Access to Health

Access to Health

Research & Development

Research & Development
Access to Health
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Our passion is improving health. That is what keeps us at the forefront of scientific discovery and innovation. Applying breakthrough science to develop meaningful new medicines and vaccines is our legacy.

Our Commitments

  • We will evaluate and reflect, where appropriate, the needs of emerging markets in the R&D of our products
  • We will conduct our clinical trials, including trials in Low Income and Middle Income countries, in accordance with the global standards of Good Clinical Practices, applicable local regulatory requirements and following the ethical principles that have their origin in the Declaration of Helsinki
  • We will collaborate with diverse partners to expand our R&D capacity to address unmet needs, including those in emerging markets and least-developed countries
  • We will pursue opportunities to provide access to compound libraries and molecules to spur development of new products

We are committed to addressing unmet medical needs through innovative research and development: R&D expenses were $6.7 billion in 2015. The talent of our scientists, combined with recent scientific and technological advances, is leading to an exciting period for research as we seek new and more effective ways to treat diseases.

Pediatric R&D

We are including pediatric development programs in the company’s new drug and vaccine development strategies worldwide, where relevant, in response to unmet clinical needs.

Further, where appropriate, we will develop and seek approval for pediatric indications and develop age-specific formulations. We utilize an internal Pediatric Development Committee to review and provide input into all pediatric development strategies across various therapeutic areas. The committee serves as a Center of Excellence within our company to consult on pediatric development issues and key pediatric policy questions. For a listing of all clinical trials, including pediatric clinical trials, click here.

Our R&D model is designed to increase productivity and improve the probability of success by prioritizing resources based on scientific opportunity, medical need and commercial potential.

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

Faced with the complex challenges of bringing important new therapies to patients while simultaneously controlling the rising costs of innovation, we are using important new preclinical, clinical and quantitative tools to help us rapidly differentiate between developmental candidates that will clearly meet patient needs and those that will not. A focus on genetics, genomics and translational medicine is also critical to these efforts, for instance, by enabling us to develop biomarkers—those characteristics that can be objectively measured and evaluated as indicators (or markers) of normal biologic processes, disease 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 provide preliminary evidence of their potential benefit before proceeding with further development.

In addition, we are using novel quantitative approaches to analyzing preclinical experiments to inform our clinical trials and to develop models based on published literature. By integrating our knowledge from these sources, we can develop mathematical models that allow us to explore possible clinical trial scenarios. We now have the capability to first simulate a trial thousands of times, exploring the impact of different factors that influence a specific disease and/or patient population, and the related efficacy and safety of responses.

With this integrated approach we can optimize the next phase of clinical trials and, importantly, make pivotal decisions earlier and more confidently, increasing productivity and improving the probability of success. By eliminating likely failures sooner and focusing on those mechanisms that appear more promising, we believe we can bring innovative products to patients faster, while still maintaining a rigorous focus on scientific excellence and safety.

We recognize that real-world data have a significant impact on how medicines and vaccines are developed and evaluated. Through the contributions of epidemiologists, health economists, outcome researchers, data specialists and other health-policy researchers across our company, we strive to comprehensively assess the best available information on the value of our medicines and vaccines.

Our Pipeline

Our company prioritizes its research and development 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.

This focus includes candidates that target many of the world’s most urgent global health challenges, such as our immuno-oncology program, including KEYTRUDA® (pembrolizumab), which is being studied in multiple oncology indications; a BACE inhibitor for Alzheimer’s disease (MK-8931) and an investigational HIV medicine (doravirine). We pursue therapies in a variety of modalities, including small molecules, vaccines and biologics.

Our company is prominently positioned at the intersection of invention and the burden of disease. 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 to treatment-resistant diseases such as hepatitis C and antibiotic-resistant infections.

Our research pipeline illustrates the progress of our R&D efforts. We currently have a number of candidates under regulatory review in the U.S. and internationally. An update on our R&D activities can be found in the company’s 10-K Report.

External Outreach

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

Our Investigator Studies Program is an example of our efforts to advance science and improve patient care by supporting, through the provision of drugs and vaccines and/or total or partial funding, high-quality research that is initiated, designed, implemented and sponsored by external investigators. Results are documented and properly disseminated in peer-reviewed publications.

Our company is a member of and supports numerous professional associations, including the American Association for the Advancement of Science (AAAS), the U.S. National Institutes of Health (NIH), the U.S. National Science Foundation (NSF), the World Medical Association (WMA) and the National Academies. 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—that cover areas in which our company is conducting research.

