Research & Development

Research & Development

For more than a century, our company has been inventing medicines and vaccines for many of the world’s most challenging diseases. We embrace our responsibility to address the health needs of patients and society by inventing for life through world-class science.


We are committed to addressing unmet medical needs through innovative research and development (R&D). R&D expenses were $10.2 billion in 2017, $10.1 billion in 2016, and $6.7 billion in 2015 (which included restructuring costs and acquisition- and divestiture-related costs in all years). The talent of our scientists, combined with scientific and technological advances that enable the rapid invention of expanding classes of therapeutics and higher resolution translational medicine studies, are transforming the way we conduct research.

Our pipeline programs are prioritized based on medical need, scientific opportunity and commercial potential.

We are committed to advancing the most promising research and clinical development strategies to bring forward new medicines and vaccines that will make a meaningful difference in patients’ lives.

For instance, we are able to develop biomarkers—those characteristics that can be objectively measured and evaluated as indicators (or markers) of normal biologic processes, disease processes or responses to therapy. Since biomarkers provide critical information in the drug discovery and development processes, our intent is to apply them very early in the development of novel therapeutic candidates to enhance the efficiency of drug development by providing preliminary evidence of a drug candidate’s potential benefit before proceeding with further research.

The use of biomarkers is one part of our integrated approach to drug development. Coupled with the application of novel quantitative approaches that harness the power of mathematical modeling to analyze preclinical experiments to inform our clinical trial designs, we can optimize the next phase of clinical trials and, importantly, make pivotal decisions earlier and more confidently, increasing productivity and the efficient use of our resources. By eliminating likely failures sooner and focusing on those mechanisms that appear more promising, we believe we can bring innovative products to patients faster and more efficiently, while still maintaining a rigorous focus on scientific excellence and patient safety.

We recognize that real-world evidence has an increasing role in how medicines and vaccines are developed and evaluated. Through the contributions of epidemiologists, health economists, health outcomes researchers, data specialists and health-policy researchers across our company, we strive to comprehensively assess the best available information on the value of our medicines and vaccines that we develop and commercialize.


To address unmet clinical needs in children, we include pediatric development programs in the company’s new drug and vaccine development strategies worldwide.

When appropriate, we will develop and seek approval for pediatric indications and develop age-specific formulations. We rely on an internal Pediatric Development Committee that pools pediatric expertise across disciplines and therapeutic areas to review and provide input into pediatric development strategies. The Committee serves as a Center of Excellence within our company to consult on pediatric development issues and key pediatric policy questions.

For example, ISENTRESS® (raltegravir), our integrase inhibitor, in combination with other antiretroviral agents, is approved for the treatment of HIV-1 infection in pediatric patients weighing at least 2 kg. In addition, EMEND® (aprepitant) capsules can now be used in patients 12 to 17 for the prevention of acute and delayed nausea and vomiting associated with cancer chemotherapy.

Our Pipeline

We prioritize our R&D efforts on advancing candidates that we believe represent the next areas of breakthrough science that will make a difference and deliver value for patients, physicians and payers.

To develop important new products that improve the quality of life and satisfy customer needs, our company is poised where invention and the burden of disease meet. Our products and research priorities are aligned with the current and projected global burden of disease as defined by the World Health Organization (WHO), as well as with the increasing need for new therapies targeted at diseases such as cancer, HIV/AIDS, Alzheimer’s disease and antibiotic-resistant infections, as well as in disease prevention through vaccine development.

Our research pipeline illustrates the progress of our R&D efforts. An update on our R&D activities can be found in our Form 10K or on our corporate website.

External Collaboration

We support academic and community-based physicians and researchers in expanding clinical and scientific knowledge, and in improving understanding of the appropriate use of our products.

Our Investigator Studies Program exemplifies our efforts to advance science and improve patient care. Through this program, we provide medicines, vaccines and/or funding for peer-reviewed research that is initiated, designed, implemented and sponsored by external investigators. This program fosters collaboration with researchers throughout the world who are active in emerging research areas of interest, and has established a track record for scientific exchange through presentations and the publication of findings in peer-reviewed journals.

We are active participants within the scientific community providing support to many professional associations, including the American Association for the Advancement of Science, the U.S. National Institutes of Health, the U.S. National Science Foundation (NSF), the World Medical Association and the Council for International Organizations of Medical Societies. In addition to promoting dialogue and the exchange of ideas in research, we sponsor research conferences—such as selected Gordon Research Conferences, an international forum in which researchers discuss advances in biologic, chemical and physical science.


