Rare diseases: although statistically uncommon, their burden is significant.
Between 25 and 30 million Americans live with rare diseases. These diseases include cystic fibrosis, muscular dystrophy, sickle cell disease, hemophilia, and as many as 7,000 other rare conditions. Although research has come a long way when diagnosing, treating, and preventing rare diseases, 95 percent of these conditions still have no treatments.
The rare disease drugs that exist—sometimes referred to as “orphan” drugs—are often costly and complex. As a result, adhering to treatment plans and taking medications as prescribed is challenging for many rare disease patients. The negative consequences of that non-adherence can be difficult or even deadly.
Let’s talk about how pharmaceutical companies can support patients with rare diseases to improve awareness, affordability, adherence, and outcomes.
How Can Pharma Companies Support Patients with Rare Diseases?
Patients with rare diseases don’t always get the support they need and deserve, but drug manufacturers can change that with the right approach. Learn five ways pharma companies can support patients living with rare diseases:
1. Commit to ongoing drug development
With so few treatments available for rare diseases, innovation is essential. That’s why first and foremost, pharma companies can support patients with rare diseases by committing to ongoing drug research and development.
To do so, many organizations partner with rare disease advocacy groups to learn about patients’ needs and advance the drug development process. Some pharma companies take it a step further by developing grant programs to support independent research in areas with unmet medical needs.
2. Provide patient and prescriber education
Education is another area in which pharma companies can lend their support. Because so many rare disease therapies are new and emerging, awareness is often limited. As such, proactively raising awareness and educating prescribers is critical to ensure patients living with rare diseases have access to treatments as they become available.
Patient and caregiver education is perhaps even more crucial. Once started on a rare disease therapy, pharma companies can provide educational resources to help patients learn about their disease and prescribed treatment. Ensuring that patients know how to properly administer rare disease drugs and manage any associated side effects is key.
3. Improve access to specialty drugs
Improving access to specialty drugs is another way that pharma companies can support patients with rare diseases. Difficulty accessing and/or affording rare disease therapies can lead to gaps in care such as medication non-adherence. In some cases, patients with rare diseases may never fill their prescriptions at all.
To improve access (and in turn, medication adherence), many pharma companies offer bridge programs or free good programs for patients experiencing coverage gaps. Additionally, pharma companies can improve patient access by ensuring that a breadth of specialty pharmacy networks offer drugs for treating rare diseases.
4. Use AI to predict patient non-adherence
The right resources can make all the difference when it comes to rare disease patient support programs. Take artificial intelligence (AI) for example. AI technology optimizes patient support programs by predicting which patients are not only at risk for non-adherence, but also most likely to respond to interventions.
By accurately predicting which rare disease patients are at risk of not beginning therapy or becoming non-adherent, pharma companies can deploy targeted, cost-effective interventions to the patients who are likely to benefit from extra support.
5. Intervene with personalized outreach
After using AI to predict patients who are most likely to become non-adherent, pharma companies can use the same technology to intervene with personalized patient engagement to have the greatest impact.
How? It’s easy: The AI predicts the optimal intervention channel, messaging, and timing for each patient. That means patients with rare diseases who need higher-touch support won’t be overlooked, and resources won’t be wasted on those who would benefit from a simple text message reminder.
AI is the Answer: Supporting Rare Disease Patients with Smart Data
These are just a few ways pharma companies can provide much-needed support to patients living with rare diseases. AllazoHealth’s AI engine— which produces smart data—helps overcome adherence barriers to encourage therapy initiation, increase days on therapy, improve patient engagement, and optimize medication use.