Abstract
We developed a new model for initiating, coordinating, funding, and managing biomedical research projects. The concept involves engaging the patients with chronic conditions with no known cures into goal-oriented research activities. In this model, the patient seeks the help of a research organization to bring together a multidisciplinary team of research scientists and physicians to initiate research projects using the patient's grant funding, samples, as well as the management expertise. This model may be of interest to other research institutions because it has many benefits, including new sources of private research funding, when government funding is getting scarce, motivating scientists and physicians to work closely together on goal- and patient-oriented research projects, and using patients' management skills.
Introduction
What we propose here is a significant extension of this process where a team of researchers is recruited to aid clinicians by using both experimental and bioinformatics tools in an attempt to discover a novel disease management strategy. Currently research and clinical practice in privately funded health care are largely separate. This may be impeding progress and the adoption of cutting-edge methods into mainstream clinical use. Many top-rated medical and academic institutions are using the concepts of concierge medicine to subsidize other areas 7 without involving patients directly into the research projects. Many private individuals with medical problems donate to research or medical organizations, without engaging in research activities.
Here we propose a new model in which the patient becomes directly involved with the research project to find new solutions for unmet medical needs. In addition to paying for the research and providing clinical samples, the patient helps to coordinate the research activities. This may lead to improvements in the way the research is integrated directly into clinical practice.
Involving Patients in Personalized Science to Fund Research Projects
Many high-net-worth individuals with excellent management skills donate billions of dollars to research via foundations and fellowships, without being directly involved with the research projects. These foundations are typically set up to address a broad cause and usually do not have a core goal-oriented project. A team of experts is hired to supervise research activities and provide grants to scientists who are usually already funded by the government, academia, or industry. Some high-net-worth individuals, besides donating to basic research, would also like to solve personal medical problems and steer some of the research effort. These individuals often have excellent project management and organizational skills, but lack the scientific background and confidence to start their own research projects. In contrast, many young scientists and medical doctors would like to apply their skills to conduct cutting-edge research and work with patients, but lack management expertise. Personalized science projects could bridge this gap and link patients with management expertise with teams of scientists and physicians who are interested in pursuing goal-oriented science, the patient provides research grants, samples, and management expertise. The team of physicians and research scientists executes research projects that address the patient's future needs and research interests.
To test this personalized science concept, we organized a group of young scientists and physicians with over 500 active members and conducted a series of 30 weekly lectures on regenerative medicine. In parallel, we used social media to create multidisciplinary teams of biophysicists, biologists, geneticists, and physicians to work on 25 research projects with topics spanning from metastatic mineralization of the connective tissue to bioinformatics approaches to personalized medicine in oncology. Using this pool of scientists and physicians, it became possible to launch research-personalized science projects to address both patient research interests and needs. Whenever a high-net-worth individual who has medical problems or would like to prevent possible future medical problems becomes interested in pursuing a research project, the project is announced to the community to form a core team that is funded and co-managed by that individual. These small, goal-oriented teams dedicated to solving specific patient problems may be more effective than large foundations or industries (Fig. 1).
Power to the patient. Patients provide grant funding to a research organization. The organization provides funding and research infrastructure to young researchers. The research team then interacts with the patient and leading experts in the field to provide care.
Process
The high-net-worth individual creates a description of a medical problem to be addressed and proposes the research budget and a set of milestones (Fig. 2). The research organization then describes and announces the project to the community of scientists and physicians that can submit applications to join the research team. This creates a situation in which candidates are not writing proposals based on extensions of the research they have already done, but instead they address, a health question that has already been elaborated. The research organization, together with the initiator of the project, can then select a qualified team from a pool of interested candidates. The patient helps the effort by coordinating the project, setting targets, providing samples, and providing the funding. The physicians perform a comprehensive clinical review of the medical problem, engaging top experts in the field whenever possible. The physicians decide what factors are likely to be relevant to the target problem and form collaborations with the scientists. The scientists perform various bioinformatics analyses of the factors and target problem, focusing on the most promising of the therapeutic approaches. The physicians focus on performing patient-specific studies, such as various “omics” analyses, imaging, and any other additional tests as indicated by the medical problem. Together, the physicians and scientists develop a working treatment hypothesis. At this point, the physicians and scientists design a series of experiments to test the hypothesis. Finally, the team provides the managing patient with a research report and review of the current information. They list current and possible new treatment options for the patient. The team can also propose further investigation to extend the project and possibly lead it to commercialization.
Process description. Teams of patients, MDs, and PhDs work together to determine the best course of action for personalized medical care.
