Patient-First Model: High Tech Meets High Touch for Individuals with Rare Disorders

Patient-First Model: High Tech Meets High Touch to Optimize Data, Inform Health Care Decisions, Enhance Population Health Management for Individuals with Rare Disorders
Donovan Quill, President and CEO, Optime Care

Industry experts state that orphan drugs will be a major trend to watch in the years ahead, accounting for almost 40% of the Food and Drug Administration approvals this year. This market has become more competitive in the past few years, increasing the potential for reduced costs and broader patient accessibility. Currently, these products are often expensive because they target specific conditions and cost on average $147,000 or more per year, making commercialization optimization particularly critical for success. 

At the same time 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.

This is important because personalized medicine has the capacity to detect the onset of disease at its earliest stages, pre-empt the progression of the disease and increase the efficiency of the health care system by improving quality, accessibility, and affordability.

These factors lay the groundwork for specialty pharmaceutical companies that are developing and commercializing personalized drugs for orphan and ultra-orphan diseases to pursue productive collaboration and meaningful partnership with a specialty pharmacy, distribution, and patient management service provider. This relationship offers manufacturers a patient-first model to align with market trends and optimize the opportunity, maximize therapeutic opportunities for personalized medicines, and help to contain costs of specialty pharmacy for orphan and rare disorders. 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.

Furthermore, and of concern to specialty pharmaceutical providers, is the opportunity to leverage a patient-first strategy in streamlining patient enrollment in clinical trials. This model also maximizes interaction with patients for adherence and compliance, hastens time to commercialization, and provides continuity of care to avoid lapses in therapy — during and after clinical trials through commercialization and beyond for the whole life cycle of a product. Concurrently, the patient-first approach also provides exceptional support to caregivers, healthcare providers, and biopharma partners.


Integrating Data with Human Interaction

When it comes to personalized medicine for the rare orphan market, tailoring IT, technology, and data solutions based upon client needs—and a high-touch approach—can improve patient engagement from clinical trials to commercialization and compliance. 

Rare and orphan disease patients require an intense level of support and benefit from high touch service. A care team, including the program manager, care coordinator, pharmacist, nurse, and specialists, should be 100% dedicated to the disease state, patient community, and therapy. This is a critical feature to look for when seeking a specialty pharmacy, distribution, and patient management provider. The key to effective care is to balance technology solutions with methods for addressing human needs and variability.  

With a patient-first approach, wholesale distributors, specialty pharmacies, and hub service providers connect seamlessly, instead of operating independently. The continuity across the entire patient journey strengthens communication, yields rich data for more informed decision making, and improves 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. 

As genome science becomes part of the standard of routine care, the vast amount of genetic data will allow the medicine to become more precise and more personal. In fact, the growing understanding of how large sets of genes may contribute to disease helps to identify patients at risk from common diseases like 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 pre-emptive action. 

What’s more, the increase in other forms of data about individuals—such as molecular information from medical tests, electronic health records, 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. 


Telehealth in the Age of Pandemics

During the COVID-19 pandemic, and beyond, it has become imperative that any specialty pharmacy, distribution, and patient management provider must offer a fully integrated telehealth option to provide care coordination for patients, customized care plans based on conversations with each patient, medication counseling, education on disease states and expectations for each drug. 

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

Each touchpoint has a care plan. For instance, a product may require the pharmacist to reach out to the patient after one week 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 patient’s daily routine, changes in behavior and so on. 

This approach captures relevant information in a standardized way so that every pharmacist and patient is receiving the same assessment based on each drug, which can be compared to overall responses. Information is gathered by an operating system and data aggregator and shared with the manufacturer, who may make alterations to the care plan based on the story of the patient journey created for them. 

Just as important, patients know that help is a phone call away and trust the information and guidance that pharmacists provide.


About Donovan Quill, President and CEO, Optime Care 

Donovan Quill is the President and CEO of Optime Care, a nationally recognized pharmacy, distribution, and patient management organization that creates the trusted path to a fulfilled life for patients with rare and orphan disorders. Donovan entered the world of healthcare after a successful coaching career and teaching at the collegiate level. His personal mission was to help patients who suffer from an orphan disorder that has affected his entire family (Alpha-1 Antitrypsin Deficiency). Donovan became a Patient Advocate for Centric Health Resources and traveled the country raising awareness, improving detection, and providing education to patients and healthcare providers.


