Giving voice to patients


Steagal, MA & Dainesi, SM


Recently, a lot of companies working in the health care arena started to work with the concept of “patient centricity” in their organizations. The same can be observed in hospitals and health service providers. For the medical doctors, this “new” concept is not new indeed, as they hear about it since the first day of medical school. Hippocrates, called the father of Medicine, was the first to urge physicians to think about the patient and not only the disease they have. “Cure sometimes, relieve often, comfort always” is a known citation, assigned more frequently to William Osler, but also to Edward Trudeau and even to Hippocrates (1).

The healthcare ecosystem is moving from a health care professional and payers-centric model to a patient-centric model. The randomized trials have now given way to a situation where drug and device companies need to prove, through real-world evidence and measurable outcomes, that their products are effective and do solve the patients’ problem (2).

Patients, who were previously merely participants, can be included in clinical trial design assuming the role of key drivers of trials, and hence looking for clinical trials more efficient and productive (3).

In 2012, the U.S. Food and Drug Administration (FDA) established the Patient-Focused Drug Development (PFDD) initiative to more systematically obtain the patient perspective on specific diseases and their available treatments. Some meetings were organized and provided key stakeholders, including researchers, drug developers, patient advocates, healthcare providers, and government officials, an opportunity to hear the patient's voice. Since then, the incorporation of the patient's voice in drug development continues to be a priority for FDA (4,5). A number of initiatives emerged, such as: encourage identification and use of best practices to facilitate patient enrollment and minimizing the burden of patient participation in clinical trials; define methods to capture information on patient preferences; identify the information that is most important to patients related to treatment benefits, risks, and burden, and how to best communicate the information to give them the enough empowerment and support their decision making (6). A number of “Voice of the Patient” reports were prepared summarizing the input provided by patients and patient representatives at each of these public meetings (7).

Similarly, in Europe, the main role of patients in discussions at the European Agency of Medicines (EMA) is not expected to be of a scientific nature. Although experience has demonstrated that they very often are able to contribute scientifically into the discussion, the added value of having patients in benefit-risk discussions is to bring a unique and critical input based on their real-life experience of being affected by a disease and its current therapeutic environment. This involvement bridges the gap between patients and drug developers and other stakeholders in life sciences and it can increase confidence and trust in the regulatory process and lead to higher level of transparency (8).

In Brazil, Anvisa is also working on listening to the patients, however the focus is in the safety of medicines (Pharmacovigilance) which is of course of great relevance too (9).

Following this trend and recognizing the importance of listening to the patients, one example of how much we can learn from a patient is provided below:


The voice of a patient:


“I would like to thank for the kind invitation to bring the perspective of the patients to this discussion. Talking about clinical research always delights us because it represents hope and new opportunities to improve the treatment outcomes of thousands of people with cancer or serious diseases.

In a scenario where health solutions are increasingly complex, due to the many factors that include, since the early and accurate diagnosis of the disease, permeating (up to) the issue of economic impact and access among many others factors, we are obliged to believe that the best – maybe the only - way is through collaboration. Collaboration between the actors involved, collaboration between different interests and collaboration between different knowledges.

In this sense, the involvement of the patient in the whole process, as she/he is the most interested in the solution, is increasingly important. This means placing patients and their needs at the center of health decisions. Healthcare unfortunately does not work like other sectors that are structured to give a good customer experience. In health, many times the thinking is guided by the interests of the team, the institution, private insurance services or public source of payment, suppliers of medicines and equipment. Therefore, the structure of the services reflects the point of view and ideas of these stakeholders, instead of the patients. However, the recent trend observed in the health technology sector in order to focus on patient needs are gradually expanding the understanding and attention given to patients' considerations.

For patients, what does it mean to place patient needs at the center of decisions throughout the whole health care process?

Patient Centricity means: Having the diagnosis facilitated, agile and accurate and communicated with empathy and care. Honest and kind communication is crucial at the time of diagnosis. Many patients miss this care and feel the short time the doctor and staff give to clarify their doubts. Waiting for a diagnosis is very painful and depending on the disease, the city and the treatment center, this delay can be very long and cause unnecessary suffering to the patient. Many patients report that their symptoms are minimized by health professionals’ teams who try to reduce the associated stress, anxiety, aging, infection and other complications.

Patient Centricity means: Provide information about illnesses and available treatments in clear language that is understandable to the patient and family members, provide details about available therapies, pros and cons of each option, and allow the patient to participate in decisions of the treatment to be performed. Here for example the doctor may suggest that the patient needs to take diuretic every day, but depending on their work activity,  this would be very complicated; in this case, the doctor can accept and arrange the prescription in a way that the drug is taken four times per week,  offering to the patient the alternative of drinking less fluids, lowering salt in food and other things that will also contribute to the desired outcome. The opening hours for appointments, exams and treatment should be flexible to meet the needs of each patient. Allowing and providing a system for scheduling exams and procedures can already have a very positive impact on people's lives.

