Advocacy efforts lead the ecosystem of research, therapy development, access and reimbursement. Her sons lost their battle with DMD in their teenage years, but Pat Furlong continues to fight—in their honor and for all the community to this day. JIA is the most common chronic rheumatic disorder in children and is a major cause of short-term and long-term disability.
JIA is defined as having an inflamed joint before the age of 16 without a clear cause that persists for more than 6 weeks; it is a chronic disorder, which if neglected, can lead to serious complications.
In developing a network for biological research for patients with Childhood Arthritis doctors and scientists at the Wilhelmina Center of Excellence strongly think that input from and collaboration with patients and patient organizations is crucial. Patients, their parents, doctors and researchers all share the same common goal, namely that progress in basic science is translated in real tangible products for patients with childhood arthritis. In a patient council was formed in this Department.
Together with professionals the JIA patient council explore research priority setting by reviewing the research topics, safety and efficacy of immunizations, as well as stopping medications. In addition to this, a jointly written application was obtained for a project with focus groups for patients that was also led by a parent.
The patient council selected a topic which was the most frequent concern expressed by patients: the uncertainty patients feel due to the impact of the unpredictable course of their disease pain, relapses in their activities of daily life activities at school for younger children and later work, sports and social contacts.
Focus groups further analyzed the effects of the unpredictable course of the disease. Information was written for websites and two youtube movies were made. The JLA is a non-profit making initiative established in and it brings patients, caregivers and clinicians together in Priority Setting Partnerships PSPs to identify and prioritize the top 10 uncertainties, or unanswered questions, about the effects of treatments.
The aim of this is to make sure that health research funders are aware of the issues that matter most to patients and clinicians. In this process the input from clinicians, patients and their caregivers will be equally valued. Additionally, focus groups will be organized to involve young people with JIA.
The involvement of all contributors will be monitored and evaluated. In this manner, the project will contribute to the growing body of literature on how to involve young people in agenda setting in a meaningful way. This approach, despite still at its infancy, will inform researchers and research funders about the most important research questions for JIA and this will hopefully lead research agenda for research that really matters The examples provided show how patients and their care givers can be the catalysts of a change that is highly needed in translational medicine but they remain, as per today, sporadic cases led by unique human beings or by particularly inspired institutions.
Many obstacles remain. Qualitative research showed that the involvement of patients and caregivers is challenging: real co-design does not happen by itself First, specific educational programs are needed to improve the process of shared decision-making, for both partners, the patient and the physician.
These programs are missing and importantly clinicians are often limited in their time-management. Educate and engage patients is a time-consuming process but health insurances—as well as hospitals—push more and more to reduce the time spent with patients, as costs of medication, exams, and personnel are dramatically increasing. Scientists are even farther away from this process, as they often do not have direct contact with the patients. Current criteria for promotion in the medical field still rely heavily on individual research output such as high impact publications, h-index, grants, and invited lectures.
There is tremendous pressure and on top of this pressure, there is really no space for a patient-centric view that needs time, patience and dedication. Especially in a system where these activities are not properly recognized and, as a consequence, rewarded.
To change this, institutions need to ensure that their tenure and promotions systems are able to evaluate and recognize the contributions investigators conducting translational medicine make. DORA recognizes the need to improve the ways in which the outputs of scholarly research are evaluated.
It is a worldwide initiative covering all scholarly disciplines and all key stakeholders including funders, publishers, professional societies, institutions, and researchers. It is a first step toward assessing research based on its own merits rather than on the basis of the journal in which the research is published. In conclusion, translational medicine is a very complex branch of medicine. Translational medicine combines the skills of researchers, pharmacologists, and clinicians to evaluate new treatments and ultimately accelerate all the stages of drug development.
It is applicable to all diseases. Today, translational medicine more closely connects research and care, which ultimately accelerates decision-making for the launch of a drug on the market.
This is called personalized medicine. A biomarker is a measurable biological characteristic related to a normal or pathological process. A biomarker can be measured from different biological fluids, such as blood, and also from patient biopsies tissue samples. Imaging techniques scans, X-rays may also enable the identification of biomarkers.
Patients and patient organisations play a very important role in ensuring continuous feedback and communication among the diverse stakeholders in this field, which are essential for success. The term translational medicine was introduced in the s but only gained wide usage in the early s. Originally, translational medical research emerged from the concept of bench-to-bedside B2B , as a class of medical research aiming to eliminate the barriers between laboratory and clinical research.
In , the Institute of Medicine Clinical Research Roundtable described the current terminology and model of translational research as a two-phase process of research, progressing from:. Independently of the definition, what remains clear is the enormous need for translational medicine, mostly due to:. A final goal of translational medicine is to help patients with a more rapid development of new diagnostics, medicinal products, and new medical knowledge for treating diseases, giving access to care for people at reasonable costs.
This type of medicine has helped to translate the remarkable scientific innovations that occurred in the last years into health gains for the general population. This has been accomplished by:. Translational research is bolstered by quantitative, model-based and mechanistic understanding of disease biology and pharmacology.
Consequently core disciplines, including clinical pharmacology, pharmacogenomics, systems pharmacology, precision medicine, as well as others play an integral role in enabling translational research and translational medicine.
A new vision of definition, commentary, and understanding in clinical and translational medicine.
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