Getting the right data to doctors is next hurdle for precision medicine

dna, genomics

The future of precision medicine will come only as quickly as doctors can pick out clinically useful information from the genetic data being gathered on their patients.

3D Printing Makes Medical Devices More Personal

Personalized
medicine is a major trend in pharmaceutical R&D—and it’s transforming the
way we think of therapeutics. Unlocking the secrets of the genome has made it
possible to create treatments for disease that are more suited to the
individual. But personalized medicine isn’t a concept that only applies to drug
therapies. It is also highly relevant in the area of medical devices.

Many patients
depend on medical devices to help them recover from or manage diseases and
medical conditions. These devices can range from cranial implants to
pacemakers. And, as with one-size-fits-all therapeutics, even the best medical
devices have not always worked as hoped for every patient. However, the
personalized approach allows for tailoring certain devices to better serve the
individual.

Medical
devices and prosthetics

The advent of additive manufacturing (more commonly known as 3D printing) has been one of the key developments in enabling personalized medical devices. Previously, it wasn’t realistic to expect manufacturers to produce highly customized versions of one basic type of medical device. But 3D printing makes the process much quicker and more affordable, and can provide a design to fit the patient perfectly. ConforMIS custom knee implants, for instance, use 3D bone scanning and printing technology to produce the implant, even printing custom tools for the surgeon to use in the procedure.

3D printing is also helping to make prosthetics that are more effective and better suited to the patient. In a journal article published in Procedia CIRP, which includes a case study of a prosthetic arm, the authors wrote that: “Personalized medicine will allow for a reduction of rejection levels, an increase of patient’s quality of life and to a reduction or a delaying of downstream problems.”

Bioprinting

Although 3D printing actual human organs is still only a dream, it is not completely the stuff of science fiction anymore. The more delicate and still-developing version of 3D printing known as bioprinting is a process of recreating tissue for a patient. It involves using “bio-inks” and 3D printing techniques to print structures made of biomaterials and cells. In time, bioprinting could replace autografts. And, perhaps one day, people in need of an organ replacement will be able to turn to bioprinting rather than waiting for a donor match.

Models

3D printing is
also being used to create precise models, which is another way to make medicine
more personalized. A unique model replica of a patient’s organ can be used for
diagnostic purposes or to help doctors prepare for a surgery. Models like this
also have incredible implications for research. If researchers can use 3D
scanning and printing to replicate the organ of a particular type of patient
suffering from a particular type of cancer, for example, then they can study
that model to learn more and perhaps develop better personalized treatments.

3D Printing Makes Medical Devices More Personal

Personalized
medicine is a major trend in pharmaceutical R&D—and it’s transforming the
way we think of therapeutics. Unlocking the secrets of the genome has made it
possible to create treatments for disease that are more suited to the
individual. But personalized medicine isn’t a concept that only applies to drug
therapies. It is also highly relevant in the area of medical devices.

Many patients
depend on medical devices to help them recover from or manage diseases and
medical conditions. These devices can range from cranial implants to
pacemakers. And, as with one-size-fits-all therapeutics, even the best medical
devices have not always worked as hoped for every patient. However, the
personalized approach allows for tailoring certain devices to better serve the
individual.

Medical
devices and prosthetics

The advent of additive manufacturing (more commonly known as 3D printing) has been one of the key developments in enabling personalized medical devices. Previously, it wasn’t realistic to expect manufacturers to produce highly customized versions of one basic type of medical device. But 3D printing makes the process much quicker and more affordable, and can provide a design to fit the patient perfectly. ConforMIS custom knee implants, for instance, use 3D bone scanning and printing technology to produce the implant, even printing custom tools for the surgeon to use in the procedure.

3D printing is also helping to make prosthetics that are more effective and better suited to the patient. In a journal article published in Procedia CIRP, which includes a case study of a prosthetic arm, the authors wrote that: “Personalized medicine will allow for a reduction of rejection levels, an increase of patient’s quality of life and to a reduction or a delaying of downstream problems.”

