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


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.

Rare lung diseases, 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.

Focus on Alpha-1

To highlight the level of complexity involved in treating rare pulmonary diseases, let’s look at alpha-1, a genetic condition that may result in serious lung disease in adults and/or liver disease at any age. This diseaseincreases 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.

The specific therapy 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 ultimate goal of therapy is to slow or stop 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.

Specialty Pharma on the Rise

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.

Approximately 33% 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.

Mitigating Clinical Trial Disruption

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.

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