Bak A, Burlage R, Greene N, Nambiar P, Lu X, Templeton A. 2024. Accelerating drug product development and approval: Early development and evaluation. Pharm Res 41(1):1-6; doi: 10.1007/s11095-023-03566-1.
Abstract
A major discovery and early development challenge has been and still is how to develop the best and most impactful medicines for patients as fast as possible, two aims that can appear at face value to be contradicting. This is especially the case in recent times, where the modality landscape has expanded significantly to not only include traditional small molecules and proteins, but also peptides, antibody drug conjugates (ADC), a variety of nucleotide-based therapies such as antisense oligonucleotides (ASO), small interfering ribonucleic acids (siRNA), messenger RNAs (mRNA), and cell and gene therapies just to name a few. See Fig. 1 (Source data from) for an overview over BLA (Biologics License Application) and NME (New Molecular Entity) applications approved by CDER. From the figure it can be seen that the number of BLAs trends up, while the number of traditional NMEs trend down. Chen et al. (2023) have looked at this data in further details and accordingly it can be seen that the number of small molecule filings are going down while the number of protein therapeutics are increasing. All modalities have their own requirements for preclinical and drug product development, which can lead to complexity and attrition.
