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Filtering on: "topics orphan products"

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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

House Bill Would Incentivize Drugmakers to 'Repurpose' Drugs for Rare Diseases By Michael Mezher - Published 02 March 2017

Rep. Gus Bilirakis (R-FL) on Monday reintroduced a bill that would grant drugmakers an additional six months exclusivity for repurposing already-approved drugs to treat rare diseases.

Categories: News, US, FDA, Biologics and biotechnology, Drugs, Government affairs, Orphan products, Submission and registration

Tags: OPEN Act, Marketing Exclusivity,

EMA Reflects on its First Decade of Conditional Marketing Authorizations By Michael Mezher - Published 24 January 2017

Ten years after the development of the conditional marketing authorization (CMA) pathway, European Medicines Agency (EMA) on Monday said the program is working as intended, and has led to earlier patient access to 30 drugs, nearly half of which were for orphan indications, and 80% of which target seriously debilitating or life-threatening conditions.

Categories: News, Europe, EMA, Biologics and biotechnology, Clinical, Drugs, Orphan products, Regulatory strategy, Submission and registration

Tags: Conditional Marketing Authorisation, CMA

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

FDA’s Office of New Drugs Director to Retire By Zachary Brennan - Published 05 December 2016

After nearly 25 years of service at the US Food and Drug Administration (FDA), John Jenkins, MD, Director of the Office of New Drugs (OND), will retire from the federal government on 6 January 2017.

Categories: News, US, CDER, Biologics and biotechnology, Drugs, Government affairs, Orphan products, Regulatory intelligence, Regulatory strategy

Tags: John Jenkins, Office of New Drugs, OND, CDER's OND, new drug applications

European Commission to Overhaul Concept of ‘Similar’ Medicines Within Context of Orphan Legislation By Zachary Brennan - Published 29 July 2016

The European Commission on Friday proposed to review the concept of “similar medicinal products” in the context of its orphan legislation as part of a wider effort to adapt the text to technical progress.

Categories: News, Europe, EC, Active pharmaceutical ingredients, Biologics and biotechnology, Drugs, Government affairs, Orphan products

Tags: orphan drug legislation, European Commission, gene therapy, ATMP

Can Health Technology Assessment be Used to Demonstrate Significant Benefit for an Orphan Medicinal Product in the EU? By Richard Phillips, Adriaan Fruijtier, Mauro Placchi - Published 07 July 2016

This article discusses how the conceptual grounds for significant benefit are addressed in the context of the orphan medicinal product regulations in the EU and how techniques used in Health Technology Assessment (HTA) may support the justification for the claim of significant benefit.

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

EMA's 2015 Annual Report: Interest in Scientific Advice Increasing By Michael Mezher - Published 17 May 2016

The European Medicines Agency (EMA) on Tuesday released its Annual Report 2015 touting its performance and key achievements for the year.

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

Tags: Guido Rasi, Sir Kent Woods, New active substances, Scientific Advice

Study: Orphan Drugs More Innovative Than Non-Orphan Products By Michael Mezher - Published 23 March 2016

A recent study of new drugs approved by the US Food and Drug Administration (FDA) finds that orphan products are more innovative than their non-orphan counterparts, representing roughly 40% of first-in-class drug approvals between 1983 and 2014.

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

EU High Court Upholds Orphan Exclusivity Ruling By Michael Mezher - Published 23 March 2016

Earlier this month, the EU's top court upheld a major ruling with implications for orphan drug exclusivity by rejecting an appeal by generic drugmaker Teva Pharmaceuticals Europe.

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

Tags: Glivec, Gleevec, Tasigna, Teva, Novartis, Market exclusivity

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

$2 Million in Funding From FDA for Rare Disease Natural History Studies By Michael Mezher - Published 29 February 2016

The US Food and Drug Administration (FDA) will award $2 million in grants to fund natural history studies for rare diseases.