We believe in broad disclosure of financial relationships between physicians and the pharmaceutical industry. We comply with the Physician Payments Sunshine Act provisions of the U.S. Affordable Care Act, which requires pharmaceutical manufacturers to annually disclose information on certain additional payments and other transfer of value furnished to U.S. licensed physicians and U.S. teaching hospitals to the Department of Health and Human Services (HHS). In addition to submitting such information to HHS’s Center for Medicare & Medicaid Services each year, we post the information annually on this website. Visit the transparency disclosures section of the report for more information and a full list of disclosures.

We also collaborate with external researchers and other members of the pharmaceutical industry by participating 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. These consortia are typically in the form of public-private partnerships.

Public-Private Research Partnerships

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 to complement our substantial internal research capabilities, including research collaborations, as well as license agreements for preclinical and clinical compounds that have the potential to drive both near- and long-term growth. In this regard, our research laboratories established significant external alliances to advance drug discovery and development, improve R&D productivity, and successfully commercialize novel therapeutics and vaccines.

Accelerating Medicines Partnership (AMP)—New Diagnostics & Therapies
We are a member of the Accelerating Medicines Partnership (AMP), a venture between the National Institutes of Health (NIH), 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.

AMP will begin with three-to-five-year pilot projects in the three disease areas of Alzheimer’s disease, type 2 diabetes, and autoimmune disorders of rheumatoid arthritis and systemic lupus erythematosus (lupus). Our company is contributing to the type 2 diabetes and rheumatoid arthritis/lupus projects.

Bayer—soluble guanylate cyclase (sGC) modulators
Initiated in 2014, we are continuing to make substantial progress in our development and commercialization partnership with Bayer for soluble guanylate cyclase (sGC) modulators, a novel class of compounds affecting the nitric oxide pathway with promise in various cardiovascular indications.

At this time, the partnership includes two compounds, riociguat and vericiguat. Riociguat (ADEMPAS®), already approved for the treatment of primary pulmonary hypertension (PAH) and pulmonary hypertension secondary to chronic thromboembolism (CETPH), is subject to various Phase IV life-cycle management studies as well as Phase II indication expansion studies to further explore the potential antifibrotic benefit in other orphan diseases.

The development of vericiguat focuses on its potential utility in the treatment of patients with worsening chronic heart failure. Setup as a development partnership for compounds in Phase 2 and beyond, the discovery and early development teams from both companies continue to explore additional sGC modulators for potential future inclusion in the collaboration to exploit the potent vasodilatory and antifibrotic properties in various cardiovascular and non-cardiovascular areas of huge unmet need.

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). Its goal is to combine the forces of the public and private sectors to accelerate development of biomarker-based technologies, medicines and therapies for the prevention, early detection, diagnosis and treatment of disease. Working together, the members of the Biomarkers Consortium are building uniquely powerful collaborations that are accelerating the development of biomarker-based technologies, medicines and therapies.

To date, the Biomarkers Consortium has launched 10 projects in areas as diverse as Alzheimer’s disease, cardiovascular disease 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.

cCAM Biotherapeutics (acquisition)
In July 2015, we announced acquisition of cCAM Biotherapeutics, a privately held biopharmaceutical company focused on the discovery and development of novel cancer immunotherapies.

The acquisition provides our company with several early immunotherapy candidates including cCAM Biotherapeutics’ lead pipeline candidate, CM-24─a novel monoclonal antibody (mAb) targeting the immune checkpoint protein CEACAM1 that is currently being evaluated in a Phase 1 study for the treatment of advanced or recurrent malignancies including melanoma, non-small-cell lung, bladder, gastric, colorectal and ovarian cancers.

Clinical Trials Transformation Initiative (CTTI)/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. Current working groups are focused on patient-informed consent processes and standards, on Investigational New Drug (IND) safety reporting, and on best practices for Data Monitoring Committees.

GE Healthcare—Alzheimer’s Disease
We are aggressively advancing research on Alzheimer’s disease through projects with imaging and diagnostic companies to help determine which patients may benefit most from novel treatments in development.

We have a clinical study collaboration, license and supply agreement with GE Healthcare for the use of Flutemetamol, an investigational positron emission tomography (PET) imaging agent, to support our company’s development of MK-8931, a novel oral beta amyloid precursor protein site cleaving enzyme (BACE) inhibitor and our lead investigational candidate for Alzheimer’s disease.

