We recently caught up with Debra Michaels, MS, RDN, senior scientist at DIA, and asked her what she thought has the most potential to support patient-centric clinical trials and what the challenges are of implementation.
If you could choose only three technologies/innovations as having the most potential to support patient-centric clinical trials, what would they be and why?
Telemedicine will facilitate patient access to trials and care, as well as patient convenience, feedback, and input.
The “Learning Health System” – the integration of clinical research with health care delivery so that trials could become a seamless care option for all patients. EMR data would contribute to trial data in the LHS, lessening the burden on patients and making patient outcomes the driver of the research process.
Computing and modeling technologies to enable the design and operation of “Master Protocols” to speed new medicines development and reduce the need for duplicative trials that expose more patients to trials and placebos.
Master protocols may test multiple treatments for one disease or one treatment for many diseases with common molecular mechanisms.
These complex designs will reduce the need for numbers of patients in trials, and for the number of controls exposed to placebos. They will also be more efficient at determining the impact of treatment on population subgroups.
What are the main challenges to implementation?
Ensuring that patient input is representative of the patients to be served – including patients with all stages of the disease and all abilities to live with the disease burden; including “hard to reach patients” and those who can’t express themselves, such as Alzheimer’s disease patients.
Incorporating patient centricity systematically is not a quick process; it involves building a culture, which takes time.
Negative perception of industry motives – patients do not always trust that the industry is motivated by a true interest in their well-being. A concerning new trend is the public perception that industry, through its efforts to collaborate with patients, is seeking to influence patient group statements and positions for commercial gain.
Data standards must be developed for data from digital devices, and digital data validated, to ensure meaningfulness of the data collected from patients through wearables and other digital devices for patient input.
What role has the patient played in this movement?
The patient has been the catalyst in the patient centricity movement.
Beginning with the activism of AIDS patients in the early 1980’s to establish their voice in therapeutic development programs, a steady stream of incremental changes has been driven by patients who want a voice in how and when their needs will be addressed.
How will this role evolve with more access to information, technology, etc.?
The internet has been an enabler, providing the ability to widen communication. The result has been an enhanced ability for patients to identify and coalesce patient communities which function as strong advocates for the rights of patients. The internet has given view to the pace of scientific and technological development so that patients see what is possible in terms of better treatments and better care.
Patients now understand the value of their data, and as their role evolves, patients will have increasing expectations for access to and control of their data. This is reinforcing the drive to view patients as partners, with a say in policy decisions that influence the treatments that are developed, access to those treatments, treatment cost, care, and quality of services provided to them.
How do you expect the idea of patient centricity to evolve over the next five, ten years?
More emphasis will be placed on addressing the needs and reaching out to underserved populations – populations defined by demographics, by disease or condition, by economics, as well as by geographic areas.
Some of these underserved may be included in clinical trials by bringing care directly to them in the home setting or through remote participation and monitoring via digital technologies.
Increasing focus on access to care and treatment and on sustainability of care – how we pay for our treatments and the cost of those treatments
Especially over the next five years, intensive efforts to educate industry on ways to systematize/build comprehensive patient centricity programs within their organizations; and the education of patients to enhance their ability to act as partners in the complex medicines development and approval process.
What are some of the key initiatives the industry is undertaking to improve patient centricity?
Starting with one of the traditional touchpoints of the industry with patients, clinical trial participation, industry is moving to involve patients in protocol design.
Industry is also working on understanding the full patient experience (sometimes referred to as the patient “journey”) – from the burden of living with their disease or condition, to the experience of diagnosis, to patient perspectives on treatment experience and outcomes – as the foundation for planning the development and lifecycle maintenance of needed treatments
Companies, even large, complex companies, are beginning to incorporate patient centricity systematically throughout the organization and its work. Strategic planning, policy setting, process development, staff training and goal setting, in addition to efforts to involve patients in specific activities such as protocol design, are structured to include the patient in the decision-making process.
Birth of the “science of patient input,” which is developing ways to quantify patient input to ensure that the input is representative of the patients affected and is expressed in a way that facilitates its use in decision-making in the drug development and approval process
Use of digital technology to facilitate the patient’s ability to participate in trials (digitally enabled patient-reported outcomes), to utilize treatments (digitally enabled delivery devices such as inhalers), and to use prescribed treatments appropriately for the best outcomes (smart pills, smart bandages, etc.)