Skip to content

Future Treatments Excite Rare Disease Patients & Providers

On this Rare Disease Day, patients, clinicians and advocacy organizations around the country are elevating the profile of rare conditions. One such condition is IgG4-related disease.

IgG4-RD is an immune-mediated fibroinflammatory disorder that disrupts the immune system, causing tumor-like masses, enlargement and other organ damage. This condition has seen significant strides in education and potential treatments since its discovery two decades ago. But timely access to appropriate care is critical, a new clinician-authored white paper from the Alliance for Patient Access explains.

Treatment plans for IgG4-RD are currently a mix of off-label medications to minimize symptoms. But these treatments can introduce a variety of side effects and face coverage challenges. There is hope for patients, however. “Addressing Access Barriers for IgG4-RD Patients” highlights that, like advocacy efforts, innovation has also focused on this rare condition. New medications are being developed specifically to treat IgG4-RD.

Looming Access Barriers

But many patients, patient advocates and clinicians are concerned that insurance barriers could mean these innovative treatment options may not be easily accessible. IgG4-RD patients already face barriers to getting off-label treatments.

New treatments may face roadblocks like:

  • New-to-market exclusions, where access to new medications may be limited, despite rare disease patients experiencing lengthy diagnosis journeys and off-label treatments.
  • Utilization management tactics, such as prior authorization, step therapy or non-medical switching, which can delay or deny patients’ access to their prescribed medication.
  • Copay accumulator adjustment programs, where copay cards no longer count toward a patient’s deductible and leave patients to bear heavy financial burdens.

These challenges could compound the struggles that IgG4-RD patients already face. Mental health challenges, low awareness and limited specialists can also complicate a patient’s journey. Many rare diseases, like IgG4-RD, are relatively unknown. Physicians may not be aware of these conditions, and patients may have to visit specialist after specialist before obtaining the right diagnosis.

Solutions for Rare Disease Patients

Innovation and awareness are the two greatest tools for improving rare disease patients’ lives. Worldwide events like Rare Disease Day go a long way in raising awareness. Advocates should channel that same energy into informing policy that encourages innovation and keeps innovative treatments within patients’ grasp.

Learn more about IgG4-RD in “Addressing Access Barriers for IgG4-RD Patients.”

Related Articles