Search Results for orphan drugs

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European Regulatory Roundup: EU Stands Firm on Demand for UK to Pay for EMA’s Exit (7 December 2017) By Nick Paul Taylor - Published 07 December 2017

Welcome to our European Regulatory Roundup, our weekly overview of the top EU regulatory news.

Categories: News, Europe, EC, EMA, Biologics and biotechnology, Drugs, Government affairs

Tags: Brexit, PRAC, orphan drugs

FDA's Frey Reflects on Final Year of PDUFA V By Michael Mezher - Published 06 December 2017

As the year comes to a close, Patrick Frey, senior advisor to the director of the Office of New Drugs (OND) at the Center for Drug Evaluation and Research (CDER), looked back at FDA's performance over the final year of the fifth iteration of the Prescription Drug User Fee Act (PDUFA V) and what's to come in PDUFA VI.

Categories: News, US, CDER, Biologics and biotechnology, Communication, Drugs, Orphan products, Submission and registration

Tags: PDUFA, NDA, BLA, Orphan Drugs, Approvals

FDA Analyst Counters Critiques of Orphan Drug Act By Michael Mezher - Published 18 October 2017

While recent reports have claimed that drugmakers are manipulating the incentives provided by the Orphan Drug Act, Mike Lanthier, an operations research analyst at the US Food and Drug Administration (FDA), says that in most cases, the act is working as intended.

Categories: News, FDA, Biologics and biotechnology, Drugs, Orphan products

Tags: Orphan Drugs, Orphan Drug Act

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

TGA to Move Forward With Orphan Drug Revamp By Michael Mezher - Published 18 April 2017

Australia's pharmaceutical industry is "broadly supportive" of proposed changes to the country's orphan drug program, the Therapeutic Goods Administration (TGA) says.

Categories: News, Oceania, TGA, Biologics and biotechnology, Drugs, Orphan products, Regulatory strategy, Submission and registration

Tags: Orphan Drugs, Australia

Sen. Grassley Opens Inquiry Into Orphan Drug Act By Zachary Brennan - Published 10 February 2017

Sen. Charles Grassley (R-IA) on Friday confirmed to Focus that he is gathering more information and discussing with his staff and interested parties a possible inquiry into the Orphan Drug Act’s abuses leading to high drug prices.

Categories: News, US, FDA, Drugs, Government affairs, Regulatory intelligence, Regulatory strategy

Tags: Orphan Drug Act, Grassley, orphan drugs

European Commission Clears up Questions on Orphan Drug Regulation By Michael Mezher - Published 05 January 2017

Following a public consultation in November 2015, the European Commission (EC) has released a new communication intended to clarify some lingering questions about the EU's Orphan Regulation.

Categories: News, Europe, EC, EMA, Biologics and biotechnology, Drugs, Orphan products

Tags: Orphan Regulation, Orphan Drugs, Orphan Designation

Regulators, Industry Weigh in on Proposed EU Orphan Regulation Changes By Zachary Brennan - Published 07 March 2016

As the number of orphan drug approvals continues to rise in the EU, the European Commission is now considering feedback from European governments, regulators and industry on how changes to five aspects of the Orphan Regulation may further incentivize the development of drugs for rare diseases.

Categories: News, Europe, EC, EMA, MHRA, Drugs, Government affairs, Orphan products

Tags: orphan drugs, Orphan Regulation, EC consultation, rare disease drugs

Sanofi Redeems $245M Priority Review Voucher for Type 2 Diabetes Treatment By Zachary Brennan - Published 22 February 2016

The US Food and Drug Administration (FDA) accepted for review the New Drug Application (NDA) for Sanofi’s investigational type 2 diabetes treatment on Monday and granted the use of another Priority Review Voucher (PRV), which speeds up FDA’s decision by four months.

Categories: News, US, FDA, Biologics and biotechnology, Compliance, Drugs, Due Diligence, Government affairs, Regulatory intelligence, Regulatory strategy, Submission and registration

Tags: priority review voucher, PRV, tropical disease, rare pediatric disease, orphan drugs

Industry Seeks More Specifics on FDA's Flexibility With Orphan Drug Guidance By Michael Mezher - Published 04 November 2015

Two industry associations and a rare disease patient advocacy group say they want the US Food and Drug Administration (FDA) to clarify its "regulatory flexibility" with orphan drug reviews.

Categories: News, US, FDA, Biologics and biotechnology, Clinical, Drugs, Orphan products, Preclinical, Regulatory strategy

Tags: Orphan drugs, Natural history studies, PhRMA, BIO, NORD

Studies Raise New Concerns Over FDA's Expedited Approval Designations, Supplemental Indications By Zachary Brennan - Published 24 September 2015

An increasing number of newly-approved drugs by the US Food and Drug Administration (FDA) have been associated with expedited development or review programs, though many of these approved drugs are not first in class and potentially less innovative, according to two new studies published in the British Medical Journal (BMJ).

Categories: News, US, FDA, Drugs, Due Diligence, Government affairs, Orphan products, Postmarket surveillance, Regulatory intelligence, Regulatory strategy

Tags: orphan drugs, expedited review, fast-track approval, FDA, Orphan Drug Act, rare disease

Bipartisan Group of Senators Introduces Bill to Speed Development of Rare Disease Drugs By Zachary Brennan - Published 16 September 2015

A new bipartisan bill aims to help accelerate the development of targeted drugs to treat rare diseases, including Duchenne muscular dystrophy, cystic fibrosis, some cancers and other genetic diseases.

