Search Results for Rare Diseases

Showing 1 – 9

Rare Diseases: FDA Awards Grants for 21 Clinical, Natural History Studies By Michael Mezher - Published 06 October 2017

The US Food and Drug Administration (FDA) on Friday awarded $32 million in research grants to fund 15 clinical studies and six natural history studies for rare diseases.

Categories: News, US, FDA, Biologics and biotechnology, Clinical, Drugs

Tags: Natural History Studies, Rare Diseases, Orphan Drugs, Grants

FDA Calls for End to Priority Review Vouchers as GAO Says Too Early to Gauge Effectiveness By Zachary Brennan - Published 02 March 2016

The US Government Accountability Office (GAO) said Wednesday in a new report that it’s still too early to assess whether the Food and Drug Administration’s (FDA) three-year-old pediatric priority review voucher (PRV) program has stimulated the development of drugs to treat or prevent rare pediatric diseases.

Categories: News, US, FDA, Active pharmaceutical ingredients, Clinical, Drugs, Government affairs, Manufacturing, Research and development, Submission and registration

Tags: PRV, priority review voucher, pediatric rare diseases

Rare Disease Patient Group Commends EMA, Wants EU-Wide Access to Treatment By Michael Mezher - Published 02 March 2015

A rare disease advocacy network has applauded the European Medicines Agency’s (EMA) efforts to incentivize the development of treatments for rare diseases, but says  the variance in treatment access across the EU is “unacceptable.”

Categories: News, Europe, EC, EMA, Government affairs, Regulatory strategy, Submission and registration

Tags: Orphan product designation, rare diseases

FDA Guidance: How to Improve Your Meetings With the Office of Orphan Product Development By Alexander Gaffney, RAC - Published 10 April 2014

Categories: Meetings, US, FDA

Tags: Orphan Drug Designation, Orphan Drug Act, Meeting, Latest News, OOPD, Orphan Drugs, Rare Diseases, draft guidance

FDA Announces $14 Million in Grants for Trials in Support of Rare Disease Therapies By Alexander Gaffney, RF News Editor - Published 21 October 2013

Categories: US, FDA

Tags: Neglected Diseases, Grant, Orphan Drug Act, Latest News, Orphan Drugs, Rare Diseases

OECD Calls for Regulatory Harmonization to Support Global Clinical Trials System By Alexander Gaffney, RF News Editor - Published 25 February 2013

Categories: OECD

Tags: Orphan Diseases, harmonization, Latest News, research, Rare Diseases, clinical trials

In Landmark Reversal of Policy, Health Canada Announces Launch of Orphan Drug Policy By Alexander Gaffney - Published 04 October 2012

Categories: Health Canada

Tags: Orphanet, Orphan Drug Act, Latest News, Orphan Drugs, Rare Diseases

Perspective: A Modern Progressive Approval System for Rare Diseases By Marc Dunoyer - Published 16 April 2012

The recent dramatic increase in the number of orphan drug designations has prompted patient groups, pharmaceutical companies, legislators and many other stakeholders to look for ways to accelerate the delivery of innovative new medicines to people with rare diseases. In particular, patients suffering from illnesses for which there are no adequate licensed therapies want access to promising new products earlier in the drug development cycle.

Tags: Orphan Diseases, Perspective, pharmaceutical, rare, Orphan, Rare Diseases, development, drug, disease

Group Advocates Treating Superbugs Like Rare Diseases By Alexander Gaffney - Published 08 March 2012

Categories: FDA

Tags: antibiotics, bacteria, Super bugs, IDSA, Orphan Drug Act, Superbugs, Orphan Diseases, Latest News, Rare Diseases