By Martin Young
For decades, the pharmaceutical industry has thrived upon a business model that focused on the production of blockbuster drugs aimed at large patient populations with high sales potential. This established development and sales paradigm has been evolving in a new direction over the last several years as the market opportunities for blockbuster drugs become scarcer.
Greater emphasis is now being placed on targeting drugs to smaller patient populations and bringing a greater number of products to market.
Shifting R&D focus away from the blockbuster model, in fact, necessitates that organizations bring more drugs to market if they are to continue to grow and remain competitive. Recently, we have witnessed new partnerships and mergers between large pharmaceutical firms, small to midsize pharma and biotech companies, and academic institutions to bolster development pipelines and provide access to new molecular entities and research tools. While this is an effective short-term plan, it is ultimately a zero-sum strategy, and organizations will need to retool their development processes if they are to successfully maneuver this industry transformation.
Regulatory requirements are consistent regardless of the size of the intended patient population, which keeps the cost of bringing a new drug to market relatively the same. As one might imagine, testing more drugs and running more trials concurrently increases overall R&D costs and creates additional resource challenges. A key requirement for any new development model, then, is increasing R&D efficiency and output.
One of the trends supporting this new model has been the increase in strategic outsourcing and externalization of drug development with CROs. Outsourcing and leveraging third-party expertise and resources can be a cost-effective way to manage the clinical development process. Sponsors can focus on their own core competencies rather than manage the entire trial process themselves.
Outsourcing has become a more viable option as new technologies provide higher quality information and give more visibility into processes and data. Trial sponsors can confidently farm out a portion of their development while maintaining operational visibility and management control, allowing them to make better decisions.
Regardless of whether an organization leverages external relationships for research or conducts most of its clinical trials in-house, advances in technology hold the key to streamlining drug development processes and increasing productivity and capacity.
The Gateway Technology: Moving To Maturation
Electronic data capture (EDC) has been a significant innovation that has helped drug companies achieve greater efficiency. EDC systems automate the collection and validation of clinical trial data in-stream to explore the efficacy and safety of a drug. This technology facilitates near real-time access to clinical study data, which is essential to lowering costs and reducing downtime between pipeline introductions.
According to a 2010 Health Industry Insights report, the number of Phase II and III trials using EDC is approaching the 80% mark. There seem to be few, if any, obstacles to widespread EDC adoption for clinical research.
EDC, however, is not a panacea for what ails the industry. Today’s clinical trials are more complex and longer than they were just several years ago and require the management of much larger volumes of data. Some of this complexity comes from the interwoven pressures on the pharmaceutical industry described above as well as the increasing global nature of many clinical trials, but it also stems from the success of EDC itself. As users became more comfortable with the technology, they were able to apply its efficiencies to new areas of clinical development and accommodate new data sources. So while EDC has revolutionized data collection in clinical studies, it also paved the way for other technologies that have the potential to redefine clinical and safety research.
The Clarion Call Of Automation
Trial sponsors have increased their use of technology in almost all areas of clinical development, including patient recruitment, drug supply chain management, study design development, patient reported outcomes, and pharmacovigilance, among others. There are a myriad of tools employed — some developed in-house, others by third parties — but common to them all is the aim to automate processes and streamline workflow. While not all of these tools will be as universally adopted as EDC (some, however, are well on their way), these efforts should help optimize the course of bringing drugs to market and help the industry realize new R&D efficiencies.
Interactive response technology (IRT) solutions (or IVRS/IWRS) automate and centralize the overall supply chain for randomizing subjects and dispensing medication kits, which helps companies better manage their inventories and keep costs under control. This ability is particularly important in large-scale studies with thousands of patients where drug waste can be a costly overhead, as well as in small biotechnology studies where the cost of the biopharmaceuticals used can be very expensive. The randomization process — associating patients with particular treatment arms, assigning them patient IDs, and issuing them the right medication kit — is a critical process required for the majority of Phase II and III studies and, before IRT, was labor-intensive because it relied on spreadsheets and stickers.
Study design tools — which dovetailed out of EDC use — leverage standards and templates to simplify the process of building a study. Designers can reuse study components, such as forms, code lists, and rules, from one study and apply to another study with similar requirements. The process of building a study can be a time-consuming process, and actual research cannot begin until all the stakeholders —data managers, biostatisticians, site staff, etc. — agree the design satisfies all requirements. Abbreviating the duration before a trial can go live offers significant value to trial sponsors.
The growing need for patient-reported outcomes (PRO) in all aspects of drug development has placed greater emphasis on the quality of data gathered directly from subjects. Paper-based collection methods are costly and logistically challenging and can discourage patients from continuing to participate in the trial. Electronic PRO (ePRO) is a more efficient and better way to capture patient outcomes, collecting data either through a PDA device or directly over the Internet on any computer. With ePRO, organizations receive more accurate data and better visibility into the collection and analysis processes, improving the analysis and submission processes.
Heightened regulatory oversight and business needs have also magnified the need to improve patient safety and manage risk more proactively throughout the duration of a trial (as well as during postmarketing studies). Organizations are looking to identify and report potential safety problems quickly and efficiently. A host of pharmacovigilance solutions provide adverse event reporting, data mining, signal detection and evaluation capabilities, and tracking of safety issues to trial sponsors. These tools give better insight into data and allow safety professionals to make product decisions based on a much broader range of empirical data than previously possible.
The Data Management Nightmare And The Integration Cure
While all these tools have streamlined various individual processes and are helping the pharmaceutical industry achieve greater R&D efficiency, taken as a whole they have the potential to create a complex layer of data management (as well as vendor management) that could derail any of the efficiencies gained. Employing and integrating a multitude of disparate point solutions from different vendors can be a daunting task.
An integrated suite of software that can automate the management of the entire clinical development process — from study setup through regulatory submission and postapproval trials — from a single partner will help reduce total cost of ownership. A suite requires less integration management and comes with inherent delivery efficiencies, which are particularly important as pharmaceutical companies try to find additional ways to cut costs.
The amount of electronic information generated to bring a new drug or device to market continues to grow, and controlling this information from multiple collaborators, while maintaining an audit trail, is extremely difficult. Integrating data alone is not enough. Workflow integration must also be accomplished. When there is workflow integration, the process of linking data to the downstream analysis and reporting processes becomes much more efficient and gives better control to study management. For a suite to be truly viable, then, there needs to be a central data repository to transform and store the collected clinical data, as well as a statistical control environment that can automate the statistical analysis, reporting, and submission processes.
Experience has shown numerous other instances where industries have relied upon particular applications to automate business processes and ultimately pushed the evolution of the point solutions employed into a software suite. Financial systems and enterprise resource planning (ERP) software suites evolved from individual solutions into integrated systems to great success, reducing cycle times, increasing productivity, and resulting in a significant reduction in total cost of ownership. The same thing appears to be occurring in the pharmaceutical industry, and new levels of efficiencies and safer drugs should be realized as these organizations reinvent themselves and harness the full capability of the technology available.
About The Author
Martin Young is senior VP of integration and product strategy at Phase Forward. He has more than 20 years of technology and pharmaceutical experience, focusing on the implementation of new technology applications to improve pharmaceutical companies' clinical development effectiveness.