Donovan Quill

, defined as a disease that affects less than 200,000 individuals, impacts a wide array of populations. These diseases include a spectrum of disorders, including alpha-1 antitrypsin deficiency (alpha-1), lymphangioleimyomatosis, tuberous sclerosis, pulmonary alveolar proteinosis, and hereditary interstitial lung diseases.

The value of a patient-first approach and telehealth for addressing the complex health care coordination needs of patients with rare pulmonary diseases has never been more critical than during the COVID-19 pandemic. In fact, the pandemic has presented several issues facing patients, researchers, providers, and drug manufacturers in the rare and orphan disease market, including the high cost of clinical trials and patient recruitment.

Alpha-1 patients have been challenged to get much-needed support and education around their prescribed therapy and disease state, while also dealing with elevated stressors from being immunocompromised, conscious of infection, and hypersensitive to viral threats.

A patient-first approach to care management offers these patients a proactive, process-driven telehealth program that mitigates the transmission of infection, such as coronavirus. These strategies ensure care continuity across the entire patient journey with robust communication to yield rich data that clinicians can use to make more informed decisions and improve the overall patient experience.

A patient-first focus addresses all variables around collecting data, while maintaining frequent communication with patients and their families to ensure compliance and positive outcomes.

To highlight the level of complexity involved in treating rare pulmonary diseases, let’s look at 

, a genetic condition that may result in serious lung disease in adults and/or liver disease at any age. This 

increases the risk of having chronic obstructive pulmonary disease (COPD), liver disease, skin problems, and inflammation of the blood vessels.

Pulmonary issues mostly occur in adults, whereas liver and skin problems tend to occur in adults and children. The age that symptoms begin and the severity of symptoms can vary depending on how much working alpha-1 antitrypsin protein (AAT)a person has. Symptoms may include shortness of breath and wheezing, repeated infections of the lungs and liver, yellow skin, fatigue, rapid heartbeat when standing, vision problems, and weight loss.

 for the treatment of alpha-1-related lung disease is augmentation therapy—also called replacement therapy. Augmentation therapy is the use of AAT from the blood plasma of healthy human donors to increase the alpha-1 levels circulating in the blood and lungs of Alpha-1 patients diagnosed with emphysema.

The therapy is administered by a weekly intravenous infusion and, until other therapies emerge, is considered ongoing and lifelong.

While augmentation therapy is considered the only specific therapy for alpha-1 lung disease, the treatment plan for lung-affected individuals with alpha-1 should also include the appropriate use of antibiotics; an immunization program, including viral hepatitis and influenza strains; reduction or elimination of environmental risk factors; appropriate inhaled medications; an exercise program; and oxygen, if needed.

 the progression of lung destruction by replacing the deficient protein. The therapy cannot restore lost lung function nor be considered a cure. But som evidence shows that augmentation therapy can reduce the frequency and severity of pulmonary exacerbations.

In the past, rare diseases have not attracted significant pharmaceutical investment, but this is changing. Large pharmaceutical companies have begun to focus on rare diseases due to government incentives and the increased likelihood that treatments for life-threatening or severely debilitating diseases will be effective.

 of all drugs in active R&D pipelines are now included in the rare disease category, presenting scientific and operational challenges to sponsors and clinical trial ecosystem participants, as well as the adoption of new strategies, operating models and processes.

In today’s complex environment, a patient-first approach is more effective because it relies on a team of experts who bring a specific understanding to each patient’s condition to provide effective therapy and care management. This approach transcends the standard commercialization focus that too often fails to be cost-effective.

With a patient-first strategy, pharmaceutical manufacturers and their clinical research professionals can build a commercialization team that is open, engaged, and empathetic. Patient-first strategies offer targeted programs and services that deliver specialized expertise that transcends the scope of capabilities provided by traditional, legacy care organizations, which are often overly focused on scale alone.

A patient-first approach provides comprehensive, best-in-class services designed to maximize therapeutic opportunities for the rare disease community, including counseling, guidance, and education based on patient and caregiver needs.

A specialty pharmacy and patient management organization that takes a patient-first approach uses tools to enable in-home clinical services, direct-to-patient support, and remote monitoring for keeping clinical trials on track. These strategies shorten the time from the clinical trial to commercial drug access. In less than a month, a patient-centric program can transition over 90% of those patients.

Whereas traditional models are built for scale, a patient-first approach customizes services for small patient populations, delivering expertise to overcome the limitations of legacy care models and providing cost-effective programs. This includes financial advantages, assurance that products are properly and promptly distributed, and patient services designed to ensure compliance and quick, accurate reimbursement processing.