Examples
A 40-year-old patient suffering from endometriosis was the owner of a successful international manufacturing and marketing business and decided to support research in endometriosis. She contacted a large research and clinical center running a voluntary organization in regenerative medicine for young scientists. With guidance from the supervisors of the practicum, she interviewed young physicians, scientists, and students and provided research fellowships to those interested in endometriosis. She also decided that if the research project showed promise, she would fund the project on a large scale. She selected physicians (ObGyn/embryology) and scientists (bioinformatics/systems biology) to work with her on the project. The physicians consulted several leading experts in the field internationally using the patient's diagnostic data, performed a variety of tests, and prepared a report on the factors involved in endometriosis and patient-specific diagnostic data. Together with the scientists and the patient, the team developed screening and diagnostic routines that went far beyond traditional practice to monitor hormonal levels and screen for potential biomarkers in blood, saliva, stool, and urine. Also, as suggested by the patient, the team collected samples of endometrial cells, performed transcriptome analysis and signaling pathway analysis, identified perspective drug targets, and shortlisted a number of potential candidate drugs and therapies. In the meantime, the patient stabilized her condition under the supervision of the leading experts in the region, who became interested in the science component of the project.
Another example involved a high-net-worth individual interested in aging research, who briefly described the condition of one of his colleagues, which involved accelerated mineralization of connective tissue. On the basis of this suggestion, six young physicians and scientists formed a multidisciplinary team to study the condition. The team worked for 6 months using the research organization's infrastructure to perform a detailed analysis of the factors involved in mineralization, literature review, and bioinformatics analysis and proposed a series of experiments to confirm their hypotheses. The patient did not fully engage in the project, but it resulted result in closer collaboration between the scientists and medical doctors working together to address the patient-specific problem.
Benefits of Personalized Science
From the patient's perspective, there are many benefits of personalized science. Patients can achieve a better understanding of their medical problem. Due to their direct involvement with the funding aspects and the research team, they have the ability to direct research, set objectives, and apply management talents. Their own samples and information can be used for research purposes, creating the ultimate in personalized medicine. Receiving informed consent from the patient becomes exponentially easier, because they have a better comprehension of the medical issues and the treatment plan. While their personalized care is being developed by researchers, the physicians can use their networks to put their patient in contact with leading experts in the field, so that the patient can receive better treatment in the future. Finally, the patient has the potential prospect of turning the research project into a business and generating more revenue.
Researchers benefit from this new funding paradigm in a number of ways. The process creates new avenues for funding young scientists. These scientists will spend less time on grant writing and reporting and more time on the actual research problem. By using goal-oriented research with a specific patient in mind, it may be easier to determine the best course of action and experimentation. Teams with physicians and scientists working together will pool the assets from both fields. The scientists have the ability to contact authorities in the field of study and possibly publish a review or a case report at the end. Finally, researchers may be able to commercialize the research findings with an educated seed investor.
The public will benefit from personalized science, because patients will assume the cost of fundamental research. This process also creates a new bottom-up approach to solving global medical problems. The public will have faster access to cutting-edge medicine and science. Finally, it may be able to reduce health care costs by identifying optimal target-oriented therapies aimed to cure specific medical issues.
Legal and Ethical Issues of Personalized Science
Although there are clearly many benefits to personalized science, it raises some serious legal and ethical issues. The concierge medicine concept is increasingly drawing ethical criticism, 8 because today's research scientists rarely engage in behavior classified as misconduct that involves direct contact and working directly with the patients. 9 Direct patient involvement may prompt unnecessary and unregistered screening and diagnostic procedures, misuse of patient records, and self-experimentation, especially considering cases of off-label use of registered drugs. A patient making tax-deductible donations to non-profit organizations performing research using patient's material may be deriving personal benefits that are not legal from the taxation standpoint. Unavoidably, wealthier patients engaged in personalized science may get an unfair advantage compared to regular patients. A wealthy patient could also conceivably become disillusioned by the results or have unacceptably high expectations. However, all of these issues can be addressed and potentially resolved on a case-by-case basis. For example, the patient may decide that benefiting from personalized science outweighs a tax break for donating funds and forgo receiving the tax break. If wealthier patients allow other patients to come into the study at a reduced cost, less wealthy patients may benefit as well. The public benefits from this new paradigm may help compensate for these potential legal and ethical issues.
Summary
The new paradigm of personalized science yields funding, patient samples, data, and skills for goal-oriented research in the current resource-constrained environment. Young researchers working together in small teams with expert management from the patient may find new ways to treat patients. Funding personalized science will create a new wave of opportunities for researchers and provide high-net-worth patients with the ability to engage in solving their personal medical problems while subsidizing and directly contributing to research activities. These opportunities are excellent starters for new business ventures and treatment options for the public at large. Overall, personalized science is a fascinating new possibility for science.
Footnotes
Author Disclosure Statement
No competing financial interests exist.