RE:RE:RE:RE:RE:RE:RE:RE:RE:ONCY Presented Positive GOBLET Pancreatic Cancer Data @ ESMOFrom orphanet web site:
Orphan 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 :
• 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.
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The Accelerated Approval (AA) regulations were promulgated by FDA in 1992 to drive the development of new treatments for serious and life-threatening disorders, primarily motivated by the AIDS crisis and the slow pace of treatment development for HIV infection [3]. In HIV, monitoring death rate or complex endpoints such as hospitalization for opportunistic infections was difficult or unethical. Consequently, AA regulations allowed for drug approval based on the use of surrogate endpoints "reasonably likely to predict clinical benefit" [3] - a surrogate endpoint being a measure, such as a blood test or urine marker, believed to be indicative of a disease state and treatment effect, but not demonstrative of a direct health gain to the patient.
AA has been enormously successful at driving innovation in the development of cancer and HIV therapies during the first 16 years: 26 new chemical entity (NCE) cancer drugs have been approved, using either tumor load or progression-free survival as surrogate endpoints, and 29 HIV drugs (25 NCEs and 4 combination drugs) have gained approval using either CD4 count or viral load as endpoints [4]. In the case of HIV, using survival as an endpoint would have made drug trials too time-consuming due to the lengthy disease course, too costly due to the high number of required patients, and unethical due to the use of placebo in a lethal condition. Even more critical for the development of HIV treatment was the ability to test the complex combinations of drugs responsible for the substantial improvement in long-term outcome. Drug combination studies would have been impossible using a clinical endpoint given large patient numbers and extended study lengths, despite the fact that drug combinations are now essential to the HIV treatment process. Current HIV therapies are a prime example whereby great scientific ideas would nonetheless have led to little or no treatment success without AA access. These successes show that the AA regulatory pathway is having a profound impact on innovation in treatment development.
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What is a clinical trial ?
A clinical trial is a medical study conducted to test the effects of a new or already existing drug, of a biological treatment or of a medical device that might treat or curb a disease already identified. The main goal of a clinical trial is to compare two or several groups of subjects, by using two or several treatments in order to determine the efficacy of a drug or of a biological treatment.
Clinical trials are carefully and ethically conducted in order to protect patients against unwanted adverse reactions and to allow collection and accurate analysis of the information concerning the disease.
Phase II :
These studies last from some months to 2 years and are performed on a small homogeneous group of patients (10 to 40 patients).
They aim at studying the efficacy of the product and determining the smallest efficacious dosing level and the best posology for phase III.
Only one-third of experimental drugs successfully complete both phase I and phase II studies.