Categories: News, US, FDA, Orphan products

Orphan Medicines in the EU: A 15-Year Review By Zachary Brennan - Published 29 January 2016

With the 15th anniversary of the adoption of the Orphan Regulation in the EU, the European Commission notes in a new report that this landmark regulation has incentivized the development, approval and marketing of more than 100 rare disease treatments, or orphan drugs.  

Categories: News, Europe, EC, EMA, Biologics and biotechnology, Drugs, Government affairs, Manufacturing, Orphan products, Research and development

Tags: orphan medicines, rare disease drugs, cancer drugs, orphan drug incentives

FDA Approves First Drug to Improve Overall Survival in Liposarcoma By Michael Mezher - Published 28 January 2016

The US Food and Drug Administration (FDA) on Thursday approved a new indication for Eisai's Halaven (eribulin mesylate) to treat liposarcoma, a rare and deadly form of soft tissue sarcoma (STS) that affects only a few thousand patients in the US each year.

Categories: News, US, FDA, Drugs, Orphan products, Submission and registration

Tags: Halaven, erubulin, Liposarcoma

EMA Carries 2014 Momentum, Recommends 39 New Drugs and Sets Orphan Record By Michael Mezher - Published 12 January 2016

In 2015, the European Medicines Agency (EMA) maintained its momentum from 2014, granting 93 positive opinions, 39 of which were for new active substances. The agency also recommended 18 orphan drugs for approval, breaking last year's record of 17.

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

Tags: New active substance, new chemical entity, 2015 approvals, Marketing authorization

Proposed Rule Would Revise Regulations on Fixed-Combination Drugs By Zachary Brennan - Published 22 December 2015

The US Food and Drug Administration (FDA) is proposing a new rule that would apply its regulations on prescription fixed-combination drugs to a variety of nonprescription fixed-combination and co-packaged drugs, as well as to combinations of over-the-counter active ingredients.

Categories: Biologics and biotechnology, Business Skills, Clinical, Combination products, Compliance, Drugs, Ethics, Government affairs, Labeling, Orphan products, Packaging, Preclinical, Project management, Regulatory strategy, Submission and registration

Tags: fixed-combination drugs, FDA rulemaking, proposed FDA rule, OTC drug monographs, nonprescription drug combinations

European Commission to Evaluate EMA’s Fee System By Zachary Brennan - Published 22 December 2015

Late next year, the European Commission will begin reviewing the pharmaceutical company fee system that helps fund the European Medicines Agency (EMA).

Categories: News, Europe, EC, EMA, Clinical, Compliance, Drugs, Due Diligence, Ethics, Government affairs, Manufacturing, Orphan products, Regulatory strategy, Reimbursement, Submission and registration

Tags: EMA, pharmacovigilance, marketing authorization fees in EU, marketing authorisation

EMA Outlines High-Level Strategy for Next Five Years By Zachary Brennan - Published 18 December 2015

Antimicrobial resistance, ensuring timely access to new drugs, increasing transparency and working to harmonize more regulations worldwide are all included in the European Medicines Agency’s (EMA) strategic plan through 2020.

Categories: News, Europe, EMA, Active pharmaceutical ingredients, Biologics and biotechnology, Business and Leadership, Clinical, Crisis management, Drugs, Ethics, Government affairs, Manufacturing, Orphan products, Regulatory intelligence, Regulatory strategy

Tags: EMA, medicines regulation, clinical trials, harmonized regulations

Regulatory Recon: FDA Settles Free Speech Suit Over Pacira Drug’s Label (15 December 2015) By Zachary Brennan - Published 15 December 2015

Welcome to Regulatory Reconnaissance, your daily regulatory news and intelligence briefing. Want to read Recon as soon as it's posted? Follow @Michael_Mezher , @Zachary Brennan and @RAPSorg on Twitter.