Integrated telehealth features have enabled patients to get the products delivered without going to the physician’s office during the pandemic. As a result, the trials conducted during this time had significantly more patients involved, despite the national lockdown.

Clinical research professionals and manufacturers know they have the support to develop a drug and comprehensive program based on specific needs despite disruption while dedicated clinical teams can seamlessly eliminate treatment gaps for the patient.

Choosing a Patient-First Management Partner

When choosing a specialty pharmacy and patient management partner, it’s important to ensure that they offer a suite of comprehensive services tailored to maximize the therapeutic opportunities for the treatment of rare and orphan disorders. A patient-first approach can provide the trusted path for patients and other stakeholders and add much-needed support for the patient’s family and caregivers. This enables them to becomemore engaged and take ownership, leading to a stronger partnership and better patient care.

The partner’s telehealth solution should be designed to streamline patient enrollment, maximize interaction with patients for adherence and compliance, and provide continuity of care to avoid lapses in therapy. It should rely on dedicated team members who have expertise in every aspect of the patient’s drug and can address every question and concern from patients, pharmacists, physicians, providers and payers.

Effective specialty partners demonstrate expertise in navigating the insurance landscape and prior authorization process, as needed, and know how to monitor and encourage compliance. Look for a partner with dual accreditation from the URAC for compliance with specialty pharmacy and the Accreditation Commission for Health Care for specialty pharmacy services. This demonstrates commitment to providing quality care and services to these patient populations.

As the health care system continues to respond to the challenges of COVID-19, a patient-first approach to rare and orphan disease management helps stakeholders overcome key challenges around the complexity of these conditions and the patients’ daily lives.

It’s not simply about protecting already vulnerable patients from the virus, but also empowering them in their own health journeys.

Articles

The value of a patient-first approach and telehealth for addressing the complex health care coordination needs of patients with rare pulmonary diseases has never been more critical than during the COVID-19 pandemic.

Optimizing Care for Orphan Diseases With Pulmonary Issues During COVID-19 Pandemic

 in October, this is an excellent time to continue recognizing and highlighting the role that pharmacists play in our health, communities, and lives. 

Regularly interacting more frequently with patients than prescribing medical professionals, pharmacists help patients avoid dangerous drug interactions and adverse health effects. They also serve as trusted health advisors and help patients understand their medication regimens in a way that optimizes compliance and health outcomes.

These hard-working professionals are in a unique position to improve medication safety because they have the time and clinical expertise to make a difference in the way patients manage chronic conditions for which they may be taking multiple medications.

What’s more, pharmacists represent an affordable and accessible health care resource. For many patients, it is probably easier to consult with a pharmacist than with a physician.

Often, pharmacists serve as an essential first point of care for many individuals. This is especially true for rare disease patients who may be taking their first steps on a long, complicated, and challenging journey to better health.

Pharmacists Understand Specialty Challenges

Eighty-two percent of those prescribed specialty therapies reported spending an hour on the phone coordinating care to begin their therapy, with more than one-third spending at least 3 hours.

 In fact, medication access and adherence challenges can be so onerous for specialty patients, up to 27% of prescriptions and therapies are abandoned or never started at all.

Traditionally, hub support programs and specialty patient workflows have been designed to mitigate significant challenges. But their potential impact is too often compromised by patient low awareness of services and minimal connectivity.

Many times, providers are cut off from the patient journey once they’ve prescribed therapy or enrolled the patient into the manufacturer-sponsored patient services. This has resulted in treatment delays of up to 8 weeks, which can lead to deteriorating health and worsening symptoms among these vulnerable patients.

Optimizing Care for Rare Orphan Disease Patients

Pharmacists working in a patient-first environment, which means they put the needs of every patient at the center of the care process, help to ensure the best possible health outcome. By putting the patient first, specialty-focused pharmacists are better able to support patients so that they can achieve an optimal patient experience.

A patient and pharmacy management organization can also aid physicians in helping their rare disease patients overcome a number of complications, especially in dealing with insurance coverage, misdiagnosis, and lack of streamlined information.

Specialty patients, who often feel isolated and have difficulty finding a physician who understands their disease, need this added layer of support to find the right medication. Many find considerable reassurance when dealing with a pharmacist who offers disease-specific expertise.

These specialists help patients untangle challenges and provide much-needed, ongoing support. Furthermore, specialty pharmacists give physicians and specialists the opportunity to focus less on non-medical issues and more on the patient and efficiently finding the most optimal therapy.

