Patient-First Model Meets Needs of Older Adults With Rare and Orphan Diseases: Optimizes Personalized Medicine Data, Informs Health Care Decisions, and Enhances Population Health Management
By Donovan Quill

America’s aging population faces unprecedented challenges—both clinical and nonclinical—at a time when the older adult population is booming. With the dramatic shift to home-based care, new emphasis on social determinants of health, and the impact of the COVID-19 pandemic, a patient-first approach to treating patients with rare and orphan diseases can lead to improvements across this vulnerable population.

Four of the top rare medical conditions that affect older adults include multiple system atrophy, a rare degenerative neurological disease often associated with Parkinson’s disease; diffuse idiopathic skeletal hyperostosis, bone spurs that develop along the spine and joints; bullous pemphigoid, a rare skin condition that causes large blisters to develop in certain areas of the body; and Binswanger’s disease, which occurs due to widespread damage in the deep layers of white matter in the brain.

To treat this targeted population, industry experts state that orphan drugs will be a major trend to watch in the years ahead, accounting for almost 40% of FDA approvals this year. But these drugs can be expensive, costing on average $147,000 or more per year.

Personalized or precision medicine—a disease treatment and prevention approach that takes into account individual variability in genes, environment, and lifestyle for each person—is emerging as a trend for population health management. This approach utilizes advances in new technologies and data to unlock information and better target health care efforts within populations.

The Value of Personalized Medicine
As genome science becomes part of the standard of routine care, the vast amount of genetic data will allow medicine to become more precise and personal. In fact, the growing understanding of how large sets of genes may contribute to disease helps to identify patients at risk of common diseases such as diabetes, heart conditions, and cancer. In turn, this enables doctors to personalize their therapy decisions and allows individuals to better calculate their risks and potentially take preemptive action.

What’s more, the increase in other forms of data about individuals—such as molecular information from medical tests, EHRs, or digital data recorded by sensors—makes it possible to more easily capture a wealth of personal health information, as does the rise of artificial intelligence and cloud computing to analyze this data.

Personalized medicine holds great promise because it has the capacity to detect the onset of disease at its earliest stages, preempt the progression of disease, and increase the efficiency of the care provided by improving quality, accessibility, and affordability.

It’s important for geriatric care professionals to understand the rise in specialty drugs, with leading specialty pharmaceutical companies now developing and commercializing personalized drugs for orphan and ultra-orphan diseases. This can lead to productive collaboration and meaningful partnerships between manufacturers, physicians, and patients, with a specialty pharmacy, distribution, and patient management service provider at the helm of interactive communications.

This will be important for patients and physicians as manufacturers are turning toward patient-first models to align with market trends, optimize the opportunity to maximize therapeutic opportunities for personalized medicines, and help contain costs of specialty pharmacy for rare and orphan diseases. This approach leads to a more precise way of predicting the prognosis of genetic diseases, helping physicians to better determine which medical treatments and procedures will work best for each patient.

This model also maximizes interaction with patients for adherence and compliance, hastens time to commercialization for much-needed drugs, and provides continuity of care to avoid lapses in therapy. For aging individuals with specialty drug needs, a patient-first approach provides exceptional support to caregivers as well as their health care providers.

Integrating Data With Human Interaction
When it comes to personalized medicine for patients with rare and orphan diseases, it’s important to tailor technology and data solutions to their needs and take a high-touch approach to improve patient engagement and compliance.

Elderly patients with rare and orphan diseases require an intense level of support and benefit from high-touch service. A care team, including program manager, care coordinator, pharmacist, nurse, and physician specialists, should be 100% dedicated to the disease state, patient community, and therapy. The key to effective care is to balance technology solutions with methods for addressing human needs and variability.

A patient-first approach can ensure care continuity across the entire patient journey with robust communication to yield rich data clinicians can use to make more informed decisions and improve the overall patient experience. This focus addresses all variables around collecting data, while maintaining frequent communication with patients and their families to ensure compliance and positive outcomes.

Telehealth in the Age of Pandemics
As the COVID-19 pandemic continues to create significant hardship, uncertainty, and human suffering, especially for the nation’s aging population, a fully integrated telehealth option has become an imperative for delivering the following quality care and better patient outcomes:

• improved care coordination for patients;
• customized care plans based on conversations with each patient;
• medication counseling; and
• education on disease states and expectations for each drug.

A customized telehealth option enables essential discussions for understanding patient needs, a drug’s effect on overall health, assessing the number of touch points required each month, follow-up, and staying on top of side effects.

Each touch point must have a care plan. For instance, a product may require the pharmacist to reach out to the patient—and the physician, as needed—after one week of treatment to assess response to the drug from a physical and psychological perspective: asking the right questions and making necessary changes, if needed, based on the such factors as the patient’s daily routine and changes in behavior.

This approach captures relevant information in a standardized way; every pharmacist, physician, and patient receives the same assessment based on each drug, which can be compared with overall responses. Information is gathered by an operating system and data aggregator and shared with the manufacturer, which may make alterations to the care plan based on the story of the patient journey created for them. Equally important, patients know that help is a phone call away and trust the information and guidance that pharmacists provide.

— Donovan Quill is president and CEO of Optime Care.