We recognize that individuals or companies cannot successfully develop drugs single-handedly. Most cases of true innovation come from robust and honest collaboration among individuals and organizations with diverse backgrounds and capabilities, brought together by the idea of changing the course of human health.

As part of our R&D strategy, we pursue opportunities to establish external alliances that complement our internal research capabilities, including research collaborations and licensing agreements for preclinical and clinical therapeutic candidates that have the potential to drive both near- and long-term growth.

We recently expanded our agreement with Eiger BioPharmaceuticals (Eiger) to include the commercial and distribution rights for the treatment of Hutchinson-Gilford Progeria Syndrome, a rare and fatal genetic condition characterized by accelerated aging in children. Eiger will provide the investigational candidate lonafarnib for ongoing clinical trials and expanded access in Progeria and be responsible for any potential filing of an NDA for the Progeria indication based on The Progeria Research Foundation (PRF) data. Our company will not receive any milestone payments for the development of lonafarnib for the treatment of Progeria, and has waived royalty obligations from Eiger for a specified quantity of lonafarnib, estimated to supply the worldwide population of children with Progeria on an annual basis. Concurrently, Eiger announced that it has completed a collaboration agreement with PRF.

Innovative Medicines Initiative—Accelerating Research
Within Europe, we participate in a number of Innovative Medicines Initiative (IMI/IMI2) projects. IMI, Europe’s largest public-private initiative aiming to speed drug development and improve safety, supports collaborative research projects and builds networks of industrial and academic experts to boost pharmaceutical innovation.

Drug Discovery Collaborations

One recently completed project, DRIVE-AB (Driving Re-investment in research and development for antibiotics and advocating their responsible use) brought together a consortium of 23 partners including pharmaceutical companies, academic institutions, and public health organizations, to develop new economic models and recommendations to promote antibiotic innovation and the sustainable use of the resulting, novel antibiotics.

ADAPT SMART (Accelerated Development of Appropriate Patients Therapies, a Sustainable, Multi-stakeholder Approach from Research to Treatment-outcomes),  is another project that we recently concluded. ADAPT SMART is an enabling platform for the coordination of Medicines Adaptive Pathways to Patients (MAPPs) activities. MAPPS seeks to foster access to beneficial treatments for the right patient groups with high unmet medical needs at the earliest appropriate time in the product life-span in a sustainable fashion. A key component of MAPPs is stakeholder collaboration throughout development and market access in an iterative manner. We recently started a new project called PARADIGM that is co-led by the European Patients’ Forum and Industry and aims to advance meaningful patient engagement in the life cycle of medicines for better health outcomes.

Biomarker Collaborations

Accelerating Medicines Partnership—New Diagnostics & Therapies
We are a member of the Accelerating Medicines Partnership, a venture among the National Institutes of Health, 10 biopharmaceutical companies and several nonprofit organizations to transform the current model for developing new diagnostics and treatments by jointly identifying and validating promising biological targets of disease. The ultimate goal is to increase the number of new diagnostics and therapies for patients and reduce the time and cost of developing them.

Biomarkers Consortium—Biomarker-Based Technologies
The Biomarkers Consortium, in which we participate, is a public-private biomedical research partnership managed by the Foundation for the National Institutes of Health (FNIH). To date, the Biomarkers Consortium has launched 10 projects in areas as diverse as Alzheimer’s disease, cardiovascular disease, osteoporosis and breast cancer. A number of other promising projects are also moving toward implementation. Our company contributes to a number of FNIH projects within this consortium.

Multi-Regional Clinical Trials Center of Brigham and Women’s Hospital and Harvard
We are a participating member of the Harvard Multi-Regional Clinical Trials (MRCT) Center. The MRCT Center develops guidance, training resources and tools that promote safe and ethical clinical trials. We perform our work by convening representatives from industry, not-for-profit organizations and academia, as well as investigators, patients and patient advocacy groups, to create practical resources for the ethical design and conduct of multiregional clinical trials.

The MRCT has been working with global leaders to promote regulatory convergence and internationally accepted best practices; developing guidance and tools to promote safe and ethical trials; and training regulatory and clinical trial professionals with a particular focus on low- and middle-income countries. New programs focus on increasing diversity in clinical trial enrollment and study conduct, and a project to evaluate the utility and limitations of observational data.

National Institutes of Health Alzheimer’s Disease Neuroimaging Initiative 
We continue to participate in the National Institutes of Health Alzheimer’s Disease Neuroimaging Initiative (ADNI), the largest public-private partnership in Alzheimer’s disease research. This study, which is designed to gain new insights into the onset and progression of Alzheimer’s disease, has now expanded to ADNI3, with the goal of improving clinical trial design and aiding drug development. ADNI3 will seek to identify and track early changes in the brain before the onset of Alzheimer’s symptoms by using imaging techniques, including the newly developed tau PET ligands, and biomarker measures in blood and cerebrospinal fluid.

