A New Frontier in the Fight Against Rare Disease

Approximately 30 million people in the United States—1 in 10 Americans—have been diagnosed with a rare disease, approximately two-thirds of whom are children.1 Rare disease drugs and cell or gene therapies to treat these conditions offer new hope for people living with a rare condition and are among the fastest growing areas of the drug pipeline.

Although these treatments have the potential to save lives, they also represent the most dynamic and costly area in medicine. Complex in their administration, handling and care, these high-touch therapies require customized management and services beyond what traditional pharmacies can provide.

Serving Rare Disease Patients

Rare disease specialty pharmacies offer customizable pharmacy and patient services to make it easier for people living with rare, complex, and often isolating conditions to access the therapy, care, and resources they need. Patients prescribed advanced therapies may face unique difficulties, such as upwards of a 5-year diagnostic journey, challenges with social determinants, and access to physicians and specialists familiar with their disease.

Further, advanced therapies are often complex to manage and have a very high cost. Because of this, patients with rare diseases require higher levels of service and support.

Working with a rare disease specialty pharmacy enables a single point of contact to serve as the patient’s and caregiver’s liaison to services, education, and support. This same contact forms a relationship with the patient’s provider or prescribing office, and ensures that these providers, patients, and families have the additional support they need by coordinating a team of clinical access and reimbursement specialists, pharmacists, and ancillary health providers.

Through tailored programs for each therapy and disease, along with concierge patient-centric service, rare disease specialty pharmacies streamline the treatment process and support so patients can focus on achieving their best health care outcomes.

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BioPharma Global is a mission-driven corporation, operating like a not-for-profit, dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with a high unmet medical need. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

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