Bioprinting

Although 3D printing actual human organs is still only a dream, it is not completely the stuff of science fiction anymore. The more delicate and still-developing version of 3D printing known as bioprinting is a process of recreating tissue for a patient. It involves using “bio-inks” and 3D printing techniques to print structures made of biomaterials and cells. In time, bioprinting could replace autografts. And, perhaps one day, people in need of an organ replacement will be able to turn to bioprinting rather than waiting for a donor match.

Models

3D printing is
also being used to create precise models, which is another way to make medicine
more personalized. A unique model replica of a patient’s organ can be used for
diagnostic purposes or to help doctors prepare for a surgery. Models like this
also have incredible implications for research. If researchers can use 3D
scanning and printing to replicate the organ of a particular type of patient
suffering from a particular type of cancer, for example, then they can study
that model to learn more and perhaps develop better personalized treatments.

NIH Taps PhysIQ to Develop AI-Based COVID-19 Digital Biomarker

NIH Taps PhysIQ to Develop AI-Based COVID-19 Digital Biomarker

What You Should Know:

– physIQ has been selected by the National Institute of Health (NIH) to develop an innovative AI-based COVID-19 digital biomarker solution to address the COVID-19 pandemic.

– Early detection of COVID-19 decompensation in patients
is complicated by infrequent and non-specific clinical data. The first-in-kind
tool will collect and analyzes continuous physiologic data could provide early
clinical indicators of COVID-19 decompensation.

The National Cancer
Institute (NCI)
and the National
Institute of Biomedical Imaging and Bioengineering (NIBIB)
of the National Institutes of Health (NIH), have
awarded physIQ a contract to develop an
AI-based COVID-19 Decompensation Index (CDI) Digital Biomarker to address the
rapid decline of high-risk COVID-19 patients.

Why It Matters

Today, high-risk COVID-19
patients and their providers are finding out too late that in the disease
continuum they are getting sicker and need urgent care. The new early warning
system under development could allow providers to intervene sooner when a
COVID-19 patient is clinically surveilled from home and begins to worsen.
Rather than relying on point measurements, such as temperature and SpO2, that
are known to be lagging or insensitive indicators of COVID-19 decompensation,
continuous multi-parameter vital signs will be used to establish a targeted
biomarker for COVID-19.

Despite the technological advances and attention paid to COVID-19, the healthcare community is still monitoring patient vitals the very same way as we did in the 1800s,” said Steven Steinhubl MD, Director of Digital Medicine at Scripps Translational Science Institute (STSI) and a physIQ advisor. “With the advances in digital technology, AI and wearable biosensors, we can deliver personalized medicine remotely giving caregivers new tools to proactively address this pandemic. For that reason alone, this decision by the NIH has the potential to have a monumental impact on our healthcare system and how we manage COVID-19 patients.”

COVID-19 Decompensation Index (CDI) Digital Biomarker Development

PhysIQ will develop and validate a CDI algorithm that builds off existing wearable biosensor-derived analytics generated by physIQ’s pinpointIQTM end-to-end cloud platform for continuous monitoring of physiology. The data will be gathered through a clinical study of COVID-19 positive patients in collaboration with the University of Illinois Hospital and Health Sciences System (UI Health) and build upon work already in-place for monitoring COVID-19 patients convalescing at home.

In the development phase of this project, physIQ and its clinical partner will monitor participants who are confirmed COVID-19 positive, whether recovering at home or following discharge from the hospital. During the validation phase, physIQ will evaluate lead time to event statistics, decompensation severity assessments, and the ability for CDI to predict decompensation severity.

“The application of the CDI may provide a universal indicator of decompensation,” said Karen Larimer PhD, ACNP-BC, study PI and physIQ’s Director of Clinical Development. “Application of this technology could detect COVID-19 decompensation and prevent hospitalization or morbidity events in both scenarios.”

The study is designed to capture data from a large, diverse
population to investigate CDI performance differences among subgroups based on
sex/gender and racial/ethnic characteristics. This project will not only enable
the development and validation of the CDI, it will also collect rich clinical
data correlative with outcomes and symptomology related to COVID-19 infection.

This index will build on physIQ’s prior FDA-cleared, AI-based multivariate change index (MCI) that has amassed more than 1.5 million hours of physiologic data, supporting the development of this targeted digital biomarker for COVID-19. This will enable new research and further insight into using digital health to advance the public health response.