In recent years, advanced manufacturing has gained traction in the pharmaceutical industry, transforming how drugs are developed, produced, and delivered. It is recognized, however, that there is a learning curve across the industry and FDA that is inherent to the...
Premier Research
Accelerated approval is an expedited regulatory pathway designed to hasten the availability of drugs (including biologics) that treat serious conditions, offer advantages over existing therapies, and address unmet medical needs. The FDA defines a serious condition as “a disease or...
Each year, on September 17, healthcare communities worldwide observe World Patient Safety Day, an important occasion dedicated to raising awareness about patient safety and promoting efforts to reduce preventable harm in healthcare settings. Established by the World Health Organization (WHO)...
The 505(b)(2) new drug application (NDA) pathway offers a unique opportunity for small molecule developers to bring innovative products to market more efficiently by leveraging existing data they do not own or have right of reference to. Unlike the traditional...
In rare diseases, patient opinion and insight often go unheard. However, for sponsors of rare disease clinical trials, study success often rests on incorporating the patient perspective as early as possible in development. Many patients with rare diseases have undertaken...
As clinical development of an investigational product proceeds, Sponsors often conduct global clinical trials which require preparation of content to support dossiers in multiple geographies, meeting the regulatory requirements of each region. The major markets follow ICH guidelines, which affords...
On July 31, 2024, the US Food and Drug Administration (FDA) announced Fiscal Year 2025 (FY2025) Prescription Drug User Fee Amendments of 2022 (PDUFA VII) fee rates for the review of human drug and biological product applications along with prescription...
On June 26, 2024, the U.S. Food and Drug Administration (FDA) released its much-anticipated draft guidance on clinical trial diversity.1 This 23-page document outlines proposed requirements for racial and ethnic diversity action plans, which will soon become mandatory for studies...
A Target Product Profile (TPP) serves as the primary strategic framework that aligns commercial and clinical workstreams. When properly defined, the TPP enables drug developers to manage the development equation of risk x cost x time = success, and it...
Cell & Gene Therapy
FDA INTERACT Meetings: Early Interactions for Cell and Gene Therapy Sponsors
Advances in scientific knowledge and growth in the cell and gene therapy space have led to a new and exciting era of medicine for patients, as well as a new motivation for regulators to provide clear, efficient pathways for product...