When we look at statistics that show thirty million people in the U.S. has one of seven thousand rare diseases, they may not seem rare. However, when we realize that almost ninety-five percent of patients with rare diseases are unable to access an effective therapy, we begin to understand the urgency to diagnose and treat these disorders.
According to a recently published article in MedCity News, it is anticipated that the “specialty” market will continue to grow throughout 2023. This growth will be driven by the expanded use of existing therapeutics as well as new drugs appearing on the market.
Through ever-increasing knowledge of rare diseases and with each scientific breakthrough, scientists are attempting to stay ahead of demand and develop novel therapies. We must also consider patients with several conditions requiring specialty drugs.
What Are Specialty Drugs?
They are drugs generally used in the treatment of Orphan, rare, or complex conditions.
Specialty drugs require special handling, administration, distribution, and monitoring such as patient care and clinical monitoring.
The high cost of specialty medication is its most prominent characteristic
About the Cost
Out-of-pocket costs have made access to some specialty drugs prohibitive. The cost has actually outpaced the U.S. GDP over the last decade. Insurance for coverage of specialty drugs depends on where the drug is administered.
For example, outpatients would receive coverage through their pharmacy benefits. However, if the drug is administered in a hospital setting, it is likely that the patient would be covered by medical benefits in the patient’s insurance coverage.
Alternative Site of Care Programs
Patients, as well as the payer, may be eligible for substantial savings through an alternative site of care program. These alternative programs provide support and convenience plus lower costs to the patients and their payers.
Funds provided by the manufacturer are available that will offset therapy costs. Copay coupon programs are available to assist with the high cost of medications. It is anticipated that carve-outs that move specialty drugs away from traditional prescription management will continue to be utilized.
Centers of Excellence
Specialty Pharmacy networks have wide appeal as specialty drugs are coordinated and dispensed by way of specialty pharmacies with patient support programs and enhanced storage and shipping capabilities.
These pharmacies are deemed Centers of Excellence. They assist with compliance to care coordination, minimize waste by controlling excess drug accumulation, and generally strive for optimal outcomes for patients.
Seeking a Cure
It is not inconceivable to envision that diseases such as muscular dystrophy, genetic blindness, sickle cell anemia, cancer, or blood disorders are not only treated but cured by gene therapies. With giant biotech and pharmaceutical companies leading the way, the concept will gradually become a reality.
About Gene therapies
Gene therapies are not being developed to “treat” diseases but to cure them. The therapies correct the underlying genetic abnormalities that cause the disease.
There are currently over nine hundred new drug applications that have been submitted to the FDA for approval in connection with investigative clinical trials.
The FDA has indicated that it will be approving between ten to twenty gene therapies every year.
Although the new genetic therapies signify life altering-medications they come at a steep price. Discussions are already underway between payers, manufacturers, and policymakers to optimize patient access to these products.
One option being considered is a loan-based product that will convert the upfront cost of expensive therapies into a payment plan with smaller increments.
Looking Forward
A glimpse into the future suggests that the market for gene and cell therapies may move beyond strictly focusing on rare disorders. The leading specialty pharmacy innovators have and will continue to address patient concerns about health and affordability.
Please note that the NOTD organization provides instructions for patients and their caregivers in a Rare Disease Database.
