Funding Grants for Rare Diseases at the Food and Drug Administration

2012 ◽  
pp. 1-4
Author(s):  
Soumya Patel ◽  
Katherine I Miller Needleman
Keyword(s):  
Drug Administration ◽  
Rare Diseases ◽  
Food And Drug Administration

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.

PLAN TO SPEED APPROVAL OF DRUGS: MAKERS WOULD PAY FEES TO U.S

PEDIATRICS ◽  
1993 ◽  
Vol 92 (4) ◽  
pp. 558-558
Author(s):  
J. F. L.
Keyword(s):  
Drug Administration ◽  
Rare Diseases ◽  
Food And Drug Administration ◽  
Drug Industry ◽  
Staff Members ◽  
Life Saving ◽  
Congressional Staff ◽  
The Government ◽  
Biotechnology Products

WASHINGTON, Aug. 10—The Government and the drug industry are near an agreement to require the companies to pay fees that would be used by the Food and Drug Administration to hire 600 new examiners and speed approval of drugs, officials on both sides said today. The agreement, being worked out by Congressional staff members, is intended to resolve what both the Government and industry have said for the last few years is a catastrophe waiting to happen: the number of drug applications is rapidly increasing, especially because of the boom in biotechnology products, some of which might save lives if they were available. But the F.D.A. has not had enough staff members to keep up. Advocates of people with AIDS, cancer and rare diseases have all pressed for quicker approval of potentially life-saving drugs, for time can be crucial in treatment.

INSIDE INDUSTRY

2015 ◽  
Vol 19 (06) ◽  
pp. 35-39
Keyword(s):  
Bladder Cancer ◽  
Drug Administration ◽  
Rare Diseases ◽  
Oncolytic Virus ◽  
Food And Drug Administration ◽  
Phase Iii ◽  
Cancer Drugs ◽  
Research Risks ◽  
Lost Decade

Two Promising Phase III Cancer Drugs: SIRT & Afatinib CAVATAK™ Presentations at Oncolytic Virus Therapeutics Conference Reinforce Potential in Bladder Cancer and Melanoma Receptos subjected to M&A Speculation Antibiotics Research Risks another Lost Decade while Rare Diseases Research Steams Ahead, Global Patent Study Reveals Intertek gains Official Accreditation with Philippines Food and Drug Administration

scholarly journals An introspection of quality of novel drug approvals by United States Food and Drug Administration

2018 ◽  
Vol 8 (1) ◽  
pp. 84
Author(s):  
Rekha Mehani ◽  
Ajay K. Shukla
Keyword(s):  
United States ◽  
Drug Administration ◽  
Rare Diseases ◽  
Food And Drug Administration ◽  
Medical Needs ◽  
Novel Drugs ◽  
United States Food ◽  
States Food ◽  
Drug Approvals ◽  
Novel Drug

Background: United States Food and Drug Administration (FDA) is the fastest drug review agency in the world. FDA is responsible for protection of the public health by assuring that foods are safe, wholesome, sanitary and properly labelled. Approved Novel drugs are often innovative products that serve unmet medical needs or otherwise help to advance patient care.Methods: FDA novel drug approvals were analysed from calendar year (CY) 2012 to 2016 on the basis of three criteria i.e., impact, access and predictability. Impact measured on the basis of: percentage of novel drug approvals (a) first in class (b) for rare diseases. Access measured on the basis of: percentage of novel drug approvals (a) first cycle approval (b) approval in the U.S. before other countries and (c) percentage of priority reviews. Predictability measured by: the percentage of novel drug approvals that met the PDUFA goal dates for the application review.Results: Total number of novel drugs approved from CY 2012 to 2016 was 176 (average 35 novel drugs/ year). Impact of novel drug approvals: 40% were first in class and 39% were for rare diseases. Access of novel drug approvals: 84% were first cycle approval, 60% were approval in US before other countries, 51% priority reviews among novel drug approvals. Predictability of novel drug approvals: 97% approvals able to meet PDUFA goal dates for application review.Conclusions: Novel drug approvals during CY 2012-2016 had a high quality which is very much evident by their high impact, good access and high predictability.

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.

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