People with Rare Chronic Disease Benefit from Patient-First Approach to Specialty Drug Management   

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By Brandon Salke, Pharm.D., Pharmacist-in-Charge, Optime Care

In the United States, a rare disease is defined as a condition that affects fewer than 200,000 people, with the total number of rare disease patients estimated to be 25-30 million. While these diseases may be rare, the total number of people affected is significant. In fact, approximately 7,000+ rare diseases impact over 300 million people globally. 

The cost of specialty drugs is estimated to rise 21-24% annually over the next few years due to substantial direct and indirect economic burdens for the patient, unpaid family caregivers and the U.S. health system.  

In fact, there is a designated Rare Disease Day, Feb. 28, 2022, which provides an opportunity for everyone to learn more about rare diseases and help raise awareness not only about the challenges to patients, providers and payers, but also the value of innovative Specialty drug management solutions that are making a real difference.

Today, the adoption of a patient-first approach to administering and managing specialty medications has demonstrated substantial value: every decision across the care team is about doing what works best for the patient, enabling clinical teams to better support patients and family caregivers. This patient-first focus involves proactive counseling, guidance and education that is based upon individual needs. It helps to safeguard prescription accuracy, compliance and adherence to treatment, manages side effects and optimizes the therapeutic value of a specialty drug—all of which lead to enhanced clinical outcomes. 

Rare Disease Challenges 

Challenges to patients and their families include lack of access to disease-specific, specialized medical expertise, difficulty getting a diagnosis and barriers to attaining high-cost targeted medications, including gene/cell therapies. Significantly, the cost for one specialty medication used on a chronic basis is three times the average annual income for Medicare patients, placing prescription medications out of reach for many older adults or forcing them to choose between the medicines they need or buying groceries or paying rent. 

Too often, clinicians are unprepared to diagnose, treat and monitor patients with rare diseases. These shortcomings are due, in part, to the fact that rare disease clinicians encounter obstacles to gaining knowledge and experience in caring for such patients. Also, local expertise and guidelines can be scarce. 

Specialty-Focused Pharmacists

The Specialty Pharmacy (SP) environment faces rising costs of specialty drugs and emergence of ultra-high-cost specialty agents and cell/gene therapies for rare and orphan diseases. 

Specialty pharmacists have thorough training and expertise with high cost, high touch therapies for patients with chronic, complex diseases. The patient-first approach enables them to cultivate a deeper relationship with patients, offering personalized information, care insights and a higher level of support. 

 For example, a young patient with a rare condition who started a new medication. She was concerned about the unfamiliar side effects. The specialty pharmacist spent 40 minutes listening to all of her concerns and answering all of her questions, rather than simply listing the medication’s usual talking points. 

Together they came up with a plan that involved the patient, pharmacist and physician discussing the new medication in more depth and getting a second opinion. By the end of this process, the patient felt more comfortable with the new medication and was able to remain compliant. 

This type of positive experience is far more impactful than what many patients experience with a retail chain pharmacy where they place more focus on daily quotas rather than comforting vulnerable patients. 

Choosing a Patient-First Solution

The best patient-first Specialty drug management solution also offers an array of services which are available as a whole or chosen a la carte to meet particular needs:

  • Site of Care optimization (SOC), a strategy to lower costs associated with certain infused or injected drugs by encouraging the use of clinically appropriate, lower-cost care settings
  • Integration of both medical and pharmacy benefits, which helps to reduce costs, while ensuring a focus on improving patient outcomes, aiming for healthier, more productive individuals
  • Proprietary technology platform for real-time Rx data and analytics
  • National network of high-touch Specialty pharmacy and home health
  • Medication home or office delivery
  • Clinical support and patient education
  • Integrated real-time Rx fulfillment data for monitoring and transparency
  • Pharmacy accuracy and time-to-fill metrics 
  • Compliance and persistency reporting

The right solution also offers streamlined prior authorizations and appeals, data and analytics, enhanced patient care coordination and patient journey mapping. Patients who are satisfied with their care journey typically experience a higher quality of life, which in turn leads to more cost-effective care and improved clinical outcomes.