Accumulation of beta amyloid in the brain is a pathological characteristic related to Alzheimer’s disease. Currently, Alzheimer’s disease has typically been diagnosed by clinical examination (i.e., medical history, physical, neurological, psychiatric and neuropsychological exams; laboratory tests; and magnetic resonance imaging [MRI] or computed tomography [CT] scans). A diagnosis of Alzheimer’s disease was primarily confirmed through histopathological identification of characteristic features, including beta amyloid plaques, in post-mortem brain samples. There has been a serious unmet need for a reliable method for measuring beta amyloid deposits to help physicians diagnose Alzheimer’s disease. With the recent approval of several amyloid PET ligands, amyloid PET imaging is increasingly being used to diagnose Alzheimer’s disease, particularly for subjects early in the disease.

Harvard Multi-Regional Clinical Trials (MRCT) Center
We are a participating member in the Harvard Multi-Regional Clinical Trials (MRCT) Center working group, and have partnered with MRCT to develop a template for return of results for our company’s clinical trials based, in part, on EMA requirements.

The Harvard MRCT Center’s mission is to improve the design, conduct and oversight of multiregional clinical trials, focusing on trials sited in or involving the developing world; simplifying research through the use of best practices; and fostering respect for research participants, efficacy, safety and fairness in transnational, transcultural human subject research.

Innovative Medicines Initiative (IMI)—Accelerating Research
Within Europe, we participate in a number of Innovative Medicines Initiative (IMI) 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.

An ongoing IMI project centers on collaboration with the European Patients’ Academy on Therapeutic Innovation (EUPATI) by developing standards and trainings for patient advocacy group leaders. EUPATI launched the in-depth Patient Expert Training Course in October 2014 and in January 2016, launched a web-based educational toolbox, hosting educational material in English, Italian, Spanish, Polish, German, French and Russian aiming to reach 12,000 patient advocates across Europe.

Moderna—Messenger RNA-based Antiviral Vaccines and Passive Immunity Therapies
In January 2015, we announced an agreement with Moderna for the discovery and development of vaccines and passive immunity treatments against viral diseases using modified messenger RNA (mRNA). Moderna is a pioneer in the development of mRNA Therapeutics™ across a range of therapeutic applications. Moderna’s work in the collaboration will be led by Valera, its venture focused on the development of mRNA vaccines and therapeutics to fight infectious disease. Their exciting preclinical research shows the ability of mRNA to express viral antigens in vivo to induce a robust and protective immune response. By combining the benefit of Moderna’s upcoming data from their first clinical trials and our company’s strength in vaccines and antiviral development, we hope to transform the development of vaccines and therapeutics against viral diseases.

The three-year research collaboration (with the possibility of a one-year extension) is focused on the development of new mRNA-based treatments and vaccines against four undisclosed viruses.

NGM Biopharmaceuticals, Inc.—Strategic Collaboration to Discover, Develop and Commercialize Novel Biologic Therapeutics
The partnership, announced in February 2015, combines NGM’s unique biology-focused research platform with our company’s late-stage clinical and commercialization strengths to discover medicines for serious diseases.

The collaboration, initially planned for five years, includes multiple drug candidates currently in preclinical development at NGM, including NP201, which is being evaluated for the treatment of diabetes, obesity and nonalcoholic steatohepatitis (NASH).

National Academies
We continue to partner with the National Academies, an independent, nonprofit organization that works outside of government to provide unbiased and authoritative advice to decision makers and the public. We are a member of the Roundtable on the Promotion of Health Equity and the Elimination of Health Disparities. We are also actively seeking opportunities to improve the representation of racial and ethnic minorities in clinical trials.

In addition, our company worked closely with the National Academies and a range of other global organizations in the development of the Sharing Clinical Trial Data: Maximizing Benefits, Minimizing Risk report (Released: January 14, 2015), which articulated a forward-thinking set of standards for the sharing of clinical trial data information.

National Institutes of Health Alzheimer’s Disease Neuroimaging Initiative (ADNI)
We continued our participation 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.

Pfizer & Samsung Bioepis—Diabetes
In 2013, we announced early-stage diabetes and metabolic research partnerships, as well as clinical collaborations that we hope will bring forward the next wave of oral therapies.

