(Photography: Jim Shive)
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Along with the growing requirements for postmarketing studies is a growing glossary of terms to describe Phase IV clinical
trials, patient registries, observational studies, and other research conducted after a treatment's approval. For simplicity,
this article will, for the most part, refer to these as "Late Phase" studies, which life science companies are increasingly
called on to conduct.
The demands of these studies are many, and include:lifecycle management of patented drugs; real world Phase IV trials following
accelerated drug approvals; long-term safety evaluations; stepped-up regulations in the United States, EU, Japan, and other
regions; and a need for effectiveness data for reimbursement by public and private payers. This leads to a major question
facing sponsors: How to navigate today's demands while containing costs?
Technology savvy life science companies are looking to technology for help. Many are assessing Web-based EDC, now widely used
in Phases I–III clinical studies. In addition to the obstacles to EDC adoption faced in earlier phases—ensuring system access,
high data quality, and regulatory compliance—Late Phase research presents additional requirements. Notably, it is most frequently
conducted in community settings as opposed to specialized academic institutions. But busy physician practices do not have
the infrastructure to learn complex technologies; instead, they require intuitive, easy-to-use data entry methods.
In addition, Late Phase research often involves smaller subject-to-site ratios, with data collected at a large number of sites
to ensure ability to generalize results. Hence, the costs for setting up, training, and supporting sites have to be lower
than in clinical development research—otherwise these Late Phase studies would be cost prohibitive. Finally, and also due to the need for real-world data reflective of actual treatment patterns, an increasing number of Late
Phase trials and registries are conducted on a global basis. As such, these studies require an ability to have a common database
across different countries and languages (allowing for minor local differences) to facilitate the logistics and analysis of
data.
These Late Phase requirements, often coupled with the need for long-term subject follow-up, once made automated data collection
at the site level cost prohibitive. Sponsors were stuck between the anvil and the hammer: Either simplify the research questions
at the risk of not learning anything of value, or take the risk of collecting ultimately inconclusive data (too dirty, hard
to pool, etc.).
It is the fundamental contention of this article that Web-based EDC technology is allowing sponsors to improve the quality
of the data collected in Late Phase research within acceptable budget constraints. The article will explore the challenges
and opportunities sponsors face as they embrace rapid change in Late Phase research and supporting EDC technology.
Key drivers of growth
Most sources suggest that Phase IV postmarket research is growing at around 20% annually, exceeding the growth rate of Phase
II and III studies, and projected to have cost the industry more than $12 billion worldwide in 2007.1 Regulatory authorities around the world face a highly sensitized public that is concerned about the safety of marketed drugs
and are seeking additional research from sponsors to evaluate real-world safety and effectiveness.2
Postmarketing studies also play an increasingly important role in overall lifecycle management to demonstrate the value of
new therapies after commercialization and provide insight into new market opportunities, new subject populations, new indications,
and competitive positioning. Similarly, public and private payers are also seeking more evidence-based research and outcomes
data in real-world settings to make formulary and reimbursement decisions.3
Despite regulatory intent to impose postmarketing obligations, a large number of committed studies have either not been conducted
at all or only carried out after significant delays—thereby defeating the very logic of early collection of real-world data
to identify unknown safety concerns. For example, in the United States, the number of postmarketing commitments (PMCs) required
by the FDA continues to increase by more than 75% of all New Chemical Entities (NCEs), partly spurred by increasing Fast Track
Approvals and requests for pediatric population use. Yet more than 700 commitments still remain unmet4 due to a wide variety of scientific or ethical reasons and operational and budgetary obstacles.
Technology—specifically EDC—is the cornerstone of a successful strategy to facilitate data collection and subject tracking
within reasonable cost.
