Late Phase Studies: An Early Priority

By Dipti Amin, Quintiles

Late phase research has sometimes been viewed as less of a priority than the earlier trials required for product registration.

In the past, this has meant lost opportunities to expand product labels, demonstrate product value and monitor safety – and a limit to a biopharmaceutical company's ability to realize a product's full commercial potential.    

Today, biopharmaceutical companies are feeling pressure from numerous sources – demands of a competitive marketplace to develop drugs more cost-effectively and to squeeze revenue from them, combined with a new level of regulatory scrutiny surrounding post-marketing commitment studies. The demand for these studies puts a significant strain on companies to meet the regulatory requirements and manage the expense of complex protocols. However, this challenging environment also presents opportunities for innovators.

Visionary pharmaceutical and biotech companies are recognizing the opportunity to expand revenues at earlier stages in a product's lifecycle through greater investment in strategic late phase studies that are intelligent, cost effective and market savvy designs. The results of such trials bring valuable insight about real-world product use and clinical practice patterns.

In addition to the studies to expand the product's uses, pharmaceutical companies are also increasingly faced with post-marketing commitment studies, as a requirement for approval or continued marketing of their products. While the purpose of these studies is to comply with regulations and provide additional information about safety, effectiveness and optimal use, if approached strategically biopharmaceutical companies can also gather important and useful differentiating data about their product. 

There is great interest in post-marketing safety surveillance, driven by public demand for safer products and more transparency. This is reflected in increased regulatory emphasis on drug safety, such as the mandatory risk management plans (RMPs) in place in Europe, and the US FDA Risk Evaluation and Mitigation Strategies (REMS), Risk Minimization Action Plans (RiskMAPs), and authority over post-marketing surveillance.

The changing regulatory climate is making it necessary to collect better safety data to answer increasingly detailed questions about drug risks and perform more comprehensive analyses within a cost containment/reduction environment. Late phase research requires a specialized approach, including: strategic understanding of the study objectives and design options to achieve goals; knowledge of the complex nature of the results generated and how to maximize the data analysis during and after the trial; in-depth understanding of the operational issues involved in managing late phase studies; expertise in the disease or therapeutic area; global capabilities coupled with local market expertise; fit-for-purpose data management tools; and access to appropriate patients and investigators.

This approach yields quality data to satisfy regulators and payers. It provides validated assessment of safety and other outcomes in real-world settings. These multiple, fit-for-purpose data analyses, including comparative effectiveness, can enhance publication output and reach more prescribers. Implementation at the earliest possible stage in the product lifecycle can provide a welcome boost to product revenues at a time when the cost of developing new products is at an all-time high.

Dipti Amin is the Senior Vice President and Global Head of Quintiles Late Phase and Safety Services unit. Educated in the United Kingdom, Amin earned professional degrees through GKT Medical School of the University of London. She earned graduate degrees and Diplomas in medicine, surgery, family planning, obstetrics and gynecology, child health and clinical pharmacology.

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