We entered into a worldwide (except Japan) collaboration agreement with Pfizer for the development and commercialization of Pfizer’s ertugliflozin (PF-04971729/MK-8835), an investigational oral sodium glucose cotransporter (SGLT-2) inhibitor for the treatment of type 2 diabetes. Development of ertugliflozin has progressed to Phase III. Under the terms of the agreement, we are collaborating with Pfizer on the clinical development and commercialization of ertugliflozin and ertugliflozin-containing fixed-dose combinations with metformin and with JANUVIA® (sitagliptin) tablets.

We also entered into an agreement with Samsung Bioepis to develop, manufacture and commercialize MK-1293, an insulin glargine candidate for the treatment of patients with type 1 and type 2 diabetes. Under the terms of the agreement, our company is responsible for the clinical development, manufacturing and, if approved, commercialization of MK-1293.

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 (PMDA) qualified new biomarker laboratory tests that signal kidney injury

TransCelerate BioPharma Inc.
Our company joined TransCelerate BioPharma Inc., a nonprofit organization focus on improving the health of people around the world by simplifying and accelerating the research and development of innovative new therapies. We support the philosophy that collaboration on topics across the biopharmaceutical research and development community is the best way to solve our industry’s most complex challenges and drive positive change.

We actively participate in all five of the strategic priority areas: 1) Improve the Site Investigator Experience; 2) Facilitate Information Sharing; 3) Enable Harmonization of Clinical Trial Processes; 4) Enhance Sponsor Efficiencies; and 5) Improve the Patient Experience. Our colleagues contribute across a breadth of responsibilities. This includes serving on the Board of Directors and fulfilling Treasurer, Sponsor, Workstream lead and Workstream member roles.

Governance

Our governance structure is as vital to our success as the life-changing products we bring to market.

Our Research Leadership Team develops the divisional strategy, allocates resources and manages the R&D portfolio. The team comprises the heads of functional areas within our company’s research laboratories, and each area provides expert, efficient support of our drug candidates—ushering them from drug discovery through product-life-cycle management.

Code of Conduct

All of our employees must abide by our Code of Conduct, which applies to the way we work with external researchers, doctors and academics. According to our Guiding Principles for Business Practices Involving the Medical and Scientific Community, all activities involving the medical and scientific community that are sponsored or supported by our company, including our subsidiaries, are to have a legitimate, well-articulated business purpose. In addition, all activities are to be implemented in accordance with the highest standards of ethics and integrity, having the utmost regard for patient health and safety.

In accordance with our company’s policy, we do not tolerate fraud or misconduct in our research activities—whether by an employee or an external business partner. We deal promptly, directly and appropriately with all reported cases. MRL policy is aligned with our company’s Corporate Policy on Reporting and Responding to Misconduct.

Compliance

To help ensure compliance, we have clear policies in place to provide guidance to employees on ethical and lawful conduct. It is each employee’s responsibility to conduct him or herself ethically and lawfully.

Our company’s compliance program is based on Chapter 8 of the U.S. Federal Sentencing Guidelines, Sentencing of Organizations, as amended, which sets forth the elements of an effective compliance program, as well as more specific guidance for the pharmaceutical industry issued by the Office of the Inspector General in 2001. Learn more about our company’s compliance programs.

The company’s Compliance Charter has allocated responsibility and accountability for compliance to the divisional level. Therefore, each division has established its own compliance committee to tackle specific divisional issues and requirements.

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. To manage compliance the Committee is composed of the Research Leadership Team. In this way, compliance efforts encompass the entire division and go beyond simply addressing the conduct of clinical trials.

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

Performance
R&D Summary
20112012201320142015
Research and Development expenses (in billions)$8.5$8.2$7.5$7.2$6.7
Employees involved in research activities14,10013,60012,30011,40011,900
New products approved133072
Products in the pipeline and under regulatory review23441353331
Top 20 global burdens of illness addressed by our products and pipeline353%55%88%88%88%
Established significant external licenses and collaborations.45261403564
Filed U.S. patent applications223192159125185
1 Form 10-K
2 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 16, filed on February 26, 2016. This includes candidates in Phase II, Phase III, or under regulatory review as of February 19, 2016. When candidates attain regulatory approval they are removed from this pipeline view.
3 As defined by the Institute for Health Metrics and Evaluation (IHME), which replaces the previously used WHO chart of leading causes of disease condition or injury.
4 Starting in 2014, this metric no longer includes select early licenses and research collaborations that were included in the metric for previous years.