The journey of developing effective treatments for rare diseases, particularly in a pediatric population, has its own unique challenges, including (but not limited to), a small population size, disease heterogeneity, and the absence of established endpoints to support regulatory decisionmaking....
Tag: consulting 104 results
The FDA’s Oncology Center of Excellence (OCE) plays a pivotal role in fostering innovation, collaboration, and efficiency in the development and evaluation of oncology products. Established in 2017 under the 21st Century Cures Act, the OCE brings together multidisciplinary scientific...
New drug development is a long and expensive process that can be fraught with obstacles, including unexpected delays, poor efficacy results, safety issues, or regulatory challenges. Pharmaceutical companies may, at times, find themselves at an impasse during development, facing weighty...
Bringing a new therapeutic to market is a complex and multifaceted process culminating in submission and approval of a new drug application (NDA). Within the NDA, the chemistry, manufacturing, and controls (CMC) section plays a pivotal role in demonstrating drug...
Process validation is a critical regulatory requirement in pharmaceutical manufacturing. It ensures that a production process consistently yields products of predetermined quality and safety. The goal is to verify that all stages of drug production—from raw materials to final dosage...
For drug developers the 505(b)(2) pathway presents an expedited pathway to FDA approval. In our experience, the conversation on 505(b)(2) pathways commonly focuses on predicate data or bridging strategies to accelerate FDA approval but lacks dialogue on the needs of...
Inhaled combination products (ICP) have emerged as a significant advancement in the treatment of respiratory diseases such as asthma, chronic obstructive pulmonary disease (COPD), and other pulmonary conditions. Inhalation offers a targeted route of drug delivery directly to the lungs,...
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...
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...
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...