They provide education, resources, and are there for medical specialists to provide additional insight for enhanced treatment, as well as a high level of understanding and knowledge that helps them transcend clinical and communication obstacles. As part of the care team, they play a key role in optimizing specialty treatment and improving clinical outcomes and quality of life.

It’s important to find a patient and pharmacy management partner that offers dedicated pharmacy teams with expertise in each therapy area. They can deliver an elevated level of personalized care that specialty patients and their families require to maximize the benefits of their therapy. These pharmacists provide ongoing support and peace of mind. What’s more, they are readily available for consultation and prepared to head-off safety issues.

Look for dedicated pharmacy teams that provide comprehensive resources to help physicians and staff meet the needs of their patients. They should also offer extensive knowledge of the insurance environment to ensure that patients begin therapy quickly and receive the best coverage option.

These professionals can also offer assurance to payers that they are doing the right thing for their patients. A focus on specialty disorders and personalized care programs optimizes care and maximizes therapy benefits.

Most importantly, patients and loved ones benefit from a patient and pharmacy management partner who understands how to improve patient quality of life through appropriate services and therapies. A patient-first model means ongoing communication and support whenever it’s needed for long-term well-being.

Specialty access barriers: How technology can help (pharmaceuticalcommerce.com)

Specialty pharmacists give physicians and specialists the opportunity to focus less on non-medical issues and more on the patient and efficiently finding the most optimal therapy. 

Pharmacists Optimize the Health Care Experience—Especially for Rare Disease Patients  

When it comes to maximizing therapeutic opportunities for the treatment of individuals with toxoplasmosis—a 

 parasite—a patient-first approach can make a critical difference. In the battle against one of the most common parasites in the world, pharmacists and other health care professionals understand the value of care coordination for optimizing compliance and outcomes from the moment patients are diagnosed.

Currently recommended treatment drugs for toxoplasmosis target the tachyzoite stage of the parasite and do not eradicate encysted parasites in the tissues. Treatment involves a combination of drugs for at least 6 weeks and—for some patients—at least 6 months, depending on clinical response.

For this reason, it’s important for pharmacists and other health care professionals to enlist the help of a patient management organization that specializes in care coordination for patients with toxoplasmosis. They can offer dedicated care coordination, help patients gain quick and affordable access to the prescribed drug, and support them through every step of their treatment.

Infection usually occurs by eating undercooked contaminated meat, exposure from infected cat feces or mother-to-child transmission during pregnancy. Toxoplasmosis may cause flu-like symptoms in some people, but most people affected never develop signs and symptoms. For infants born to infected mothers and for people with weakened immune systems, toxoplasmosis may cause serious complications.

Individuals with a competent immune system are able to eliminate much of the parasite within a few weeks after initial infection. However, parasites protected within tissue cysts can survive long term and may become reactivated if the individual becomes immunosuppressed.

Toxoplasmosis is a leading cause of death attributed to foodborne illness in the United States. More than 40 million men, women and children in the United States carry the 

 parasite, but very few have symptoms because the immune system typically keeps the parasite from causing illness.

 during or shortly before pregnancy, and anyone with a compromised immune system, should be aware that toxoplasmosis can have severe consequences. The 

parasite can cause a serious brain disease that can potentially lead to death, eye infections, miscarriage or stillbirth, and birth defects in babies exposed to the parasite in the womb.

Pyrimethamine is considered the most effective drug against toxoplasmosis and has become a standard component of therapy.

 Dose-related suppression of the bone marrow caused by this drug can be mitigated by concurrent administration of folinic acid (leucovorin), which protects the bone marrow from the toxic effects of pyrimethamine. A second drug, such as sulfadiazine or clindamycin, should also be included.

Toxoplasmosis in immunodeficient patients is often fatal if not treated. Treatment is recommended for at least 4-6 weeks beyond the resolution of symptoms, but can take at least 6 months for these patients. Relapses can occur in patients with AIDS and maintenance therapy is recommended until a significant immunologic improvement occurs in response to antiretroviral therapy.

A patient-first approach means providers and patients have one less concern when it comes to getting started on a medication. A care coordinator works with the patient to understand their insurance coverage, financial needs, and the financial assistance programs that they may be eligible for, which helps to ensure compliance and positive outcomes.