Categories: News, Asia, Europe, Oceania, US, EMA, FDA, ICH, MHRA, TGA, Active pharmaceutical ingredients, Advertising and Promotion, Biologics and biotechnology, Combination products, Compliance, Distribution, Drugs, Due Diligence, Government affairs, Manufacturing, Medical Devices, Orphan products, Quality

Tags: Regulatory Reconnaissance, Regulatory, Links, News, Regulatory News, Regulatory Intelligence, FDA News, EMA

Importers of FDA-Regulated Products Prepare for February Shift to Automated System By Zachary Brennan - Published 09 December 2015

In partnership with Customs and Border Protection (CBP) and 46 other government agencies, the Food and Drug Administration (FDA) has developed and is quickly transitioning to a new portal through which industry can electronically submit all data required by government agencies for international trade.

Categories: News, Africa, Asia, Canada, Europe, Latin America and Caribbean, Middle East, Oceania, US, FDA, Active pharmaceutical ingredients, Audit, Biologics and biotechnology, Combination products, Distribution, Drugs, Government affairs, Medical Devices, Orphan products, Quality

Tags: FDA imports, import alert, ACE, CBP

Consumer Advocacy Group Warns on Orphan Provisions in 'Cures' Act By Michael Mezher - Published 08 December 2015

Orphan drug provisions in the House-passed 21st Century Cures Act could increase US healthcare spending by billions of dollars over the next decade, consumer advocacy group Public Citizen warns, though the bill still remains in limbo in the Senate.

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

Tags: 21st Century Cures, Orphan Product Extensions Now Act, OPEN Act, Pediatric Research Equity Act, PREA, Public Citizen

FDA Offers Guidance for Blood Establishments During Ebola Outbreaks By Michael Mezher - Published 02 December 2015

The US Food and Drug Administration (FDA) has released new recommendations for blood establishments to implement additional screening measures during Ebola virus outbreaks.

Categories: News, Africa, US, CBER, WHO, Biologics and biotechnology, Blood, In vitro diagnostics, Medical Devices, Orphan products

Tags: Ebola

Regulatory Recon: FDA Panel Finds DMD Drug Data Not Persuasive (25 November 2015 By Zachary Brennan - Published 25 November 2015

Welcome to Regulatory Reconnaissance, your daily regulatory news and intelligence briefing. Hope you have a happy Thanksgiving tomorrow. Regulatory Focus will not be publishing Thursday or Friday. Enjoy the weekend.

Categories: News, Africa, Asia, Canada, Europe, Latin America and Caribbean, US, EC, EMA, FDA, PMDA, TGA, Business and Leadership, Clinical, Compliance, Drugs, Ethics, Government affairs, Manufacturing, Medical Devices, Orphan products, Quality, Research and development

Tags: Regulatory Reconnaissance, Regulatory, Links, News, Regulatory News, Regulatory Intelligence, FDA News, EMA News

Johns Hopkins Researchers Call for Orphan Drug Reform By Michael Mezher - Published 20 November 2015

A team of researchers at Johns Hopkins are calling for reforms to the Orphan Drug Act, saying that loopholes have allowed drug companies to skirt the act's intent by taking advantage of its incentives for non-orphan conditions.

Categories: News, US, FDA, Biologics and biotechnology, Drugs, Orphan products, Regulatory strategy, Submission and registration

Tags: Orphan Drug Act, Johns Hopkins

Regulatory Recon: FDA Approves New Lung Cancer Drug, Companion Diagnostic (13 November 2015) By Zachary Brennan - Published 13 November 2015

Welcome to Regulatory Reconnaissance, your daily regulatory news and intelligence briefing. Want to read Recon as soon as it's posted? Follow @Michael_Mezher , @Zachary Brennan and @RAPSorg on Twitter.

Categories: News, Asia, Europe, Latin America and Caribbean, Middle East, Oceania, US, CDSCO, CFDA, DOJ, EMA, FDA, Audit, Business Skills, Clinical, Compliance, Crisis management, Drugs, Due Diligence, Ethics, Government affairs, In vitro diagnostics, Manufacturing, Medical Devices, Orphan products, Postmarket surveillance, Preclinical, Quality, Regulatory intelligence, Regulatory strategy, Research and development

Tags: Regulatory Reconnaissance, Regulatory, Links, News, Regulatory News, Regulatory Intelligence, FDA News, EMA News