Historically, treatment development has been viewed through a very narrow lens, with researchers only interested in a specific class of data or a specific kind of clinical interaction. This leaves a big gap in clinical development's attention to human experience with the disease and what day-to-day challenges are present that may impact the efficacy and value of the treatments being developed.

To accelerate and expand the impact of effective treatments, industry leaders must break away from the status quo and place the needs of patients at the center. The key to driving this change is the advancements in patient-centered registry technology that captures longitudinal information.

Functional Versus Clinical Endpoints

When talking about endpoints in disease research, generally, the focus is on traditional clinical endpoints. For example, when a patient with a degenerative eye condition goes in for an exam, one of the clinical endpoints is the ability to read two or three more lines on the eye chart compared to pre-treatment.

However, the current research system does not contextualize functional endpoints that impact patients’ daily lives, such as night blindness or losing mobility due to peripheral vision declines. The critical information patients value is whether the treatment will introduce the potential of retaining the activities of daily living that support a greater quality of life.

Getting Quantitative Value from Qualitative Knowledge

Non-profits and patient advocacy organizations can house a wealth of information about patients and their disease. They not only know how the disease itself impacts the body but also how it impacts the person’s goals, their families, and the communities they’re living in.

Historically, biopharma companies have found it challenging to quantify this information in the research and development process. To help support the development of new therapeutics, advocacy organizations and non-profits with patient centric registry data can shed light on the learned patient experiences and stories. This combination of quantitative insight and robust health data that registries provide will help guide researchers' decision-making to accelerate treatment innovation.

Reciprocal Value of Respecting Patient Motivation and Intelligence

There has been a long-held belief in research and clinical care that providing patients with detailed information about their diseases and the research process will do nothing but overwhelm them. However, this can become a self-fulfilling prophecy for industry professionals because the less patients are informed, the less they will be able to understand.

The better the industry gets at engaging with patients, the more patients can participate in the research process, bringing value back to everyone involved. This is where the role of technology really comes into play as the facilitator of the bidirectional flow of information that is necessary if we are to change the paradigm.

Using Technology to Facilitate Long-Term Goals and Outcomes

While, in our everyday lives, we use text, email, Slack and Zoom, to stay in touch, the research world is still trailing behind when it comes to engaging with patients. Using technology in a way that mimics how we interact with technology on an everyday basis can be beneficial in research because it's familiar.

Mindful Technology Application to Overcome Practical Hurdles

An example of where simple technology could make a big impact is when patients involved with natural history studies fill out high-level questionnaires about how their symptoms are progressing or regressing during their regular clinical visits. However, asking patients to provide a single response that encompasses 6-months of symptoms leaves a lot of information, and opportunities for care, out of the equation.

If there were a dashboard where patients could go every day to mark off how they were feeling from 1-5, or click a thumbs up or down, there would be a much clearer view of the patients’ symptom progression. It would also reduce the burden on the patient to accurately retain and relay medical details over long periods of time and would instead be integrated into their ongoing healthcare routine. Beyond just that, if a patient says they’re not feeling well for a week or two straight, the system could alert their care team and have someone reach out to the patient to schedule a visit. This not only increases the quality-of-care patients are getting, but it also incentivizes them to continue to provide robust insight to researchers on their condition on a regular basis.

Using Technology to Increase Value Reciprocity for Long-Term Patient Engagement

With the introduction of new gene and cell therapies, people are living longer than they ever have with some of the formerly life shortening rare diseases, which has introduced an entirely new cohort of patients into the research arena. If there are patient interactive dashboards, like the previous example, already in place to engage with patients on their long-term care, it will make the process of following up with a patient 5, 10, or even 15 years after their initial participation much more successful.

There is also a huge opportunity to repurpose the information gathered from existing participants when recruiting new patients. Not only will the positive experience patients have get passed down within patient organizations, but if newly diagnosed patients are able to see the journey of others who have participated, it will help contextualize their experience and motivate them to engage.

The Value-Feedback Loop with Patients at the Center

Each industry stakeholder has goals they are trying to reach, whether it’s the biopharma companies, healthcare providers, patient advocacy organizations, or the patients themselves. Although the main goal is to find treatments and cures for rare and chronic illnesses, the way in which each entity has approached the solution has been disjointed. That no longer has to be the case, user-friendly technology tools exist today that will allow patients to both provide and gain insight into their disease in a central location, accessible by all stakeholders involved in their care.

Some features may not seem as beneficial to researchers, such as the clinical care dashboard for patients and doctors to easily track consecutive days of poor symptoms. However, the information collected on that platform can go on to supplement the data researchers need to conduct clinical trials. By utilizing tools that patients gain value from in their daily lives, it will be much easier to engage with them long-term, especially as new treatments increase survival-rates and research becomes more longitudinal.

If we place the human being at the center of operations, it becomes clear that once there are systems in place to engage with their experience having the disease, and we create processes to streamline the way that information is gathered and shared, the benefits will be felt by all involved.