More than 10,000 diseases are categorized as rare, yet only about 5% have available treatments – which often are limited, complex, and expensive. By definition, a rare disease affects fewer than 200,000 Americans, although for some diseases, that number is less than 100.
Recent scientific breakthroughs, including gene therapy, have enabled the therapeutics now on the market. These products treat some of the more common rare diseases, including sickle cell disease, hemophilia, and Duchenne muscular dystrophy.
“Rare diseases typically have a genetic component. The relatively few therapies that do exist can be complicated to administer and require super-specialized handling,” said Mark Jacob, senior director for Accredo by Evernorth’s Rare Diseases Therapeutic Resource Center (TRC). “The role that a specialty pharmacy plays in handling these therapies is critical and requires specialized expertise to ensure the best possible patient outcomes.”
Accredo, which is Evernorth’s industry-leading specialty pharmacy, offers decades of condition-specific expertise, a full breadth of clinical support, and a wide variety of patient services. “We were and still are the only specialty pharmacy to gain access to Luxturna, the first commercially available gene therapy in the United States, which was approved by the Food and Drug Administration in 2017,” Jacob said. “We’ve continued to grow and remain the industry leader in dispensing exclusive and limited-distribution drugs, many of which treat rare diseases.”
Delivering exceptional care to patients with rare diseases
Evernorth is a leader in supporting specialty patients, led by Accredo, where 15 condition-specific TRCs collectively employ more than 800 specialty pharmacists.
Providing individual customized support to each patient is crucial, said Drew Mascolino, managing director for product strategy at Accredo. “The complexities of these treatments are significant, especially those that treat rare diseases, and continue to evolve. Our patient-specific care model, enabled by our TRCs, continues to evolve as well. It’s in our DNA.”
Within individual TRCs, specialty pharmacists, nurses, dietitians, social workers, patient care advocates, and physician engagement advisors focus on specific conditions. This model enables them to develop a heightened understanding of these specific diseases, their treatments, and factors that can impact therapy outcomes.
Many specialty medications – including those used to treat rare diseases – are injected or infused. When a medication is safe for home administration but requires patient education, an Accredo clinician will provide step-by-step instructions. In many instances, Accredo sends one of its 600 field nurses, who are available to patients in all 50 U.S. states, to administer infusions in the patient’s home. Each of these patients is assigned a nurse who stays in their home throughout each infusion, monitoring their vital signs while getting to know them and their families.
Supporting providers caring for patients with rare diseases
Another way Evernorth and Accredo support patients is by assisting their doctors by executing the care plans they prescribe for their patients. Accredo pharmacists work closely with doctors to ensure effective treatment and enhanced patient outcomes. This includes everything from supporting treatment plans to helping manage adherence to addressing any concerns such as efficacy and social determinants of health.
Pharmacists reach out proactively when necessary, and providers have access to dedicated clinical phone lines and an online portal that lets them check prescription status, track shipments, review necessary information, chat with Accredo specialists, and more.
In addition, Accredo’s physician engagement team supports prescribers of highly complex medications, including treatments for cancer, neurological diseases, pulmonary conditions, and bleeding disorders. The team includes account executives who meet in person with providers, their staffs, and patients.
How Evernorth’s advanced infrastructure keeps deliveries on track
The complex, delicate medications that Accredo dispenses can require specialized infrastructure, such as ultra-cold freezers. “Some of these medications must be stored at -112 degrees Fahrenheit,” Jacob said. “We have some that require shipping at -220 Fahrenheit. That requires a special container with its own electrical and cooling system.”
In addition to its dynamic cold storage capabilities, Accredo uses a proprietary IceGen algorithm for cold-chain medication distribution that dynamically optimizes packaging configurations, including the number of cooling packs needed to ensure each prescription’s safety, stability, and integrity. This patented technology specifies how to pack each order based on weather forecasts throughout its travel route, from the time it leaves the manufacturer until it reaches its destination.
Evernorth’s nationwide footprint includes about 40 care delivery sites that fill and distribute prescriptions. When one of Accredo’s care delivery sites faces a severe storm or another potentially impactful event, Evernorth’s advanced technology determines how to reroute every order, helping to ensure quick, safe, and on-time delivery.
Four sites across the country contain clean rooms, where pharmacists and technicians wearing protective suits prepare a number of complex medications, working in environments built and maintained in accordance with stringent sterilization and regulatory requirements.
Case study: Caring for patients with hereditary angioedema
About 6,000 people in the United States live with hereditary angioedema (HAE), a disease that causes painful and sometimes life-threatening episodes of swelling in the intestinal tract, airway, face, and elsewhere. Current medications can treat acute symptoms or prevent their onset, and multiple potentially curative therapies are in clinical trials.
Accredo recently launched TRC Plus for Hereditary Angioedema. Its patient-centered care includes utilizing personalized text messaging software to help patients stay on track with their medication and internet-connected syringe disposal devices that track medication adherence and provide enhanced monitoring and alerts to Accredo nurses with deep knowledge of the drugs that treat HAE.
Specialized pharmacists monitor and conduct clinical assessments and counsel each HAE patient individually throughout their care journey, sharing expertise in medications and the disease itself. The pharmacists take part in collaborative clinical reviews of patient cases, enabling them to offer data-based recommendations to prescribers.
“Caring for patients is at the heart of what we do at Evernorth,” Jacob said. “Each of our 15 TRCs at Accredo is staffed with a variety of experts who help patients and their physicians whenever they need us.”