Patient Centricity means: Provide multidisciplinary care for all patients, because for most complex diseases, this type of support is critical. Support from physiotherapists, psychologists, social workers and nutritionists (among others), all of them are very important for patients. However, support from these professionals is not always available. Patients lack information about what they can do to improve their recovery with the help of exercise, diet, attitude/behavior and complementary therapies. For example, around 70% of patients assume they need psychological support during their treatment, but few of them can access this service. Many diseases, such as cancer, affect much more than just the individual. The impact is on the lives of their family members who equally need psychological support and welcoming. Feeling safe, the patient has more courage and optimism to face long and painful treatments. Comfort is important as well. Something as simple as providing transportation for patients to go to the hospital and undergoing their treatment or follow-up exams can greatly enhance patient's compliance with treatment.

Patient Centricity means: Take in account the financial and career impact on patients' lives; how treatment will affect your household finances and even the livelihood, which can determine if the patient will be able to adhere to the proposed treatment. Courses and workshops can be offered to help patients with self-employed activities: photography, manicure, hairdresser, handicraft, sweet and savory production, etc. The patient's main concern is with his/her survival, but it is very important that he/she feels understood and supported. The focus should be what is indeed relevant to most patients' quality of life from their own perspective.

Patient associations are important allies in giving voice to the patients and their unmet needs and contributing to the empowerment of patients and their families, enabling meetings and sharing of experiences, among many other highly relevant actions and programs. At Brazilian Association of Leukemia and Lymphoma, ABRALE (10), we operate nationwide with cancer patients, and we have numerous programs that include patient support, awareness and information, training of health and medical professionals, data collection and monitoring, and political mobilization and advocacy for patients' rights. Throughout these 17 years of experience we believe that we have contributed a lot to broadening the dialogue between all players, to design the best actions that include and respect the vision and wishes of all members.

However, pleasing only the patient and forgetting the healthcare professionals, keeping them stressed and overworked, without the resources to meet people needs is definitely not a good way. We are proposing collaboration among all actors. For the patients, when we mention clinical research we are talking about future treatment and also hope. For this reason, it is important to understand the patient's entire path and what patient centricity means to the patient, to glimpse life-impacting opportunities by bringing improvements to gaps not well addressed yet but which are essential for the patient.

I was once at an international company meeting where the preliminary results of clinical research would be presented to a very small group of medical experts and representatives of the world's leading patient institutions for the condition. The doctors and the company were very optimistic and excited about the results when they were surprised by the patients' opinion. A very common side effect with the therapy was diarrhea and the patient representing the association said to the doctors: “with cancer I can go to the movies, to the restaurant, but not with diarrhea”.

I do believe we have much to contribute in clinical trial design as we are being heard about unmet needs with currently available therapies and to bring about innovation and impact on people's quality of life and survival. Ask the patient: “If you are thinking of an improved treatment for your illness, what would it be crucial for you to offer?”. Including patients in health professionals' meetings is, therefore, very important.

The patients should receive more and more information about the available clinical research studies for their diagnosis and, for that, doctors and clinical investigators must make it easier to access research, through web sites such as the clinical trials registers. A lot has already been done, but there is still room for improvement.

Thank you for including us in this event and we hope that we can continue to participate together in the solutions desired by everyone for the patients we are, care or will be. Count on us and our commitment to continue working to reduce inequalities in our precious country. Being able to help is certainly much better than needing help.

May God continue to intuit our choices and renew our energies and strengths.”


The speech was delivered at a Brazilian congress in September 2019 (11).




  1. Rezende, JM. À sombra do plátano: crônicas de história da medicina [online]. São Paulo: Editora Unifesp, 2009. Curar algumas vezes, aliviar quase sempre, consolar sempre. pp. 55-59. ISBN 978-85- 61673-63-5. Available at:
  2. Shankar A. Patient-Centricity: Empowering Patients, Enhancing Outcomes – An Empathic Approach to Discovery, Available at:
  3. Bhardwaj PKumar JYadav RK. Patients driving the clinical trial designs - Democracy in clinical research. Rev Recent Clin Trials.2019 Aug 8. Available at:
  4. FDA-led Patient-Focused Drug Development (PFDD) Public Meetings, 2012. Available at:
  5. Chalasani MVaidya PMullin T. Enhancing the incorporation of the patient's voice in drug development and evaluation. Res Involv Engagem.2018 Apr 2;4:10. Available at:
  6. CDER Patient-Focused Drug Development, 2019. Available at:
  7. The Voice of the Patient: A Series of Reports from FDA's Patient-Focused Drug Development Initiative, 2019. Available at:
  8. Incorporating patients' views during evaluation of benefit-risk by the EMA Scientific Committees, 2014. Available at:
  9. Como posso contribuir para aumentar a segurança do paciente? Orientações aos pacientes, familiares e acompanhantes, 2017. Available at:
  10. ABRALE – Associação Brasileira de Linfoma e Leucemia.
  11. Congresso Brasileiro de Medicina Farmacêutica, 2019. Available at:


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