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    Perspective: Advancing Breakthrough Therapies for Patients

    Advances in science and medicine are leading to exciting possibilities for the treatment of serious or life-threatening conditions, such as cancer. Growing knowledge of disease biology and molecular targets has shifted drug development from a traditional "shotgun" approach to targeted, precision medicines. Understanding the biological basis and natural history of a complex disease, such as cancer, unlocks the potential for new treatment options. In particular, novel-nov...
  • Bill Introduced to Speed Development and Review of Breakthrough Therapies

    A bipartisan bill introduced in the House of Representatives would work to speed up the development and review of some drugs intended to treat life-threatening diseases by creating a new "breakthrough therapy" designation at the US Food and Drug Administration (FDA). The bill, To amend chapter V of the Federal Food, Drug, and Cosmetic Act (FD&C Act) to expedite the development and review of breakthrough therapies , would amend the FD&C Act  to include pr...
  • Texas Approves New Stem Cell Therapies, Setting up Possible FDA Showdown

    The state of Texas approved new-and controversial-rules to allow doctors to use adult stem cells to conduct institutional review board (IRB)-approved procedures on patients, reports The New York Times . The changes, instituted 13 April by the Texas Medical Board, were reportedly spearheaded by Texas Governor and former Republican presidential nominee Rick Perry, who has said he experienced relief from the procedures, reports The Times . Some researchers, however, are ...
  • Paper: Small Companies Need Regulatory Assistance from EMA

    The European Medicines Agency (EMA) needs to assist companies-particularly small businesses, academic institutions and charities-in the development of advanced-therapy medicines, claims a new paper written by members of the Committee for Advanced Therapies (CAT) in the journal Molecular Therapies . These smaller drug development entities often lack the resources necessary to "navigate regulatory procedures," including clinical trials and application submissions. The pa...
  • Bill Would Encourage FDA to Seek Expert Advice About Rare Diseases

    Proposed legislation introduced in Congress on 7 March would encourage the US Food and Drug Administration (FDA) to seek and receive advice from experts in the field of rare diseases. The Expanding and Promoting Expertise in Review of Rare Treatments Act of 2012 ( EXPERRT Act ) would allow a consultation programs to be established for the purpose of allowing an exchange of expert information. The goal of the program is to allow FDA staff to better inform and strengthen...