Patient-first enables health care professionals to focus more on improving the patient experience and enhancing quality of life for the patient. The best patient-first plan includes programs, services, and specialized expertise that meets the needs of patients with toxoplasmosis. This is where partnering with a patient management organization that uses patient-centric strategies can effectively guide care for this small patient population.

Finding the Right Patient Management Organization

“Patient-first” has become a buzzword for many specialty pharmacy organizations that, in reality, only focus on profits. Therefore, it’s important to find a partner that offers dedicated teams with expertise in each therapy area.

These experts deliver the constant, consistent, and personalized care that specialty patients and their families need. Team members offer peace of mind by being available as a trusted source of ongoing support. They can also be ready to anticipate challenges in a way that fosters compliance, which leads to improved outcomes and a long-term healthy patient.

With the right specialty patient management organization, just one phone call puts patients in touch with toxoplasmosis experts who can begin to address everything from medication delivery and insurance reimbursement to compliance and education. By delivering a proactive service experience tailored to each patient’s needs, these organizations create a reliable path to a better patient experience for patients with toxoplasmosis.

Donovan Quill is President and CEO of Optime Care.

CDC - Toxoplasmosis - Resources for Health Professionals

Toxoplasmosis - Symptoms and causes - Mayo Clinic

When it comes to maximizing therapeutic opportunities for the treatment of individuals with  toxoplasmosis, pharmacists and other health care professionals understand the value of care coordination.

Patient-First Approach Helps Pharmacists, Physicians Optimize Compliance and Outcomes for Patients with Toxoplasmosis

A growing understanding genomics and of how genetics drives health, disease, and drug responses in each patient is enabling better disease prevention strategies, more accurate diagnoses, safer drug prescriptions, and more effective treatments. Personalized medicine (PM) tailors care to each patient’s unique genetic makeup, which can improve outcomes and lower costs.

For device and drug manufacturers, PM provides an opportunity to develop agents targeted for specific patient groups that don’t respond to medications.

 But effective PM requires changes in practice patterns and management strategies for health care practitioners and for manufacturers in reimbursement, regulatory practices, and knowledge sharing. What’s more, they must overcome barriers to implementation to effectively individualize diagnoses and prognoses. 

PM will be especially relevant for the orphan drug market, which was projected in early 2020 to account for almost 40% of FDA approvals that year.

 More drug manufacturers are developing orphan drug products despite their financial value, which is sometimes limited and dependent upon the size of the targeted population that may benefit from a specialty drug. At the same time, this trend is making the orphan drug market more competitive and increasing the potential for reduced costs and broader patient accessibility. The products are often expensive because they target specific conditions. According to a recent IQVIA report, spending on orphan indications amounted to 11% of the $518 billion in the United States’ invoice spending in 2019. Furthermore, the average annual cost of treatment with an orphan drug was approximately $32,000, but about 39% of drugs with orphan indications cost more than $100,000 per year.

Taking a patient-first approach to PM will benefit drug manufacturers, helping them to optimize patient care and gain a stronger financial foothold.

Genetic diseases have been traced to specific genes and can be connected to defects in the proteins they create. Most of these diseases are rare, typically affecting fewer than 200,000 individuals in the United States or fewer than 1 in 2000 in the European Union. There may be as many as 7000 rare diseases affecting the United States population.

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 more personal. In fact, the growing understanding of how large sets of genes may contribute to disease helps identify patients at risk from common diseases such as diabetes and cancer, as well as heart conditions. This in turn enables physicians to personalize their therapy decisions and allows patients to better calculate their risks and potentially take pre-emptive action.

Moreover, the increase in other forms of data on individuals—such as molecular information from medical tests, electronic health records, or digital data recorded by sensors—more easily enables the capture of a wealth of personal information, as does the rise of artificial intelligence and cloud computing to analyze it. The challenge now is for hospitals, physicians, and pharmacies to keep pace with this vast repository of information, balancing its benefits while protecting patient privacy.

With a patient-first approach, wholesale distributors, specialty pharmacies, and hub service providers connect seamlessly under one roof, instead of operating independently. Continuity across the entire patient journey, from clinical trials to commercialization of products and beyond, strengthens communication; yields rich data, including genetic 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 adherence and positive outcomes. Serving as a single-source, fully integrated partner, drug manufacturers benefit from this approach by bringing a continuity of care that is critically missing from the legacy care model. The net result is that patients have a better experience, which is a critical component of a product’s value.

