Patient-First Approach Optimizes Care for Patients with Rare and Orphan Diseases

Updated on April 18, 2022
Smiling doctor looking at a patient on a wheelchair in hospital hallway

By Michelle Hefley, Co-Founder, Managing Partner and Chairman, Optime Care 

Across the United States, about 7,000 rare diseases affect more than 25million Americans. In recognition of April 26-30 as Patient Experience Week (PX Week), an annual event to celebrate the people who impact the patient experience every day, it’s important to acknowledge the unique needs of people with rare diseases. 

Rare diseases are difficult to diagnose, with many having non-specific or unusual symptoms that often progress slowly. What’s more, these patients face a number of challenges, including difficulty finding a doctor who can diagnose the disease and is knowledgeable about treatment, dealing with frequent misdiagnosis and medications that fail to work, a sense of being marginalized by the system or isolated by their disease state, and the staggering costs of treatment.  

A patient-first approach to Specialty drug management for these patients helps to maximize therapeutic opportunities. It also helps wholesale distributors, specialty pharmacies and Hub service providers connect seamlessly, instead of operating independently. 

Finding the Right Partner

Stakeholders should seek a patient-first Specialty pharmacy management partner that offers:

  • Timely use of telehealth 
  • Tailored IT, tech and data solutions based upon client needs to improve patient engagement — from clinical trials to commercialization and compliance
  • Comprehensive suite of Specialty Pharmacy (SP) services for specialty Rx manufacturers, providers and payers, including pre-commercialization, outsourced market access, wholesale, 3PL, limited distribution drug (LDD) fulfillment, and custom Patient Support and Hub services 
  • Greater understanding of the role of data in personalized medicine
  • An array of services offered as a whole or a la carte to meet each patient’s particular needs

It’s also critical to find a partner that offers innovative financial solutions, including custom copay assistance programs. This eliminates barriers to accessing high-cost therapies and ensures that patients receive the Specialty medications they need, when they need them most, regardless of financial status.

As a single source partner, a patient-first Specialty pharmacy management partner benefits drug manufacturers by bringing 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.

Making a Difference

The best patient-first care models include targeted programs, services and specialized expertise that transcend the limitations of traditional, legacy care organizations that often fail to fully address the needs of patients with orphan and rare disease.

Patient-first team members, including care coordinators, technicians, nurse managers and pharmacists, are able to be there for the patient whenever they need them, which is particularly important when altering or starting a new medication. These professionals draw on their expertise to manage the challenges around insurance and shipping, compliance, education and care coordination.

During Patient Experience Week, it’s important for more people to understand the challenges that rare disease patients face and how a patient-first strategy to Specialty drug management offers a more compassionate way to provide better services and support.

Cool Photos from Depositphotos

The Editorial Team at Healthcare Business Today is made up of skilled healthcare writers and experts, led by our managing editor, Daniel Casciato, who has over 25 years of experience in healthcare writing. Since 1998, we have produced compelling and informative content for numerous publications, establishing ourselves as a trusted resource for health and wellness information. We offer readers access to fresh health, medicine, science, and technology developments and the latest in patient news, emphasizing how these developments affect our lives.