In our bid to discover, develop and deliver new medicines that meet patients’ needs and contribute to longer, healthier lives, it is vital that we listen to, learn from and act on what matters most to patients and their families. To do that, we are changing the way we approach research and development and building ever-stronger partnerships with patients, caregivers and their advocates (the people they trust to represent them). This new approach to involving people meaningfully in our work is how we are putting patient-focused drug development (PFDD) into action. Below we share some ways PFDD will help us make better medicines that improve patients’ lives.

As we start a program to develop a new therapy, we try to get an understanding of what it’s like to live with the disease we are focused on. We recognize and respect that patients and caregivers are the experts and we must hear from them to learn:

• how the disease affects them;
• what symptoms give them the greatest difficulties;
• what is missing from their care; and
• what degree of improvement would make a noticeable difference in their lives.

These insights will help us as we make decisions at every step along the lengthy process of developing this new medicine, including when we present our plans and data to agencies such as the U.S. Food and Drug Administration and European Medicines Agency at key milestones.  

When it’s time to test this medicine to make sure it is safe and that it delivers the benefits patients have told us they want most, we will work closely with and listen carefully to patients, caregivers and their advocates in order to design clinical trials with participants’ needs and experiences in mind. We will do our best to make our studies easy to understand for patients, and help those who agree to take part have a positive experience while they contribute to our understanding of the disease and its treatment. Clinical trials can take years to complete, and we will have ongoing discussions with patient experts to help us gather further insights and deal with any challenges that may arise.

We are also taking steps to improve our communications about the research we do and how it might improve the healthcare people receive. We will give those who take part in clinical trials (and so make these studies possible) easy-to-understand summaries of what we learn from the trials. These summaries will also be available to the public through our website. We will share information through social media and other easy-to-access information outlets, keeping in mind the many different ways people look for health-related information. We will provide educational programs that help doctors and patients make the best care decisions together, based on an individual’s personal needs and medical situation.  

It will take time to put these changes into action, so we will measure our progress and share updates. We are training staff at every level of our organization about the value of closer and more consistent interaction with patients and ways to do this that protect patients’ privacy and independence.

For this patient-focused approach to succeed, we must earn and keep public trust by behaving responsibly and following through on these and all our commitments to patients and their families. We must also strengthen our partnerships with patient advocacy organizations to support their efforts to enlist and train patients and caregivers so they are ready to take part in PFDD-related activities that we and others may invite them to.

We strongly believe that this PFDD approach will have benefits for individual patients, for patient communities, and for improved public health around the world.  

At Merck KGaA, Darmstadt, Germany, we are committed to developing treatments that meet the needs of our diverse patients. To do so, we fully acknowledge the importance of adequately representing diverse participants in our clinical studies to ensure assessment of safety and efficacy of our drugs across different patient populations.

To learn more about our approach to achieve fair and equitable enrollment of diverse patients into our clinical studies, please make sure to click here to read our full Commitment on Diversity, Equity, and Inclusion in Clinical Study Participation.

We are committed to create and deliver medicines that can help our patients to live longer and healthier lives.

We carefully conduct medical research and clinical trials to generate important information regarding the medicines we offer.

We adhere to a core set of principles that ensure we only perform research and clinical trials when there is sound, proven scientific methodology to investigate a scientific or medical question that is relevant to patients, healthcare professionals and society as a whole.

Below are some of the principles we are committed to on our goal to bringing meaningful new therapies to patients living with serious diseases. Make sure you also read about our commitment to responsible clinical trial data sharing.

Before a manufacturer can bring a drug into market, it must first prove its safety and efficacy through pre-clinical research and clinical trials for its intended uses.

International and national laws and regulations define the type and scope of the data to be submitted to the authorities; they also stipulate that animal experiments must be carried out.

In the pharmaceutical industry, such experiments are justified by the obligation to save human lives and alleviate human suffering – without animal experiments we would not be able to develop drugs.

We are committed to minimizing the number of test animals. Where possible, we use computer models, cell-culture techniques, or other methods to avoid experimentation on animals.

The performance of animal experiments is strictly monitored by the responsible authorities.

We are committed to deliver high quality innovative medicines to patients. High quality clinical research is conducted in accordance with applicable laws and regulations. When conducting multinational, multi-site trials, in both the industrialized and developing world, we follow the legal, ethical and scientific standards.

