A framework for pharmaceutical value-based innovations.

The rapidity of change and increasing complexity of today's healthcare environment dictates that pharmaceutical companies refine their drug development process to ensure that products provide maximal value to consumers. Innovation should receive the support and encouragement it deserves but the balance between cost of therapy and enhanced benefits to the end users, the patients, must not be compromised. The health or quality-of-life outcomes that therapies are likely to achieve need to be clearly stated for patients so they know what to expect before beginning treatment. A drug development program that incorporates principles of 'patient-centered medicine' early on can help define the true value of a health technology. This demands not only demonstrating value as measured by actual patient experience and patient-reported outcomes, but understanding the unmet needs of multiple stakeholders: what is their perception of what represents value? What factors impact healthcare coverage decisions? In this paper, we describe an illustrative framework designed to weave 'patient-centric' medical and real-world evidence into the phases of research and development. This evidence and value development framework works iteratively, providing useful insights parallel to product development from early phase to life-cycle management while forming a progressive evidence chain throughout the phases of research and development. This in turn leads to the creation of value-based innovation with robust foundational evidence for clinical decisions, postlaunch coverage and reimbursement evaluations.

Integrating scientific and real-world evidence within and beyond the drug development process.

Newly developed healthcare treatments face a complex environment with many stakeholders who can accelerate or decelerate adoption, most notably the healthcare system payers. Understanding and integrating their needs earlier in clinical development will ensure a smoother transition from bench to bedside. This paper describes a new approach to shaping a more effective complementary process of 'value' evidence generation both in and outside the clinical drug development process. We propose that biopharmaceutical companies consider bringing new solutions to market by marshaling cross-functional approaches to what we term an evidence-definition phase, evidence-generation phase and evidence-translation phase to drug and technology research and development. The organization of ongoing discovery, evaluation and translation with a 'real- world' perspective should provide a more streamlined approach to ensure both regulatory and eventual marketplace success.

Integrating new approaches for clinical development: translational research and relative effectiveness.

Translational research and relative effectiveness are being incorporated into drug development programs to meet the demands for more robust evidence generation to support the value of new therapies. Translational research includes translating basic research into clinical practice, controlled clinical trials into potential clinical implications, evidence-based guidelines into routine clinical practice and standard practices into population health. These research concepts link with real-world outcomes, and feed into each other to improve the efficiency of research. Translational research can run into road blocks in terms of conveying the added or comparative value of research or during adoption into clinical practice. Understanding these roadblocks and developing solutions are important for success. Comparative effectiveness research can be a useful research technique to accomplish many translational medicine goals. These studies generally include heterogeneous patient populations and evaluate outcomes of relevance to payers and health technology assessors. Comparative effectiveness research can be used in drug development; different methodologies may be useful in different phases. In this article, suggestions and examples of successful use of comparative effectiveness studies are provided. Translational research and comparative effectiveness research, although clearly independent concepts, can provide a focused approach to drug development, resulting in products entering the market that bring added benefit to patients and the healthcare system overall.

[Design and birth of drugs]. / Conceptions et naissances des médicaments.

Today the design of new clinical entities uses highly sophisticated techniques. This design is a step by step procedure: first selection and validation of the therapeutic targets, where the contribution of genomics is very important; then, setting up the different screening tests. This step permits to detect the potentially active molecules, which will be optimised, before being tested in healthy volunteers and then in patients.