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  • Regulatory NewsRegulatory News

    Bipartisan Group of Senators Introduces Bill to Speed Development of Rare Disease Drugs

    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. Although it's unclear how exactly the bill will aid in the development of targeted rare disease treatments, one of the cosponsors, Sen. Elizabeth Warren (D-MA), said the bill would clarify the US Food and Drug Administration's (FDA) current authority to consider resear...
  • Regulatory NewsRegulatory News

    EMA Wants to Help Drugmakers Show Their Orphan Products' Benefits

    The European Medicines Agency (EMA) is looking help sponsors developing new orphan drugs by clearing up questions on how they can demonstrate their product's improvement over existing therapies. To do so, the agency will be hosting a workshop on 7 December 2015, where it says industry, regulators, health technology assessment (HTA) bodies and other groups will have the opportunity to discuss approaches to demonstrating "significant benefit" for orphan products. Orphan D...
  • Regulatory NewsRegulatory News

    New FDA Guidance Addresses Common Issues in Orphan Drug Development

    The US Food and Drug Administration (FDA) has released a draft guidance intended to help drugmakers tackle common issues encountered in the development of drugs to treat rare diseases. Background In the US, orphan drugs, or drugs used to treat rare diseases which affect fewer than 200,000 people, are given incentives, such as a longer period of marketing exclusivity, tax credits and user fee waivers. These incentives were first instituted with the passage of the 1983 ...
  • Regulatory NewsRegulatory News

    Japan Edges out FDA for Fastest Approvals

    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). New Drug Approvals in ICH Countries A new report by the Centre for Innovation in Regulatory Science (CIRS), New Drug Approvals in ICH Countries 2005-2014 , examines NAS approvals in the US, EU and Japan. The report finds that FD...
  • Regulatory NewsRegulatory News

    FDA Exercising 'Extraordinary' Flexibility on Drugs for Rare Diseases, New Study Finds

    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. Background The root of this "reasonable" argument starts with a law intended to facilitate the development of new therapies for patients with rare diseases. Under the 1983 Orphan Drug Act , a rare disease is one which a...
  • Regulatory NewsRegulatory News

    Australia Wants Input on Orphan Drugs Program as it Conducts Review

    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. Orphan Drugs Regulation in Australia Orphan drugs are products intended to treat, diagnose or prevent rare diseases. Because the potential treatment populations for these drugs are small, regulators often offer incentives to encourage the development of treatments...
  • Regulatory NewsRegulatory News

    EMA in 2014: A Landmark Year for Approvals, With Eyes on the Future

    In the European Medicines Agency's (EMA) Annual Report 2014 , top regulatory officials describe the agency's role as both a "gatekeeper" and "enabler." This dual role means that the agency must not only work to ensure the safety of patients in the EU, but also to facilitate the development of new and innovative therapies. By the Numbers 2014 was a landmark year for EMA in many ways. The agency recommended record numbers of products to treat rare diseases , launched it...
  • Regulatory NewsRegulatory News

    EU Court Rules on Orphan Product Exclusivity

    A European Court has issued a ruling in an unlikely scenario dealing with overlapping periods of orphan product market exclusivity. In its ruling, which has the potential to influence how companies develop orphan products, the court found that authorized orphan products are entitled to market exclusivity, even when they are similar to an already approved product. Background Orphan product designation was first introduced in the EU in 2000 under Regulation (EC) No 141...
  • Regulatory NewsRegulatory News

    Rare Disease Patient Group Commends EMA, Wants EU-Wide Access to Treatment

    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.” In an interview with EurActiv , Terkel Andersen, president of the advocacy group EURODIS, said that EMA and the European Commission (EC) are “doing their utmost to try to make rare diseases ‘attractive’ for the pharmaceutical industry.” ...
  • Regulatory NewsRegulatory News

    Orphan Drugs in the EU: A Record-Breaking Year

    The European Medicines Agency (EMA) recommended 17 drugs with orphan product designation for approval in 2014, the most ever in a single year. This is much higher than the average of 6.75 per year between 2010 and 2013 and is a significant increase from 2013, when EMA recommended 11 products with orphan designation for approval. Background: Orphan Designation Orphan product designation was first introduced in the EU in 2000 under Regulation (EC) No 141/2000 . The reg...
  • Feature ArticlesFeature Articles

    Canada and the Orphan Drug Framework--Where Are We Now?

    Canada, one of the few developed countries without an orphan drug policy, is finally in the last stages of developing one. In the past, when queried about the lack of a policy, representatives from the country’s food and drug regulatory agency, Health Canada, stated that physicians could apply to have patients receive a non-approved orphan drug through the Special Access Programme or through clinical studies (if conducted in Canada). For manufacturers of orphan drug...
  • Feature ArticlesFeature Articles

    US Orphan Drug Exclusivity Criteria Clarified in Depomed Court Decision

    A recent US district court decision could affect US Food and Drug Administration (FDA) interpretation and determinations in the orphan drug exclusivity area. Specifically, on 5 September 2014, the United States District Court for the District of Columbia issued its memorandum opinion in the case of Depomed Inc. v. United States HHS . 1 The case centers on the Orphan Drug Act , which provides incentives to pharmaceutical companies to research and develop drugs for rare ...