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Recursos em Doenças Raras para Pessoas Refugiadas/DeslocadasOrphan drugs in the United States of America
- Public policy on orphan drugs in the USA
- Orphan drugs labelling and legal status
- Incentives to orphan drugs providers in term of R&D, intellectual property and marketing
As early as 1983, the Public Health authorities realised that legislation on orphan drugs was needed signing of the 'Orphan Drug Act'.
This law defines the 'orphan drug' with regard to prevalence (frequency) of the disease for which it is indicated in the American population.
In the US, the concept of 'orphan drug' does not simply cover pharmaceutical or biological products. It also covers medical devices and dietary or diet products. The OOPD (Office of Orphan Products Development) was created within the FDA (Food and Drug Administration). It is in charge of promoting the availability of safe and efficacious products for the treatment of rare diseases. The 'orphan' status allows the drug sponsor to benefit from incentives for the development of these products until the marketing approval.
These measures apply to all stages of the drug development :
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- research: tax credits on clinical research ; ' '
- elaboration of an application file necessary for marketing approval: technical assistance during the elaboration of the file as well as simplification of administrative procedures (reduction of the waiting period and reduction of the amount of registration fees) ; '
- marketing: exclusivity of 7 years after the marketing approval is granted.
For further information, you can go and see the list of the American orphan drugs.
Public policy on orphan drugs in the USA
The legal status of orphan drugs in the USA dates back to 4 January 1983 with the passing of a law called the Orphan Drug Act :
Orphan drugs are used in diseases or circumstances which occur so infrequently in the USA, that there is no reasonable expectation that the cost of developing and making available in the USA a drug for such disease or condition will be recovered from sales in the USA for such drugs.
Several amendments were subsequently passed, aiming at defining the criteria orphan drugs must meet.
- A 1984 amendment defined the concepts of low incidence
A rare disease or circumstance by definition :-
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- Affects less than 200 000 individuals in the USA ; '
- Affects more than 200 000 individuals in the USA without it being possible to cover the cost of development and distribution by sales on national territory.
Limit of prevalence for a rare condition : 7,5 / 10 000
- The 1985 and 1990 amendments
The definition of orphan product was extended to products other than drugs and in particular : biologics, medical devices and medical foods, mainly parenteral nutrition and nutraceuticals. - The 1988 amendment
The product must be part of the basis of a Marketing Authorisation submitted before application for orphan drugs status. The product must not have been previously approved following a New Drug Application or a Product License Application for the disease or the condition for which the applicant requests orphan drug status.
Orphan drug status : for a disease or a condition - The 1992 amendment
If the drug is theoretically similar to an orphan drug authorised for the same rare disease, the applicant must demonstrate the clinical superiority of this drug, which is then considered in the same way as a new active ingredient. The efficacy of the drug must be established with regard to prevention, diagnosis, or treatment of this disease.
' - More than one sponsor can receive designation for the same drug for the same use; the seven year marketing exclusivity is given to the first sponsor to file a complete NDA. Competitors are not prevented from making the drug available for different uses during the seven year period of exclusivity. Six and a half million patients are eligible for treatment by orphan drugs in the USA. '
Orphan drugs labelling and legal status
Orphan drugs are covered by a specific individual legal system endowing them with a special status. Granting orphan drug status is based upon an application dossier submitted to the Office of Orphan Products Developpement (OOPD), including :
- Standard administrative data (name and address of the sponsor, name and address of the manufacturer, international non-proprietary name of the drug, trade name of the drug).
- A description of the disease for which use of the drug is claimed and the intended conditions of use.
- The authorisation given by the sponsor to the FDA to publish information concerning the product and the recognised indication.
- The size and other chief characteristics of the population likely to be treated in the USA: all available information, published or otherwise available, must be supplied.
- A description of the drug and its risk/benefit ratio. A summary of the principal preclinical and clinical data concerning use of the product for the indication concerned as well as basic documentation.
- The estimate of the cost of development and distribution of the drug as well as an assessment of potential sales in the USA, confirming the absence of commercial viability of marketing the drug in specific cases. The FDA is required to provide an answer to the sponsor within a maximum of 60 days after receiving the application. When the drug is designated as 'orphan', the FDA publishes this information in the Federal Register. Orphan designation and marketing authorisation application are two necessary stages before an orphan drug can be marketed. Each decision is taken by a special entity at the FDA.
| Orphan drug status | FDA Office of Orphan Products Development. |
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| Marketing Authorisation |
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Incentives to orphan drugs providers in terms of R&D, intellectual property and marketing
Granting orphan drugs status may enable the sponsor to obtain the following advantages for the development of the product :
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- a 50% tax credit on the cost of clinical trials undertaken in the USA ; ' '
- a seven year period of marketing exclusivity following the marketing approval ; ' '
- some written recommendations provided by the FDA concerning clinical and preclinical studies to be completed in order to register the new drug ; ' '
- a fast-track procedure for the FDA to evaluate registration files ; '
- The availability of orphan drugs to patients before being granted a marketing approval is possible. In some cases of compassionate use, a Treatment Investigational New Drug (t-IND) may be obtained under specific conditions :
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- The drug must be intended for the treatment of a serious or life-threatening disease ; ' '
- No alternative drug or treatment must be available ; '
- The product must be in the process of clinical trials and in an active phase of marketing approval.
For this latter reason, t-INDs are granted for a limited period of time.
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