In the orphan disease space, many manufacturers have worked with multiple specialty pharmacy partners and an internal or external product hub, but this unique patient-first approach offers key advantages:

—When a manufacturer uses several specialty pharmacies, the payer will almost always have a distribution preference inside its network, which can force patients to change pharmacies and potentially disrupt care. The fully integrated, single-pharmacy, patient-focused model ensures that the patient has access to the right drug for a particular disease state. When there’s only one place to get this drug, insurance will cover it.

—Patients with rare and orphan diseases and their physicians must overcome massive hurdles in achieving any level of consistent care. When a new therapy is available on the market, switching to it can lead to physical, emotional, clinical, and economic challenges. These drugs can cost hundreds of thousands of dollars per patient per year, and insurance companies pay close attention to the value received for every dollar spent, prompting doctors and patients to think twice before considering a new therapy.

This unique, patient-first methodology specializes in helping patients and providers overcome reluctance to changing drugs by being a resource every step of the way. It gives them a competitive edge in addressing clinical and insurance challenges while solidifying relationships with physicians and patients.

—A core difference between the legacy and patient-first model lies in how pharmacies are compensated. With the legacy care model, pharmacies earn a margin on the products sold as they strive to keep millions of patients adherent to maintenance medications. When the focus is on the product instead of the patient, the pharmacy’s incentives can become misaligned. They’re focused more on monthly profits, which can lead them to attend to how much time hub service employees spend on the phone. Such incentives prevent pharmacies from being truly patient-centered and able to deliver the best possible care.

Addressing Human Need and Care Continuity

Patients with rare and orphan diseases require a high level of support and they benefit from high-touch service. It is important to work with a patient-first partner that offers a care team––including program manager, care coordinator, pharmacist, nurse, and specialists––that is 100% dedicated to the disease state, patient community, and therapy. This is a critical differentiator from other specialty pharmacies and hub service providers, which tend to push technology solutions that fail to address human needs and variability.

Manufacturers of every size can leverage this continuity of care for a streamlined, one-stop-shopping approach to optimize the patient journey. Furthermore, the longer a patient is adherent to treatment, the less frequently the manufacturer needs to invest in acquiring new patients. Also, this approach can help drive an adherence rate of nearly 90% for orphan and rare disease therapies, compared with industry standards of about 70% to 80% for specialty drugs and 50% for nonspecialty drugs.

In addition, a patient-first model can help manufacturers focus not simply on getting FDA approval, but on how the product affects patients’ lives. Most patients don’t fit into the right box for trials because they may be too sick, too healthy, or have comorbidities. This makes it difficult to identify them as trial participants, especially when they have not received a diagnosis because there has been no other form of treatment.

A patient-first approach builds awareness and provides education programs to aid in understanding patient groups, letting patients know there is support to develop a drug and a comprehensive program based on their needs. Such an approach adds a layer of patient advocacy, helps physicians better understand the patient journey, and creates a patient registry. Additionally, the patient-first approach assesses patients in terms of social determinants of health, such as education level, socioeconomics, lifestyle choices, and other clinical and nonclinical issues.

This level of information plays a role in the design of clinical trials and during commercialization of the drug. For manufacturers, it presents an opportunity to obtain patient-reported outcomes outside of the clinical trial protocol.

When the product enters the distribution phase, further patient information is captured and determinations are made about what is important to the patient regarding outcomes. This support helps patients transition to the product beyond the clinical trial, prevents lapses in coverage, and optimizes continuity of care.

A patient-first approach is about the best means of bringing a product to market, understanding payer coverage, and providing patient education about their disease; it serves as a guide for the manufacturer every step of the way. Once the product reaches market, a patient-first approach provides long-term health management, genetic counseling, and, if appropriate, a nutritionist throughout the clinical trial’s duration.

When it comes to adherence, a patient-first model is designed to proactively reach out to patients on a weekly or monthly basis to make sure they are taking their medication. It identifies whether the patient needs lab work; ensures physicians get the results; and makes sure the patient has access to a coordinator and physician, if needed, when they have issues with a product. This helps the clinical trial site, many of which are teaching facilities or doctor’s offices, and is the best means of removing the burden of educating patients around adherence and handling the dispensing and scheduling of medications.

A patient-first approach minimizes the daily impact of living with rare and orphan diseases by employing a single, dedicated team to provide comprehensive, best-in-class services tailored to maximize therapeutic opportunities. This improves outcomes, enhances patients’ quality of life, and rapidly advances research, even during the coronavirus disease 2019 pandemic.

Manufacturers that adopt a patient-first approach will gain the focus and customization needed for longterm success and be well positioned to capitalize on the benefits of personalized medicine for patients and other stakeholders.

is president and CEO of Optime Care in Earth City, Missouri.

