• Regulatory NewsRegulatory News

    CHMP recommends 12 new medicines, including first RSV treatment for infants

    The European Medicine Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) last week endorsed 12 new medicines for authorization, including the first treatment to prevent respiratory syncytial virus (RSV) in newborns, driving the total number of endorsed treatments to 70 since the beginning of the year. Five of the 12 approval recommendations are for orphan drugs.   The new RSV treatment, Beyfortus (nirsevimab) developed by AstraZeneca and Sanofi, was s...
  • Regulatory NewsRegulatory News

    FDA to form task force, public-private partnership on rare neurodegenerative diseases

    The US Food and Drug Administration (FDA) released a five-year action plan for accelerating drug development for rare neurodegenerative diseases, such as amyotrophic lateral sclerosis (ALS), as well as improving access to novel therapies.   “The effects of rare neurodegenerative diseases are devasting, with very few effective therapeutic options available to patients,” Robert M. Califf, MD, FDA Commissioner, said in a statement. “This action plan, especially including ...
  • Regulatory NewsRegulatory News

    RWD in clinical trials: External control arms take the lead

    The US Food and Drug Administration (FDA) has now published four draft guidances spelling out how it will consider real-world data (RWD) in regulatory decision-making, from the use of electronic health records and registries to RWD in non-interventional studies. More guidance documents are planned, including details on study designs that incorporate RWD for external control arms.   External control arms, which use data collected outside of the current trial to provide ...
  • Regulatory NewsRegulatory NewsRAPSRAPS' Latest

    Orphan drug experts discuss new book on developing rare disease treatments

    Developing therapies for rare diseases involves complex incentives, unique requirements, and often extensive patient engagement. The authors of RAPS’ Orphan Drug Development for Rare Diseases , Sundar Ramanan, PhD, MBA, and Sunny Kamlesh Dave, MPharm, recently took time to discuss with RAPS Senior Editor Gloria Hall the importance of orphan drugs, the peculiarities and challenges of obtaining an orphan drug designation (ODD), and what is covered in the book. During a “m...
  • Feature ArticlesFeature Articles

    The RACE is on: Opportunities in pediatric oncology product development

    Pediatric oncology patients continue to have significant unmet medical need. Although the Pediatric Research Equity Act (PREA) mandated evaluation of new drugs for pediatric patients, the rarity and uniquity of pediatric cancers allowed for waivers or exemptions from PREA requirements. The passing of FDA Reauthorization Act amendments in 2017 included the Research Acceleration for Cure and Equity Act, which expands the scope of oncology products subject to PREA. Sponsors d...
  • Regulatory NewsRegulatory News

    New public-private consortium will tackle gene therapies for rare diseases

    Two federal agencies are partnering with pharmaceutical companies and non-profit organizations to form a consortium aimed at boosting the development of gene therapies for rare diseases.   In a Wednesday announcement, the US Food and Drug Administration (FDA) and the National Institutes of Health (NIH) revealed the launch of the Bespoke Gene Therapy Consortium (BGTC). The consortium’s work will be managed by the Foundation for the NIH and sit within the NIH Acceleratin...
  • Regulatory NewsRegulatory News

    For gene therapies, FDA drafts trial guidance, finalizes "sameness" for orphan exclusivity

      A new draft guidance from the US Food and Drug Administration (FDA) gives cell and gene therapy developers a detailed framework for the conduct of umbrella trials, offering the potential for enhanced speed and efficiency in early-stage clinical trials.   In the umbrella trials envisioned under the draft guidance, two or more versions of a cellular or gene therapy product would be studied for one specific disease using just one trial design, shared infrastructure, a...
  • Regulatory NewsRegulatory News

    News in Focus: PDUFA VII is a step in the right direction for the rare disease community

    Craig Martin is chief executive officer of Global Genes, a nonprofit organization whose mission is to increase public awareness for rare disease and globally connect, empower and educate individuals and rare disease foundations in person, print and online. More than 400 million people worldwide are affected by 7,000+ rare diseases. In the U.S., around 1 in 10 people (more than 33 million Americans) have a rare condition. More than half of rare disease patients are chi...
  • Regulatory NewsRegulatory News

    EFPIA, others question EC's proposed orphan and pediatric drug development updates

    An initiative by the European Commission (EC) to boost the development of pediatric drugs and medicines for people with rare diseases has received stakeholder feedback that in many cases expresses concern with the proposed upending of the status quo.   A 60-day period of public consultation for the initiative’s roadmap closed at the end of July; in all, 112 comments were received from individuals, non-governmental organizations (NGOs), public authorities, medical socie...
  • Regulatory NewsRegulatory News

    Euro Convergence: How orphan drug suppliers can win labelling exemptions

    A strong local presence in EU markets is needed when seeking labelling exemptions that help reduce the cost of providing orphan products, according to experts who spoke at RAPS Euro Convergence 2021.   Manufacturers of orphan drugs can gain exemptions from some labelling requirements at an EU level and from individual member states, a potentially valuable flexibility for companies that make orphan drugs targeting very small numbers of patients. Such products face speci...
  • Regulatory NewsRegulatory News

    CHMP recommends eight new medicines in April, including two orphans

    In its April meeting, the European Medicines Agency’s (EMA’s) human medicines committee recommended eight new medicines for approval, including three new non-orphan medicinal products and two orphan medicines.   EMA’s Committee for Medical Products for Human Use (CHMP) also issued positive opinions for extensions of therapeutic indications for nine medicines, bringing the total new indications for 2021 to 23.   Leo Pharma’s Adtralza (tralkinumab) was recommended ...
  • Regulatory NewsRegulatory News

    Orphan product designation requests climb in the US, remain steady in the EU

    The number of new requests for orphan drug designation in the United States increased 41% from 2019 to 2020, bringing the number of requests to 753 for 2020, according to the U.S. Food and Drug Administration.   There was also a significant jump in rare pediatric disease designation requests, which rose 330% from 2019 to 2020, for a total of 284 requests in 2020. The agency received 20 humanitarian use device designation requests, similar to the number received in 2019...