or diagnosis of a rare disease or condition, which is one that affects less than 200,000 persons in the US" (which equates to approximately 6 cases per 10,000 population) "or meets cost recovery provisions of the act." In the European Union (EU), the European Medicines Agency (EMA) defines a drug as "orphan" if it is intended for the diagnosis, …
Global statistics
As of 2014 , there were 281 marketed orphan drugs and more than 400 orphan-designated drugs in clinical trials. More than 60% of orphan drugs were biologics. The U.S. dominated development of orphan drugs, with more than 300 in clinical trials, followed by Europe. Cancer treatment was the indication in more than 30% of orphan drug trials.
• Number of orphan drugs in clinical trials: 600
Legislation
Orphan drugs generally follow the same regulatory development path as any other pharmaceutical product, in which testing focuses on pharmacokinetics and pharmacodynamics, dosing, stability, safety and efficacy. However, some statistical burdens are lessened to maintain development momentum. For example, orphan drug regulations generally acknowledge the fact that it may not be possible to test 1,000 patients in a phase III clinical trial if fewer than that number are affecte…
Examples for selected diseases
In the 1980s, people with cystic fibrosis rarely lived beyond their early teens. Drugs like Pulmozyme and tobramycin, both developed with aid from the ODA, revolutionized treatment for cystic fibrosis patients by significantly improving their quality of life and extending their life expectancies. Now, cystic fibrosis patients often survive into their thirties and some into their fifties.
The 1985 Nobel Prize for medicine went to two researchers for their work related to familial hype…
Activism, research centers
The Center for Orphan Drug Research at the University of Minnesota College of Pharmacy helps small companies with insufficient in-house expertise and resources in drug synthesis, formulation, pharmacometrics, and bio-analysis. The Keck Graduate Institute Center for Rare Disease Therapies (CRDT) in Claremont, California, supports projects to revive potential orphan drugs whose development has stalled by identifying barriers to commercialization, such as problems with for…
Cost
According to a 2015 report published by EvaluatePharma, the economics of orphan drugs mirrors the economics of the pharmaceutical market as a whole but has a few very large differences. The market for orphan drugs is by definition very small, but while the customer base is drastically smaller the cost of research and development is very much the same as for non orphan drugs. This, the producers have claimed, causes them to charge extremely high amounts for treatment…
Orphan Drug Designation List
- The Orphan Drug Designation List was updated and developed using the methodology referenced in About the Orphan Drug List. It should be used to govern the quarter July 1 to September 30, 2022. Orphan Drug Designation List Governing July 1 to September 20, 2022 (XLSX - 452 KB)
About The Orphan Drug List
- HRSA develops this list and updates it quarterly. It is based upon specific fields captured from the list of orphan drug designations provided by the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development (OOPD). The list posted should be the source used by 340B stakeholders to ensure compliance with the orphan drug exclusion...
Program Integrity
- 340B Drug Pricing Program covered entities must ensure program integrity and maintain accurate records documenting compliance with all 340B Program requirements. Covered entities are subject to audit by manufacturers or the federal government. Failure to comply may make the 340B covered entity liable to manufacturers for refunds of discounts obtained. Learn more: Prog…