In May 2024, the International Council of Harmonization of Technical Requirements for Pharmaceuticals for Human Use (ICH) published a final draft of the newly developed scientific guidance on drug-drug interaction (DDI) studies, ICH M12, that will be adopted by ICH-abiding regulatory agencies (FDA, EMA, Japan). Read on to discover what is new about this DDI guidance...
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 starts by keeping the end in mind. Defining the needs of patients, physicians, and payers...
If there is one document that encapsulates the commercial-clinical handshake it is the Target Product Profile (TPP). It is the core strategic framework that serves to define and align commercial, clinical, medical, manufacturing, safety, and regulatory requirements. This group of key stakeholders all have an equal voice in the creation of a TPP. When this...
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 developers. These therapies frequently target diseases in patients that are rare (affecting fewer than 200,000...
As a cornerstone of the drug development process, nonclinical investigational new drug (IND)-enabling studies are essential for supporting first-in-human (FIH) dosing for novel therapeutics. High-quality IND-enabling studies demonstrate that a drug is reasonably safe for use in humans and that it exhibits sufficient pharmacological activity to justify further development, ensuring the highest probability of transitioning...
When pitching to investors, a sponsor is not just selling them on the product; it is also promoting its ability to develop that product. Investors want to know the risks involved with the program, but they are also evaluating the sponsor as a risk factor of its own. Investing in emerging biopharma companies is a...
Asking the appropriate questions during a pre-IND meeting with the FDA is a critical step in planning a development program. A Pre-Investigational New Drug Application (pre-IND) meeting can be a valuable component in planning a development program. For companies that have not previously interacted with the FDA in the early stages of a product’s development,...
Development of cell and gene therapies is growing rapidly, given the major advances in genomic technologies and increasing scientific understanding of genetic regulation and immunology. The Cell and Gene Therapy (CGT) field has unprecedented potential to effectively treat and/or cure genetic-based rare and orphan diseases and expand the reach of personalized medicine. This blog will...
Designing an adequate nonclinical program to support the safety of cell or gene therapy products is not always straightforward. In this blog, we will address common questions we receive from our clients and aspects to consider when designing those nonclinical studies. In which species should the toxicity study be conducted? Does it have to be...
As regulatory requirements become increasingly harmonized across the globe, the development and marketing of pharmaceutical products worldwide are also becoming more streamlined. However, global regulations are not one-size-fits-all, and sponsors aiming to market their products in multiple regions should be aware of the current standards and processes they may encounter during the development process. The...