Vogenberg FR, Barash CI, Pursel M. Personalized medicine: part 1: evolution and development into theranostics. P T. 2010;35(10):560-576.

OptumRx Drug Pipeline Insights Report. Optum. 2020. Accessed March 10, 2021. https://www.optum.com/content/dam/optum3/optum/en/resources/PDFs/2569650-orx-q2-2020-pipeline-insights-report-final.pdf

Orphan drugs in the United States. IQVIA. December 3, 2020. Accessed March 19, 2021. https://www.iqvia.com/insights/the-iqvia-institute/reports/orphan-drugs-in-the-united-states-rare-disease-innovation-and-cost-trends-through-2019

FAQs about rare diseases. National Institutes of Health: Genetic and Rare Diseases Information Center. Updated January 26, 2021. Accessed March 9, 2021. https://rarediseases.info.nih.gov/diseases/pages/31/faqs-about-rare-diseases

Medicine is getting to grips with individuality. Economist. March 12, 2020. Accessed March 19, 2021. https://www.economist.com/technology-quarterly/2020/03/12/medicine-is-getting-to-grips-with-individuality

Health care practitioners and manufacturers must change practice patterns and management strategies.

For Genetic Diseases, a Patient-First Approach Delivers More Precise Prognoses

The clinical trials market is projected to reach $65.2 billion by 2025, with oncology anticipated to be the fastest growing segment.

 The coronavirus disease 2019 (COVID-19) pandemic has exacerbated the high cost of these studies, as well as challenges surrounding patient recruitment. This is especially true for the rare and orphan market.

Thirty million Americans live with a range of 7000 rare or orphan diseases. Although many patients have already struggled with reduced care, isolation, and adherence challenges for most of their lives, self-isolation during the pandemic has affected their quality of life. These individuals now face elevated stressors resulting from immunosuppression, concern about potential infection, and hypersensitivity to viral threats.

Despite restrictions, patients continue to require a high level of therapy adherence support at a time when some pharmacies have reduced their hours. This can impede communication with physicians and other advocates, which has already been severely disrupted because of the need for social distancing.

Many drug manufacturers also report that patient enrollment in clinical trials has been compromised, prompting contract research organizations (CROs) and their partners to seek technology solutions that mitigate the impact of the pandemic and other challenges.

According to client needs, customizing IT, tech, and data solutions can help resolve these issues and improve patient engagement—from clinical trials to commercialization and adherence.

A patient-first approach is sharply focused on orphan diseases to better address the patient experience, outcomes, and clinical trial requirements. It is a model that focuses on and adapts services for small patient populations, with targeted programs and services that deliver specialized expertise beyond the scope of traditional legacy care organizations.

COVID-19 has catalyzed improved clinical trial solutions, with manufacturers seeking to get patients enrolled in clinical trials more rapidly to advance research goals, including goals involving the pandemic. However, for other conditions, clinical trials lag in response to COVID-19 restrictions, making it crucial to identify innovative ways to move trials forward.

Tools that enable home-based clinical services, direct-to-patient support, and remote monitoring offer critical value for keeping clinical trials running. The FDA published guidance on managing clinical trials during the pandemic in March 2020, with updates made as recently as January 2021. The guidance was offered to benefit the safety of trial participants, maintain good clinical practice, and minimize risks to trial integrity during the pandemic. Carrying out assessments by telephone or by virtual visits and offering additional safety monitoring for clinical trial participants who can no longer access an investigational product or site are among the suggested methods.

As a preferred care partner for manufacturers, the patient-first methodology provides a streamlined approach that includes a single-source, direct-to-patient distribution model, a more controlled patient experience, and national access to therapy. Products are properly and promptly distributed directly to the patient, and patient services are designed to ensure adherence and retention and alleviate the need to schedule an onsite visit to obtain clinical trial medication.

An unnamed pharmacy, distribution, and patient management organization for the treatment of orphan and rare disorders was involved in 2 clinical trials at the outset of the pandemic. This participation helped patients access products without going to the doctor’s office, with the help of telehealth to oversee the process. As a result, the trials had significantly more patients involved, despite the national lockdown, and the organization was able to ensure that patients and staff remained safe while participating in the clinical trials.