Predictive Safety Testing Consortium—Safety-Testing Methods
We are a member of the Predictive Safety Testing Consortium (PSTC), a unique public-private partnership led by the nonprofit Critical Path Institute (C-Path). The PSTC brings together pharmaceutical companies to share and validate their safety-testing methods under the advisement of the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA).

The 18 corporate members of the consortium share internal experiences with preclinical and clinical safety biomarkers in kidney, liver, skeletal muscle, testicular toxicity, vascular injury and cardiac hypertrophy. All biomarker research programs have a strong translational focus to select new safety tools that are applicable across the drug-development spectrum. Advancing the science and use of biomarkers in drug development is a critical area of focus for our company.

The following are notable PSTC achievements:

  • The FDA and EMA qualified seven new urine tests that signal kidney injury
  • The PSTC opened a biomarker-qualification process with the FDA for new biomarkers of drug-induced liver and skeletal muscle injury
  • The Japanese Pharmaceuticals and Medical Devices Agency qualified new biomarker laboratory tests that signal kidney injury


Clinical Trials Transformation Initiative/Duke University—FDA
We continue to focus efforts on improving the way in which clinical trials are recruited and conducted. To further those efforts, we are an active member of the Clinical Trials Transformation Initiative (CTTI), a public-private partnership led by FDA and the Duke Clinical Research Institute. CTTI works with industry and academic partners to identify and promote practices that will increase the quality and efficiency of clinical trials, and will foster a high-quality clinical trial system that is patient-centered and efficient, enabling reliable and timely access to evidence-based prevention and treatment options. Recent CTTI projects have focused on patient-informed consent processes and standards, Investigational New Drug safety reporting and on best practices for Data Monitoring Committees.

We remain committed to developing partnerships and ideas to help improve care and access to medicines around the world. Read more about Business Development & Licensing.

Industry Consortia

We also collaborate with external researchers and other members of the biopharmaceutical industry through participation in selected scientific consortia. Consortia are an important mechanism by which researchers can work together in a precompetitive manner to address complex scientific challenges common to all parties.

TransCelerate BioPharma Inc.
We are an active member of TransCelerate BioPharma Inc. (TransCerelate). TransCelerate is a nonprofit organization with the mission to collaborate across the global biopharmaceutical research and development community to identify, prioritize, design and facilitate implementation of solutions designed to drive the efficient, effective, and high-quality delivery of new medicines.

TransCelerate works in three strategic priority areas:  Improving the Patient and Site Experience, Harmonizing Process & Sharing Information, and Enhancing Sponsor Efficiencies & Drug Safety. Our colleagues contribute across all strategic priority areas in a broad range of responsibilities. These include serving on the Board of Directors, Oversight Committee and Pharmacovigilance Steering Committee, as well as both leading and contributing roles within the portfolio of projects.


The stated objective of the Compliance Committee Charter within our research laboratories is to ensure ongoing compliance with applicable laws and requirements in all business areas through appropriate management structure, processes and training.

In order to manage compliance, the Compliance Committee is composed of members of the Research Leadership Team. As a result, compliance efforts encompass the entire division and go beyond simply addressing the conduct of clinical trials.

The Compliance Committee also promotes ethical science and provides guidance to our employees within the research organization on our company’s standards and corporate policies, as well as necessary education related to specific requirements applicable to the research community.


RESEARCH & DEVELOPMENT2013 2014 2015 2016 2017
Research and development expenses (in billions) $7.5 $7.2 $6.7 $10.1 $10.2
Employees involved in research activities 12,300 11,400 11,900 12,300 12,700
New products approved1 0 7 2 3 4
Products in the pipeline and under regulatory review35 33 31 39 26
Top 20 global burdens of illness addressed by our products and pipeline2 88% 88% 88% 88% 88%
Established significant external licenses and collaborations40 35 64 57 55
Filed U.S. patent applications 159 125 185 195 190
1. Candidates in our company’s research pipeline or under regulatory review as reported in the United States Securities and Exchange Commission Form 10-K, page 6–7, filed on February 27, 2018. Approval of new products only. This does not include approvals for supplemental indications. When candidates attain regulatory approval they are removed from this pipeline view.
2. As defined by the Institute for Health Metrics and Evaluation (IHME) using GBD2015 data.