Unveiling the impacts of FDA Breakthrough Therapy Designation: a dual-perspective examination of economic and developmental outcomes for biotechnology companies.

Biotechnology and small pharma companies must recognize the opportunity presented by FDA Breakthrough Therapy Designation (BTD) to expedite drug development for serious conditions. This paper evaluates BTD economic and developmental impacts on such companies. Analysis of 29 BTD events from 2017 to 2022 revealed immediate share price boosts and improved drug approval rates, alongside faster development times. Yet, long-term share prices underperformed relative to the broader market, underscoring the persistent risks in pharma investments. Although offering new insights, the limited sample size calls for further investigation.

Evaluating the impact of the FDA's orphan drug designation on share prices of biotechnology companies.

This analysis explores the impact of the FDA's orphan drug designation (ODD) on biotechnology companies' share prices in the short and long term. Our analysis reveals that there is a significant immediate increase in share prices following an ODD announcement, which underscores its potential as a robust indicator of short-term investor returns. However, the long-term findings present a more complex picture, with a sustained impact within the biotech industry but a significant depreciation against the broader market over time. These findings elucidate the nuanced effects of regulatory milestones on the financial performance of pharmaceutical companies and highlight the need for more comprehensive studies to further unravel these complex dynamics.

Review of the impact of the FDA's Fast Track Designation on biotechnology companies' share prices.

This study examines the effect of the FDA's Fast Track Designation (FTD) on biotech company share prices. Using an event-study approach on 25 FTD announcements between June 2019 and June 2020, notable short- and long-term share price hikes were observed, with a 5-day cumulative average abnormal returns of 21.59%, 30-day at 38.34%, 1-year at 76.64% and 3-year at 111.37% against the XBI benchmark. These surges surpass prior research findings, indicating stronger investor reactions. The role of the COVID-19 pandemic as a confounder is discussed. Although the sample size is limited, the results offer valuable insights for investors and small pharma firms. Further research is recommended to delve into the driving market factors.

Small pharma vendor management practices in clinical trials: A case study within Faron Pharmaceuticals.

Many small pharmaceutical companies find that they lack the resources, knowledge and expertise of the regulatory landscape for adequate vendor management in clinical trials, making the organization vulnerable. Recent research suggests that some pharmaceutical companies have found themselves out of compliance with ICH, FDA or EMA guidelines. This paper aims to perform a comprehensive review of the regulatory landscape for vendor selection, oversight and ongoing evaluation in clinical trials. In addition, the case study performed studies the practices recently implemented at small pharmaceutical company Faron Pharmaceuticals to assess regulatory compliance and identify any potential best practices. Faron Pharmaceuticals conducted a process improvement activity at the beginning of 2022 to improve the vendor selection, oversight and evaluation of their clinical trial partners. The results of this case study indicate that Faron Pharmaceuticals' processes are regulatory compliant, suggesting that QTLs, KPIs, SOPs and communication plans are effective vendor oversight mechanisms for small pharmaceutical companies to utilize.