With a patient-first approach, the clinical trial sites, CROs, specialty pharmacy, physicians, patients, site coordinators, and care coordinators connect effortlessly, instead of operating independently. The continuity across the entire patient journey—beginning with clinical trials—strengthens communication, yields rich data for more informed decision-making, and improves the overall patient experience. This dedicated clinical team seamlessly manages the transition from clinical trial product to commercial product and ensures there are no treatment gaps for the patient.

A patient-first focus also addresses all variables around collecting data, while maintaining frequent communication with patients and their families to ensure adherence and positive outcomes. This approach benefits drug manufacturers by bringing care continuity that is critically missing from the legacy care model. As a result, patients have a better experience, which is critical for the product’s value.

In the specialty space, most manufacturers have worked with multiple specialty pharmacy partners and an internal or external product hub, but this unique patient-first approach offers key advantages:

When a manufacturer uses several specialty pharmacies and a patient in a clinical trial switches to the commercial drug, the payer will almost always have a distribution preference inside its network, forcing patients to change pharmacies—potentially disrupting care. The single pharmacy, patient-focused model finds the right drug for the patient. When there’s only 1 place you can get this drug, it will be covered by insurance.

Patients with rare and orphan disorders and their physicians must overcome massive hurdles in achieving any level of consistent care. When a new therapy is available on the market, switching to that new drug can lead to physical, emotional, clinical, and economic challenges. These drugs can cost hundreds of thousands of dollars per patient per year, and insurance companies pay close attention to the value received for every dollar spent, prompting doctors and patients to think twice before considering a new therapy. The patient-first methodology specializes in helping patients and providers overcome this reluctance. This provides a competitive edge in addressing clinical and insurance challenges.

A core difference between the legacy and patient-first model is how pharmacies are compensated. With the legacy care model, pharmacies earn a margin on the products sold as they strive to keep millions of patients adherent to maintenance medications. When the focus is on the product instead of the patient, the pharmacy’s incentives can become misaligned. They’re focused more on monthly profits, leading them to focus on how much time employees spend on the phone. These incentives prevent pharmacies from being truly patient focused and able to deliver the best possible care.

Patients with rare and orphan diseases require a high level of support and have much to gain from high-touch service. A care team, including program managers, care coordinators, pharmacists, nurses, and specialists, is 100% dedicated to the disease state, patient community, and therapy. This is a critical differentiator that improves the standard specialty pharmacies and hub service providers, which rely too heavily on technology solutions that fail to address human needs and variability.

Manufacturers of every size can leverage this continuity of care for a streamlined, one-stop-shop approach to optimize the patient journey. What’s more, the longer a patient is adherent to treatment, the more manufacturers will recognize the benefits of a branded program.

This patient-first approach also improves patient outcomes and can drive an adherence rate of nearly 90% for orphan and rare disease therapies, compared with industry standards of about 70% to 80% for specialty drugs and 50% for nonspecialty drugs.

Global clinical trials market projected to exceed $65 billion by 2025—shift towards personalized medicine drives market growth—ResearchAndMarkets.com.News release. Business Wire. August 1, 2019. Accessed November 20, 2020. 

https://www.businesswire.com/news/home/20190801005504/en/

Breining G. Rare diseases difficult to diagnose, cures hard to come by. Association of American Medical Colleges. April 11, 2017. Accessed October 14, 2020. 

https://www.aamc.org/news-insights/rare-diseases-difficult-diagnose-cures-hard-come

FDA. FDA guidance on conduct of clinical trials of medical products during COVID-19 public health emergency.Updated September 21, 2020. Accessed November 20, 2020. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/fda-guidance-conduct-clinical-trials-medical-products-during-covid-19-public-health-emergency

This model focuses sharply on orphan diseases to better address
the patient experience, outcomes, and requirements of each study.

Patient-First Approach Is Improving Access to Clinical Trials for Specialty Pharmacy

During the coronavirus disease 2019 (COVID-19) pandemic, patients with orphan and ultraorphan diseases have been challenged to get much-needed therapy adherence support. This means that 30 million Americans with a range of 7000 rare or orphan diseases—including Huntington disease, amyotrophic lateral sclerosis, Cushing syndrome, α1-antitrypsin, chronic immune thrombocytopenic purpura, and muscular dystrophy&mdash;have been at greater risk while self-protecting.

Many of these patients have struggled with reduced care, isolation, and adherence issues for most of their lives, but they now face elevated stressors because they are immunosuppressed, conscious of infection, and hypersensitive to viral threats. Adding to the challenge is that some pharmacies have reduced their hours. This impacts communication with physicians, which has already been severely disrupted due to the need for social distancing.