Categories: News, US, FDA, Clinical, Drugs, Government affairs, Orphan products, Regulatory strategy

Tags: rare disease drug development, orphan drugs, OOPD, FDA, bipartisan legislation

New FDA Guidance Addresses Common Issues in Orphan Drug Development By Michael Mezher - Published 17 August 2015

The US Food and Drug Administration (FDA) has released a draft guidance intended to help drugmakers tackle common issues encountered in the development of drugs to treat rare diseases.

Categories: News, US, FDA, Biologics and biotechnology, Clinical, Drugs, Orphan products, Preclinical, Regulatory strategy, Research and development

Tags: Orphan drugs, Guidance documents

Japan Edges out FDA for Fastest Approvals By Michael Mezher - Published 31 July 2015

In 2014, Japan's regulator, the Pharmaceuticals and Medical Devices Agency (PMDA) edged out the US Food and Drug Administration (FDA) as the world's fastest regulator, with a median approval time of 306 days for new active substances (NASs).

Categories: News, Japan, Europe, US, EMA, FDA, MHLW, PMDA, Biologics and biotechnology, Drugs, Orphan products, Regulatory strategy, Submission and registration

Tags: New drug approvals, New active substance, NAS, Centre for Innovation in Regulatory Science, CIRS, ICH, Orphan drugs, Expedited review, Priority Review

FDA Exercising 'Extraordinary' Flexibility on Drugs for Rare Diseases, New Study Finds By Alexander Gaffney, RAC - Published 04 May 2015

The US Food and Drug Administration (FDA) is a reasonable regulator. That's the conclusion of a new analysis looking at the measures taken by FDA to review and approve new drugs intended for use in rare diseases affecting small populations of patients.

Categories: News, US, CDER, Biologics and biotechnology, Drugs, Orphan products

Tags: Orphan Drug Act, Rare Disease, Orphan Drugs

Australia Wants Input on Orphan Drugs Program as it Conducts Review By Michael Mezher - Published 01 May 2015

Regulators in Australia today released a discussion paper intended to solicit comments on its orphan drugs program, in light of changes in the landscape for rare diseases since the program's inception in 1997.

Categories: News, Oceania, TGA, Drugs, Orphan products, Regulatory strategy, Submission and registration

Tags: Orphan drugs, rare disease

Orphan Drugs in the EU: A Record-Breaking Year By Michael Mezher - Published 13 January 2015

The European Medicines Agency (EMA) recommended 17 drugs with orphan product designation for approval in 2014, the most ever in a single year.

Categories: News, Europe, EC, EMA, Biologics and biotechnology, Drugs, Orphan products, Submission and registration

Tags: Orphan Drugs, Rare Disease, Orphan Designation, COMP, Orphan Product Designation

Canada and the Orphan Drug Framework--Where Are We Now? By Patricia Anderson, RAC - Published 11 December 2014

Canada is in the final stages of developing an orphan drug policy for drugs affecting populations of 5 in 10,000 people. Bill C-17 has passed the legislature and is awaiting final approval. The new policy will then be open for comment. Fee mitigation, accelerated review and HTA collaboration are incentives but marketing exclusivity is not, although discussions on that topic are taking place. The final hurdle is provincial and private payment plans.

Categories: Features, Canada, EMA, FDA, Health Canada, Prescription drugs, Government affairs, Orphan products, Reimbursement

Tags: orphan drugs, Bill C-17, Vanessa's law, payment plans

US Orphan Drug Exclusivity Criteria Clarified in Depomed Court Decision By Alan Minsk, JD - Published 04 December 2014

Key points and observations about a recent federal court decision on orphan drug exclusivity as outlined in the case of Depomed v. US. The central issue concerned what conditions a drug must satisfy to qualify for marketing exclusivity.

Categories: Features, US, Generic drugs, Orphan products

Tags: Depomed v US, orphan drugs, Neurontin, Gralise, post-herpetic neuralgia, exclusivity, clinical superiority

Orphan Drugs Around the World: The Regulations and Requirements You Need to Know By Meredith Brown-Tuttle, RAC - Published 17 September 2014

"Orphan drugs" are product intended for use in the treatment of a "rare" disease—those diseases that occur infrequently or rarely in the general population of a specific location. 

Categories: Features, Asia, Canada, Europe, Latin America and Caribbean, Middle East, Oceania, US, EC, EMA, FDA, PMDA, TGA, Biologics and biotechnology, Drugs

Tags: Orphan Drugs, Orphan Disease, Orphan Drug Act, Ultra-Orphan Diseases, Orphan Drug Regulations

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

As US Population Grows, Rare Diseases Become Even Rarer By Alexander Gaffney, RAC - Published 15 January 2014

Categories: US

Tags: Orphan Drug Act, rare disease, Orphan Diseases, Latest News, Orphan Drugs

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

EMA Report: Approval Numbers Likely to be Lower than 2011 By Alexander Gaffney - Published 10 October 2012

Categories: EMA

Tags: Drug Approvals, Rasi, Report, Approvals, Latest News, Orphan Drugs

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