Our company only performs clinical trials when there is sound, proven scientific methodology to investigate a scientific or medical question that is relevant to patients, healthcare professionals and society as a whole.

We only enroll the number of participants required to answer the scientific question(s) under investigation.

Overall responsibility for product development and related governance sits with our Head of Development and is supported by a Development Operating Committee.

Our Medical Safety and Ethics Board (MSEB) makes decisions on product safety issues to ensure the best interests of patients are being served. The board is chaired by the Chief Medical Officer (CMO) and is composed of other senior physicians and scientists. The Board’s remit is to examine and endorse benefit-risk assessments throughout the entire lifecycle of our products during development and commercialization.

We recognize and value the critical role a research participant plays in each clinical trial as the data he/she provides can be the foundation for tomorrow’s treatments. 

We conduct our clinical trials in a manner that recognizes the importance of protecting the safety of and respecting the research participants.

In order to protect patient safety during the clinical trial process, we collect, analyze, characterize and communicate the safety data of our products on an ongoing and global basis. If applicable, we also look at information from similar drugs for potential safety signals.

We collect adverse-event reports from all over the world as part of our continuous monitoring. We promptly share new findings and emerging concerns with investigators, regulators and/or research participants to appropriately manage risks associated with the use of our medicines.

This type of communication is typically conducted via correspondence with the investigator, updates to the investigator brochure and/or updates to the informed-consent document for the clinical trials affected.

Merck KGaA, Darmstadt, Germany, requires that clinical investigators obtain and document informed consent, freely given without coercion, from all potential research participants. Participants may only be enrolled in a study after providing their voluntary informed consent.

We are committed to respecting and maintaining the privacy of research participants by ensuring that each individual’s data is deidentified in order to conceal the participant’s identity. We safeguard the confidentiality of each participant’s medical information in accordance with all applicable laws and regulations.

Our company only performs clinical trials when there is sound, proven scientific methodology to investigate a scientific or medical question that is relevant to patients, healthcare professionals and society as a whole.

Our clinical trials are based on scientifically designed protocols, which balance potential risk to the research participant with the possible benefit to the participant and to society.

Those aspects of a clinical trial that particularly affect the research participants such as inclusion/exclusion criteria, endpoints and choice of control (including active and/or placebo control) are guided by internationally adapted standards, such as the Declaration of Helsinki.

Placebo-controlled trials allow researchers to reliably evaluate safety and efficacy of an experimental treatment while minimizing the number of necessary participants exposed to the treatment.

Regulatory authorities often require the use of placebo as a comparator to treatment in the design of clinical trials. We only conduct placebo-controlled trials when there is a genuine uncertainty about the therapeutic merits of the proposed treatments being studied (clinical equipoise) and where doing so does not present an undue risk to the research participant.

For any of our trials that include an active control group, the standard of care provided is equivalent to established and commonly employed treatments that are medically and ethically appropriate for treating the disease.

We strive for a high protection of all clinical trial participants, however, some research populations are particularly vulnerable and need special protection.

Vulnerable populations are those who are relatively (or absolutely) incapable of protecting their own interests.

Merck KGaA, Darmstadt, Germany, supports research in vulnerable populations to ensure appropriate use of products and provide healthcare providers with information on the appropriate modality of use in vulnerable populations.

The well-being of the individual is an absolute priority at our company and we involve vulnerable populations only when there is special scientific justification, such as:

• the research could not be carried out equally well with less vulnerable subjects;
• the research is intended to obtain knowledge that will lead to appropriate, evidence-based pharmaceutical intervention, where vulnerable populations may be unique (e.g. pediatrics, elderly, etc.);
• the research will lead to improved diagnosis, prevention or treatment of disease characteristics of, or unique to, the vulnerable class;
• the risks attached to interventions or procedures that do not hold out the prospect of direct health-related benefit will not exceed those associated with routine medical or psychological examination of such persons.

The research subjects and other members of the vulnerable population from which subjects are recruited will be provided reasonable access to any diagnostic, preventive or therapeutic product that may become available as a result of the research.

When the prospective subjects are unable to give informed consent, their agreement will be supplemented by the permission of their legal guardians or other appropriate representatives and in accordance with all applicable laws and regulations.

Regardless of location, Merck KGaA, Darmstadt, Germany, conducts all its clinical trials in compliance with internationally agreed scientific and ethical standards, in addition to the locally applicable laws and regulations. Historically, the pharmaceutical industry has focused its development activities in the United States and Europe. However, the vast majority of the world's population resides outside these regions.

