Liaison(s): May Orfali, Cara Cassino, Jack Mardekian
Pfizer, Inc. is a research-based, global biopharmaceutical company with a diversified portfolio spanning from human to animal biologics, small molecule entities, vaccines, and nutritional products. The company has demonstrated a long-standing commitment to rare disease drug development through its ongoing leadership in the orphan space, resulting in 17 approved orphan drugs in the US market. Pfizer is interested in expanding its focus in this area by adopting innovative drug development strategies, public health policies and patient-focused treatment designs. The 1983 US Orphan Drug Act has facilitated the development of therapies for rare diseases. Orphan drugs are defined by the Orphan Drug Act to be drugs which target rare diseases affecting fewer than 200,000 people in the US. Drugs that have attained the orphan drug designation are eligible for grants to perform clinical trials, a 50% tax credit for clinical testing costs and, most importantly, a 7-year marketing exclusivity for the drug. The orphan drug manufacturers are also eligible for expedited review by the US Food and Drug Administration (FDA) and a fee waiver incentive for New Drug Applications (NDAs). The goal of this TMP was to present an objective comparison of the orphan and non-orphan drug development pathway which leads to regulatory approval. This analysis will provide the necessary information to evaluate rare disease drugs using the adjusted metrics required for the small rare disease patient population and the risk benefit profile for these diseases. The team was asked to perform a systematic comparison of the regulatory pathways of orphan and non-orphan drugs and biologics first approved by the FDA from January 1, 2001 to December 31, 2011. To accomplish this objective, the team completed a detailed analysis of clinical trial characteristics extracted from package inserts for newly approved drugs. Ultimately, the Pfizer TMP compiled this information into a white paper which examined a number of clinical trial characteristics including: 1, number of trials performed; 2, largest trial size; 3, total number of patients enrolled; 4, randomization; 5, blinding; and 6, trial endpoint. Results showed that there was a statistically significant difference in a number of trial characteristics. In the therapeutic areas of specialty neuroscience (SNS), pulmonology, hematology, and endocrinology, we observed significant differences in: 1, the number of trials; 2, the largest trial size; and 3, the total number of patients enrolled.