Picture a river cutting through a canyon. On one side stands a patient who just received a rare disease diagnosis after years of searching. On the other: the treatment that could change their lives. But, specialty medication barriers make the gap between prescribing and access seem insurmountable without help.
Pharmaceutical brands have an opportunity to build a bridge using upstream prescription engagement to help people diagnosed with rare diseases access medications and support resources that offer real hope.
After five or more years spent consulting with up to 12 specialists just to reach a diagnosis, roughly 30 million patients in America discover their rare disease journey has barely begun. While less than 10% of 7,000 known rare diseases have an approved treatment available, those numbers are changing rapidly. In recent years, the FDA has accelerated orphan drug decisions, with medications for rare diseases representing over 50% of approvals between 2020 and 2024.
Even when a therapy exists, specialty medication barriers create a chasm that can take patients months to cross. Often, cost is a significant barrier: Orphan drugs can average $186,000 per year, and some therapies exceed $2-4 million per treatment. For many patients, the barrier feels insurmountable.
Between diagnosis and treatment, patients can be thrown off course by several challenges.
Rare disease therapies often require a series of prior authorizations. A 2019 NORD survey found that 61% of patients diagnosed with rare diseases faced PA denials or delays. The pharmacy authorization may depend on documentation—such as genetic testing results or specialist confirmation—that requires other medical authorizations first. Initial denials are common, and appeals can take by weeks or months. Meanwhile, prescribers are drowning in administrative tasks, while patients watch a potential solution drift out of reach.
Given their high price and complex handling requirements, orphan drugs are often distributed exclusively through carefully chosen specialty pharmacy partners. These exclusive or limited distribution networks introduce more barriers. The dispensing specialty pharmacy must be identified, and the patient must coordinate delivery. There may even be a secondary medical PA requirement depending on how the drug is administered. Each handoff is a potential breakdown point. Patients often don’t know who to call or where they are in the process.
Out-of-pocket costs for such expensive medications can reach thousands of dollars. Typically, healthcare costs are 10 times higher for patients with rare diseases than for those with more common diagnoses. Patient assistance programs exist, but patients and their caregivers may feel overwhelmed trying to navigate support options. By the time patients understand which assistance programs they qualify for, momentum may be lost.
Most pharmaceutical patient support programs are built downstream—on the treatment side of the canyon, such as:
The problem is that patients don’t know how to get across on their own.
Building a bridge downstream and hoping patients will find it doesn’t work. Instead, pharmaceutical manufacturers need to link prescribers and patients to the right information at the right times, so they can navigate the process more effectively. That’s where TimelySM by DrFirst® makes a difference.
Upstream prescription engagement changes the journey by integrating actionable decision support in the prescribers’ workflow for prior authorization, pharmacy routing, and patients’ out-of-pocket costs. Then, minutes after a prescription is sent, patients receive a personalized text message from their provider with educational resources about the prescribed therapy, next steps, and direct links to enroll for financial assistance. Patients don’t have to search for help. Help finds them at the moment they need it most.
A campaign for an injectable specialty medication shows the difference. More than 22,000 newly prescribed patients were engaged through provider-facilitated messaging, achieving 75% engagement. The result: a 3.5% absolute incremental increase in first-fill rates.
Rare diseases are uncommon, but the struggle to access treatment is a daily reality.
After spending years searching for a diagnosis, patients deserve better than being left to navigate specialty medication barriers alone. They don’t need lifelines thrown from the far side of the chasm; they need support that meets them at the moment of prescribing and guides them all the way to treatment and long-term adherence.
Learn how Timely by DrFirst helps pharmaceutical brands accelerate therapy initiation for patients prescribed orphan drugs.