Evaluation of Orphan Products by the U.S. Food and Drug Administration

1992 ◽  
Vol 8 (4) ◽  
pp. 647-657 ◽  
Author(s):  
Marlene E. Haffner ◽  
John V. Kelsey
Keyword(s):  
Drug Administration ◽  
Rare Diseases ◽  
Food And Drug Administration ◽  
Orphan Drugs ◽  
Marketing Approval ◽  
Drug Products ◽  
Governmental Agencies ◽  
Number Of Patients ◽  
Life Threatening ◽  
The U.S

AbstractOrphan drug products generally are used in treating or preventing rare diseases. The small number of patients available for study may create special problems in the evaluation of these products. This paper examines some of the special problems that are associated with the design and implementation of studies to evaluate the safety and efficacy of orphan drugs. The U.S. Food and Drug Administration (FDA) has not established special criteria for evaluating orphan drugs per se, but the FDA has been flexible in evaluating drug products that present special problems, especially when these products are for treatment of serious of life-threatening illnesses. The FDA and other U.S. governmental agencies also have taken steps to promote the development and availability of drugs for rare diseases, including making these products available to patients who are in need, even before the drugs have full FDA marketing approval.

Oncology Drug Review Process

2003 ◽  
Vol 1 (1) ◽  
pp. 109-113
Author(s):  
Ann T. Farrell ◽  
Richard Pazdur
Keyword(s):  
Drug Administration ◽  
Food And Drug Administration ◽  
Drug Application ◽  
Surrogate Endpoint ◽  
Therapeutic Benefit ◽  
Favorable Effect ◽  
Priority Review ◽  
Marketing Approval ◽  
Life Threatening ◽  
A Priority

The U.S. Food and Drug Administration facilitates the development of drugs intended to treat cancer and other serious or life-threatening diseases. This article describes the new drug application (NDA) process, endpoints used in oncologic trials, and recent initiatives to expedite the review of drugs used to treat serious and life-threatening diseases. The Food and Drug Administration can grant either regular or accelerated marketing approval for oncology drugs. Regular approval is based on endpoints demonstrating that the drug provides a clinical benefit, such as longer life, enhanced quality of life, or a favorable effect on an established surrogate for longer or better life, such as long-term complete responses. Accelerated approval is based on a surrogate endpoint that is less well established but is reasonably likely to predict a longer or better life and can be granted for drugs that are intended to treat a serious or life-threatening illness and that provide meaningful therapeutic benefit to patients over existing therapies. The Agency classifies an NDA as either a priority or a standard application. Drugs that receive priority review are agents that appear to represent significant improvements over existing therapies. A priority NDA is reviewed by the Agency within 6 months; a standard review is accomplished within 10 months. The Agency communicates with sponsors throughout the drug development process, suggesting appropriate trial designs and meaningful endpoints.

Abuse-deterrent Opioid Formulations

2018 ◽  
Vol 128 (5) ◽  
pp. 1015-1026 ◽  
Author(s):  
Ronald S. Litman ◽  
Olivia H. Pagán ◽  
Theodore J. Cicero
Keyword(s):  
Intravenous Injection ◽  
Drug Administration ◽  
Food And Drug Administration ◽  
Opioid Abuse ◽  
Marketing Approval ◽  
Opioid Overdose ◽  
Insufficient Evidence ◽  
Fda Approval ◽  
The U.S

Abstract Abuse-deterrent opioid formulations have been suggested as one way to decrease the abuse, addiction, and overdose of orally prescribed opioids. Ten oral opioid formulations have received abuse-deterrent labeling by the U.S. Food and Drug Administration (FDA). Their properties consist of physical and/or chemical means by which the pills resist manipulation and create a barrier to unintended administration, such as chewing, nasal snorting, smoking, and intravenous injection. In this review, we describe the mechanisms of abuse-deterrent technology, the types of premarketing studies required for FDA approval, the pharmacology of the currently approved abuse-deterrent opioid formulations, and the evidence for and against their influence on opioid abuse. We conclude that there is currently insufficient evidence to indicate that the availability of abuse-deterrent opioid formulations has altered the trajectory of opioid overdose and addiction; however, postmarketing studies are in their infancy, and novel deterrent formulations are continually being developed and submitted for marketing approval.

A Portrait of Nanomedicine and its Bioethical Implications

2012 ◽  
Vol 40 (4) ◽  
pp. 763-779 ◽  
Author(s):  
Rebecca M. Hall ◽  
Tong Sun ◽  
Mauro Ferrari
Keyword(s):  
Anticancer Drug ◽  
Drug Administration ◽  
Food And Drug Administration ◽  
Functional Element ◽  
European Countries ◽  
Scientific Societies ◽  
Governmental Agencies ◽  
Albumin Nanoparticles ◽  
The U.S

While the definitions employed by different governmental agencies and scientific societies differ somewhat, the term “nanotechnology” is generally understood to refer to the manufacturing, characterization, and use of man-made devices with dimensions on the order of 1-100 nanometers (1 nanometer [nm] = 1 billionth of a meter). Devices that comprise a fundamental functional element that is nanotechnological are also frequently comprised within nanotechnology, as are manufactured objects with dimensions less than one micrometer. The differences in definition lead to occasional paradoxes, such as the fact that the most widely used nanodrug (albumin nanoparticles of dimensions up to 300 nm, comprising the anticancer drug paclitaxel) is labeled a “nanopharmaceutical” by governments of European countries, Canada, and Australia, but it is not a nanotechnology for the U.S. Food and Drug Administration (FDA). It is also common in scientific domains to restrict the term “nanotechnology” to objects that possess special, “emerging” properties that only arise because of their nanoscale dimension.

scholarly journals Farmaci orfani e malattie rare: la situazione in Italia

2001 ◽  
Vol 2 (4) ◽  
pp. 261-266
Author(s):  
S. Martina ◽  
C. Denti ◽  
E. Daina ◽  
L. Garattini
Keyword(s):  
Drug Administration ◽  
Rare Diseases ◽  
Food And Drug Administration ◽  
Orphan Drugs ◽  
Ministry Of Health ◽  
Italian Market ◽  
The World ◽  
Economic Profile

Aim of this study is to check the Italian market of the orphan drugs in connection with rare diseases. Data for the research have been found on the Food and Drug Administration Database, the Centro di Ricerche Cliniche per le Malattie Rare “Aldo e Cele Daccò” Database and L’informatore farmaceutico. This study also compared the Italian market with the American market, that counts the greater number of designated orphan drugs and commercial authorizations in the world. In Italy only 24 orphan drugs have been authorized by the Ministry of Health and the present work analyzes their definition under the economic profile, how many diseases and what kind of pathologies they can treat, the number of products on the market and if they are refundable or not.

HIV

2018 ◽  
Author(s):  
Joshua M. Sharfstein
Keyword(s):  
Public Health ◽  
Drug Administration ◽  
Food And Drug Administration ◽  
Research Process ◽  
National Institutes Of Health ◽  
Health Agencies ◽  
Act Up ◽  
The U.S

The emergence of AIDS in the early 1980s caused a profound crisis for federal health agencies, particularly the National Institutes of Health (NIH) and the U.S. Food and Drug Administration (FDA). Activists in ACT UP, charging that these agencies were failing patients with AIDS, initiated a series of escalating protests. NIH officials, led by Dr. Anthony Fauci, began to talk with the advocates and make major changes in the research process. However, over at the FDA, a protest involving the arrest of hundreds of AIDS activists undermined the agency’s public health image. Eventually, under a new commissioner, the FDA earned back the trust of activists.

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