Our company is actively shifting its development activities to more diverse markets in order to address the health care needs in these regions as well as to support the development of their health care systems. In performing clinical trials in these developing health care systems, we apply the same principles there (sound methodology; importance of issue under investigation; quality and monitoring) that we would apply when conducting such trials in a developed health care system. When we conduct clinical trials in a developing health care system, we

• only do so in an environment that can follow Good Clinical Practice (GCP), meaning one that has appropriately set-up ethics committees and well-trained clinical investigators who perform the studies in accordance with the GCP principles;
• only address diseases and test innovative medicines that are relevant for the local population;
• conduct clinical trials in countries where there is a reasonable expectation that the drug tested will be submitted for marketing authorization and be made available to patients/subjects after proof of efficacy and safety;
• also assure that enrollment into a clinical trial does not discriminate against participants on the basis of ethnic origin, gender or socio economic status;

We are committed to developing new medicines for patients impacted by difficult-to-treat diseases as safely and quickly as possible. We believe the best way to fulfill this commitment is through clinical studies that evaluate whether the medicine works and is safe, which can lead to the medicine being approved and made available to the broadest number of patients possible. Because clinical studies depend on patient-volunteers, we encourage patients to consider participating in clinical studies. To search for actively recruiting studies or to learn about new medicines being tested visit www.clinicaltrials.gov.

We recognize that access to experimental medicine after a clinical study ends is often necessary for the continued health and well-being of patients participating in the studies. We also recognize that continued access may be a legal, regulatory and/or ethical obligation under some circumstances. Click here to read our Position Statement on Post-Study Access to Experimental Medicine.

We also believe that Investigator Sponsored Studies play a valuable role in our development of new medicines. We welcome qualified investigators to submit their study ideas to us for consideration.  For submissions in the therapeutic area of Neurology & Immunology, please visit www.emdserono.com/us-en/neurology-immunology-iss.html. For all other therapeutics areas, please visit www.ist.emdserono.com.

Although many patients may gain access to an experimental medicine during clinical studies, we understand that some patients may not be eligible or able to participate in a study. In certain circumstances, we may provide patients with access to experimental medicine outside the clinical study process via our Early Access program. To get an overview of how early access works click here for more information from the FDA. To search for actively recruiting early access studies click here and scroll down to select “Expanded Access: Available”. Additional search criteria may also be entered.

If a physician wants to request one of our experimental medicines for a patient with a serious or life-threatening disease or condition when there is no comparable or satisfactory alternative, the physician should send a request to earlyaccess@emdserono.com. Before doing so, the physician should review our Position Statement on Early Access to Experimental Medicine, download the PDF here, which outlines the principles and criteria we use to evaluate all early access requests. We will respond to requests within three business days.  

The requirements described in "Stem Cells and Human Cloning Principle" enable us to conduct stem cell research within a strictly defined framework; they thus prevent us from violating applicable laws and regulations while enabling us to work within the framework of established ethical principles.

Merck KGaA, Darmstadt, Germany, is not involved in programs utilizing stem cells or human cloning for therapeutic purposes and does not pursue such approaches.

International ethical views, laws as well as medical practice for the treatment of infertility differ and are furthermore subject to changes in society and science as well as between countries. Merck KGaA, Darmstadt, Germany, as a global company that operates in different countries around the world needs a clear framework addressing ethical and legal concerns in this heterogeneous environment. To provide clarity, Merck KGaA, Darmstadt, Germany, has developed the Fertility Principle which provides background information on Fertility treatments and the current position of the company on the research and application of drugs and technologies in the treatment of infertility.

New Merck KGaA, Darmstadt, Germany, technologies, services and therapies to improve fertility treatment outcomes as well as collaborations in this area undergo internal review, in close relationship with, seeking and taking advice, from the Ethics Advisory Panel (MEAP).

Bioethical issues are debated in a diverse social environment. Many of these questions are viewed very differently from country to country and are furthermore subject to general shifts in society. Being a global company, we must therefore address these developments at an international level; we must detect the changes and incorporate them into our position on bioethical issues.

In order to do so, Merck KGaA, Darmstadt, Germany, instituted the Ethics Advisory Panel (MEAP) in 2011, which convenes at least once a year and consists of renowned bioethics experts. Its members represent a range of pertinent disciplines such as law, theology and medical ethics.