Specialty Pharmacy Services
Rare and Genetic Disease Specialty Pharmacy
Accord Specialty Pharmacy serves patients with rare and ultra-rare conditions that require highly specialized medications, restricted distribution access, and individualized support. We understand the complexity of rare disease treatment and work closely with patients, caregivers, and specialists to remove barriers to therapy.
Conditions We Serve
Rare and Genetic Conditions
Duchenne Muscular Dystrophy (DMD)
A severe, progressive muscle-wasting disease caused by mutations in the dystrophin gene, primarily affecting boys. Deflazacort (Emflaza) is a corticosteroid approved specifically for DMD and is dispensed through specialty pharmacies with a dedicated patient support program.
Hereditary Transthyretin Amyloidosis (hATTR)
A rare, progressive disease caused by mutations in the TTR gene that lead to the buildup of abnormal protein deposits in the nerves and heart. Tafamidis (Vyndaqel, Vyndamax) and siRNA therapies are available through specialty distribution channels.
Gaucher Disease
The most common lysosomal storage disorder, caused by a deficiency of the enzyme glucocerebrosidase. Enzyme replacement therapies and substrate reduction therapies for Gaucher disease are exclusively dispensed through specialty pharmacies, often with manufacturer coordination.
Hereditary Angioedema (HAE)
A rare genetic disorder causing recurrent episodes of severe swelling in the skin, gastrointestinal tract, and airway. Prophylactic and acute treatments for HAE include monoclonal antibodies and plasma-derived therapies requiring specialty pharmacy access.
Neurogenic Orthostatic Hypotension
A condition characterized by a significant drop in blood pressure upon standing, often associated with Parkinson's disease and other autonomic disorders. Droxidopa (Northera) is the only FDA-approved treatment for symptomatic neurogenic orthostatic hypotension and requires specialty pharmacy dispensing.
Specialty Support for Rare Disease Patients
Restricted Distribution Access
Many rare disease therapies are available only through a limited number of authorized specialty pharmacies. Accord maintains enrollment in the distribution networks necessary to dispense these medications directly to patients.
Manufacturer Program Navigation
Rare disease manufacturers typically offer robust patient support programs with dedicated case managers, sample supplies, and co-pay assistance. Accord coordinates enrollment in these programs and serves as a consistent point of contact throughout the process.
Financial and Foundation Assistance
Rare disease medications can carry extraordinary costs. We help patients identify and apply for manufacturer patient assistance programs, disease-specific foundation grants, and government assistance options to reduce or eliminate out-of-pocket expenses.
Doorstep Delivery
Accord ships rare disease medications to patients in states where Accord holds an active pharmacy license. We coordinate delivery schedules to align with clinical appointments and infusion centers, and manage cold-chain requirements for temperature-sensitive therapies.
What We Dispense
Rare Disease Specialty Medications
Dedicated Medication Pages
Additional Medications
Vyndaqel
tafamidis meglumine
hATTR Amyloidosis OralVyndamax
tafamidis
hATTR Amyloidosis OralTakhzyro
lanadelumab
Hereditary Angioedema Self-InjectionBerinert
C1-inhibitor (human)
Hereditary Angioedema InfusionCerezyme
imiglucerase
Gaucher Disease InfusionCerdelga
eliglustat
Gaucher Disease OralNot seeing a medication? Call us at 1-855-222-6730 to confirm availability for your patient's specific therapy.
Frequently Asked Questions
Does Accord carry Emflaza (deflazacort) for Duchenne muscular dystrophy?
Yes. Accord Specialty Pharmacy dispenses Emflaza (deflazacort) for Duchenne muscular dystrophy through the PTC Therapeutics patient support program. Our team assists with prior authorization, enrollment in the manufacturer support program, and coordinates ongoing refills with the patient's neuromuscular specialist.
Can Accord dispense Northera (droxidopa) for neurogenic orthostatic hypotension?
Yes. Accord carries Northera (droxidopa) for symptomatic neurogenic orthostatic hypotension associated with Parkinson's disease and related autonomic disorders. We manage REMS program requirements and prior authorization, and assist with financial assistance enrollment.
How does Accord access restricted or limited-distribution rare disease medications?
Accord maintains enrollment in the limited distribution and specialty pharmacy networks required to dispense rare disease medications. When a patient is referred, we confirm that we are an authorized dispenser for that specific therapy and coordinate directly with the manufacturer hub if required.
What financial assistance is available for rare disease medications?
Most rare disease manufacturers offer patient assistance programs or free drug programs for patients who cannot afford their medications. In addition, disease-specific nonprofit foundations often provide grants to help cover medication costs. Accord helps patients identify and apply for all available resources at the time of enrollment.
How do I refer a patient with a rare disease to Accord?
Contact our team directly at 1-855-222-6730 or fax a referral to 386-385-7871. For rare disease patients, it is helpful to include the diagnosis, ICD-10 code, the specific medication, and any relevant insurance information. Our team will contact you within one business day to confirm next steps.
Does Accord work with caregivers for pediatric rare disease patients?
Yes. Many rare disease patients, including those with Duchenne muscular dystrophy and lysosomal storage disorders, are children. Accord works closely with parents and caregivers to coordinate care, explain the enrollment process, and ensure medications arrive on time and in the proper condition.
For Patients and Caregivers
We Understand Rare Disease
Getting started on a rare disease therapy can be overwhelming. Call our team and we will walk you through every step, from enrollment to first delivery.
Call 1-855-222-6730For Prescribers
Refer a Patient to Accord
Call us directly at 386-456-3000 or fax a referral to 386-385-7871. We are familiar with the enrollment requirements for rare disease therapies and handle the process from start to finish.
Call 386-456-3000