As an integral part of the care team, pharmacies serve as lifelines for these patients by helping to prevent lapses in continuity of care. For this reason, pharmacists who integrate with a telehealth solution can ensure that patients in rural locations—and those now living in self-imposed isolation&mdash;have consistent and reliable contact with pharmacy services during these unprecedented times.

A recent analysis found that telehealth visits increased 50% in March 2020, and a recent poll showed that more than 1 in 8 Americans engaged in a video consult with a health care professional in recent months.

Telepharmacy: Key Factor in Evolving Health Care Landscape

Accessing pharmacy services via telehealth is particularly important for addressing the unique health care coordination needs of patients diagnosed with a rare or ultrarare condition and, more importantly, the newly diagnosed patient. 

When telehealth is part of a larger personalized care plan tied specifically to a particular specialty drug, pharmacists can be better positioned to help their patients by providing the right plan to the right patient, including adherence tools specific to the patient’s needs. This gives the patient a better chance of avoiding adverse events, visits to the emergency department, or hospitalization. 

By partnering with a patient-first organization that integrates specialty pharmacy services, product distribution, and patient management into a dedicated program, pharmacists enable patients to receive the intense care, outreach, and dependable support they require to ensure positive outcomes. 

A patient-first care model provides a proactive, process-driven program that can educate the patient on potential risks to mitigate the transmission of possible infections such as COVID-19. The model should be grounded in customized care coordination and telehealth solutions as a strategy for fostering discussion between the patient and pharmacist. This is critical for understanding the patient’s needs, providing focus on the drug’s impact, and monitoring their overall health. 

By incorporating assessments and predetermined touch points each month, the care team is able to rapidly address adverse effects (AEs) and capture real-world evidence around the therapy, disorder, and patient’s well-being.

A single-source partner offers additional support in ensuring continuity of care that is absent in legacy care models. As a result, patients benefit from better care and more consistent experience. The patient-first model has been shown to lead to improved compliance and overall outcomes. 

Clinical care teams reach out to patients via videoconference or phone call to determine their level of understanding, health literacy, and status around a particular disorder. This way, they are able to help mitigate potential AEs, offer encouragement, and provide a level of emotional support to patients and their caregivers.

A telehealth option can be fully integrated for pharmacies to provide care coordination based on each patient’s specific needs, including, but not limited to, medication counseling, education on disease states, and expectations for each drug. These customized care plans are designed to better serve patients, caregivers, providers, and payers, to help overcome hurdles around the complexity of rare, orphan, and ultraorphan disorders. 

Finding the Right Patient Management Partner

Ideally, the right specialty patient management partner should be focused on personalized care with a focus on disorders affecting smaller patient communities. The key is to look for a suite of comprehensive services tailored to maximize the therapeutic opportunities for the treatment of these disorders and a patient-first approach that provides a trusted path for patients and all those involved in the treatment journey.

A patient-first model adds a much-needed layer of support for the patient’s family and caregivers, allowing them to become more engaged and take ownership in the care process, which leads to a stronger partnership and better patient care.

The telehealth solution should be designed to streamline patient enrollment, maximize interaction with patients for adherence, and provide continuity of care to avoid lapses in adherence. It should also offer dedicated team members who are up-to-date on every aspect of the patient’s drug and have the expertise to answer every question and concern from patients, pharmacists, physicians, providers, and payers. It is also important for them to have experience in navigating the insurance landscape and prior authorization process, as needed, and to know how to monitor and encourage compliance. 

Look for management solutions with dual accreditation from URAC for compliance with specialty pharmacy and the Accreditation Commission for Health Care for specialty pharmacy services. These designations demonstrate a commitment to providing quality care and services to specialty patient populations. 

Overall, the most effective patient-first care management solutions are designed to meet the needs of e

nt’s journey—from specialty drug manufacturers to pharmacists, caregivers, and physicians&mdash;in a way that is delivered from a single, central point of contact. 

 is president and CEO of Optime Care, a pharmacy, distribution, and patient management organization.

Breining G. Rare diseases difficult to diagnose, cures hard to come by. Association of American Medical Colleges. Published April 11, 2017. Accessed September 3, 2020. 

Telehealth basics for pharmacists during COVID-19 and beyond. American Pharmacists Association. Published May 20, 2020. Accessed September 3, 2020. 

https://www.pharmacist.com/article/telehealth-basics-pharmacists-during-covid-19-and-beyond

A recent poll showed that more than 1 in 8 Americans engaged in a video consult with a health care professional in recent months.