The Value of Innovation: Impact on Health, Life Quality, Safety, and Regulatory Research: Volume 16

Where is the Life we have lost in living?Where is the wisdom we have lost in knowledge?Where is the knowledge we have lost in information? Thomas Stearns Eliot.This book is a collection of multi-author contributed research devoted to exploring the synergy of new science-based health-risks approaches, innovative information technologies, and innovation in outcomes research. Time and again we find evidence that logistics of health care delivery calls for innovative approaches in every aspect of intervention, prevention, discovery, and insurance. Without transparency and real-time availability of adequate, multipurpose, reliable, health-risks and outcomes data, the needs of consumer safety, disease management, regulatory research, and targeted discovery will remain poorly equipped for either individual health system quality assurance or nation-wide decision-making. Each chapter in this collection strives to create knowledge by creating adequate data and advancing, at least, one step forward in the relevant field.

Newly marketed drugs present unavoidable risks, no matter how diligent the level of pre-market review. Numerous adverse drug events attest to the need for post-market vigilance. However, the Food and Drug Administration monitors drugs with considerably less rigor after launch than before. This burdens both public health and public trust in the safety of new medicines. As new technologies such as genomics guide a larger share of drug development, the issue will become more acute. Most reform proposals present considerable logistical challenges. A promising alternative is to harness existing managed care databases to search for drug effects.

We review the conceptualization of quality of life (QOL) past and present, providing a new definition that transcends the traditional approach. We discuss the importance of QOL as a mandatory assessment in patient care and clinical trials, concurring with the need for disease-specific tools and focusing on a nerve fiber-specific tool for assessing impacts of diabetic neuropathies on QOL and activities of daily living (ADLs) used in multi-center clinical trials and translated into different languages. By relating neuropathic disabilities to different nerve fibers, the Norfolk Quality of Life – Diabetic Neuropathy (QOL-DN) is able to measure impacts of nerve-fiber-specific neurotrophic therapies, providing pertinent endpoints to changes in health status and QOL.

Recent guidance from the United States Food and Drug Administration discusses patient-reported outcomes as endpoints in clinical trials (FDA, 2006). Using methods consistent with this guidance, we developed symptom indexes for patients with advanced cancer. Input on the most important symptoms was obtained from 533 patients recruited from National Comprehensive Cancer Network (NCCN) member institutions and four non-profit social service organizations. Diagnoses included the following 11 primary cancers: bladder, brain, breast, colorectal, head/neck, hepatobiliary/pancreatic, kidney, lung, lymphoma, ovarian and prostate. Physician experts in each of 11 diseases were also surveyed to differentiate symptoms that were predominantly disease-based from those that were predominantly treatment-induced. Results were evaluated alongside previously published indexes for 9 of these 11 advanced cancers that were created based on expert provider surveys, also at NCCN institutions (Cella et al., 2003). The final results are 11 symptom indexes that reflect the highest priorities of people affected by these 11 advanced cancers and the experienced perspective of the people who provide their medical treatment. Beyond the clinical value of such indexes, they may also contribute significantly to satisfying regulatory requirements for a standardized tool to evaluate drug efficacy with respect to symptomatology.

Confounding is of central importance in epidemiologic studies. Its definition has been under wide debate over the past decades. The classical definition is straightforward and easy-to-implement. Nevertheless, it is data-driven and has drawbacks. The more recent counterfactual definition captures the essential roles a confounder plays in causal inference. It would be beneficial for researchers to grasp substantive knowledge in causal structure and broadly adopt the latter definition. There are various methods of handling confounding issues. The choice of one option over another depends on various factors, including the nature of the study, sample size and rarity of events.

This chapter examines the role of pharmaceutical patents in the on-going support of pharmaceutical innovation. The social value of pharmaceutical innovation and the importance of its sustained growth are explained. The government buy-outs of patents to reduce drug prices for all American consumers while preserving vital drug innovation are proposed.

The relationship between patents and the pharmaceutical industry is both complex and important. While many believe that patents are manipulated by the pharmaceutical industry for its own economic ends, a historical approach to the utilization of patents combined with an analysis of current patent issues places this relationship in its proper context. Though patents were created long before the pharmaceutical industry reached its current status as a major industry in the United States, a historical, analytic approach shows that the pharmaceutical industry has adjusted to constantly evolving legislation enacted to provide the most effective and efficient system by which to research, invent, regulate and patent new medicines.

This study proposes targeted modernization of the Department of Defense (DoD's) Joint Forces Ammunition Logistics information system by implementing the optimized innovative information technology open architecture design and integrating Radio Frequency Identification Device data technologies and real-time optimization and control mechanisms as the critical technology components of the solution. The innovative information technology, which pursues the focused logistics, will be deployed in 36 months at the estimated cost of $568 million in constant dollars. We estimate that the Systems, Applications, Products (SAP)-based enterprise integration solution that the Army currently pursues will cost another $1.5 billion through the year 2014; however, it is unlikely to deliver the intended technical capabilities.

This chapter proposes an optimized innovative information technology as a means for achieving operational functionalities of real-time portable electronic health records, system interoperability, longitudinal health-risks research cohort and surveillance of adverse events infrastructure, and clinical, genome regions – disease and interventional prevention infrastructure. In application to the Dod-VA (Department of Defense and Veteran's Administration) health information systems, the proposed modernization can be carried out as an “add-on” expansion (estimated at $288 million in constant dollars) or as a “stand-alone” innovative information technology system (estimated at $489.7 million), and either solution will prototype an infrastructure for nation-wide health information systems interoperability, portable real-time electronic health records (EHRs), adverse events surveillance, and interventional prevention based on targeted single nucleotide polymorphisms (SNPs) discovery.

The U.S. healthcare system is very complex and inundated with many challenges, ranging from escalating healthcare expenditure to increasing prevalence of medication errors. Emphasis on cost reduction and prevention of medication errors has given impetus to the development of computerized information technologies. Advanced technological innovation such as e-prescribing has the potential to improve quality of care, reduce costs and enhance patient satisfaction. Reaping these benefits is dependent on successful diffusion of innovation. This chapter summarizes the current literature covering diffusion of e-prescribing technology with an emphasis on the barriers and facilitators, and strategies to overcome these potential barriers.

The applicability and shortcomings of a well-defined cost-estimating process to forecasting resources required for developing and fielding innovative technologies are examined. Whereas the process itself provides a suitable approach for estimating the cost of any program, investment is required for collecting historical data on analogous programs to serve as the foundation for the estimating methodologies. Particular challenges in costing innovation are summarized. An appropriate form of the cost probability distribution for research programs is offered.