Regulatory Guidelines and Approval Process for Drugs and Formulations for Alzheimer’s Disease (AD)

Alzheimer’s disease (AD) is a prevalent condition that impacts more than 6.5 million individuals in the United States. This persistent medical condition progressively hampers memory, cognitive abilities, and the capacity to carry out everyday activities. AD is characterized by specific changes in the brain, including the presence of tau tangles and amyloid beta plaques, although the exact cause is still unknown. The FDA (Food and Drug Administration) guarantees the safety and efficacy of authorized drugs in the United States by conducting a comprehensive evaluation process. The Prescription Drug User Fee Act (PDUFA) of 1992 (505(b)(1)) implemented a two-tiered examination process for New Drug Applications (NDAs), specifically for Standard Review and Priority Review. The objective of implementing these techniques was to reduce the duration of drug reviews. In addition, the FDA has the authority to approve drugs that target serious medical conditions through the Breakthrough Therapy, Accelerated Approval, and Fast Track programs. This is particularly relevant in cases where there are few effective treatments available, and a drug has demonstrated a noticeable impact on a surrogate endpoint that can reasonably serve as a substitute for a therapeutic benefit for patients. This chapter aims to offer readers comprehensive guidelines and a concise overview of the regulatory approval process for drugs and formulations designed specifically for the treatment of AD.