Some new specialty treatments for patients with rare and chronic diseases are nothing short of transformational, allowing many to live longer and healthier lives. However, these new treatments are often expensive, creating challenges for health insurance companies to design and implement policies that offer patients access to the care they need at an affordable price.

Shifting regulatory environments, changing economic conditions, and new medical discoveries all play a role in shaping payer polices relative to these specialty treatments. Insurers now face greater challenges in developing drug policies that cover the needs of a wider range of patients. Some of these policies shift costs to patients and/or use more restrictive formularies, which may cause unintended medical problems, such as adverse reactions to a new medication.

As President and Founder of the Immune Deficiency Foundation (IDF), the national patient organization dedicated to improving the diagnosis, treatment, and quality of life for people with primary immunodeficiency diseases (PI), I have seen firsthand the impact that restrictive insurance policies can have on patients’ medical and financial wellbeing.

Primary immunodeficiency diseases represent a group of more than 250 rare disorders in which the body’s immune system is missing or functions improperly. Many of our patients require lifesaving immunoglobulin (Ig) treatment on an ongoing basis throughout their lifetimes — in contrast to others who may only use Ig for a short time period.

In recent years, as payers work to manage costs and implement policies based on treatment quality and outcomes, our patients have increasingly faced payer policies that restrict their access to Ig. Some patients who were doing well on a specific Ig replacement product have been required to delay treatment in order to reconfirm a diagnosis that had already been confirmed by the appropriate standards of care or switch to a different policy-mandated product instead. These delays in treatment or product switching can pose significant health risks to patients with PI, causing serious illness that can lead to substantial health care bills.

Surveys of patients with PI that IDF has conducted over the years have shown that a change in Ig replacement therapies can cause a range of health problems, including fever, disabling headaches and body aches, as well as sudden drops in blood pressure, anaphylaxis, thromboembolic events, and even death.

The high cost of addressing these unintended consequences can quickly outstrip the cost savings a payer may have realized by imposing the restrictive policy, not to mention putting the patient’s health and life at risk.

Thankfully, I have also seen firsthand the benefits insurance companies can realize when they collaborate with patient organizations like IDF as they draft new policies or alter existing ones. This type of collaboration is a win-win for both insurers and patients.

Touting A Success Story

In early 2014, several of our patients alerted us that new policy guidelines from health plans provided by Health Care Service Corporation (HCSC) required a group of patients with PI to halt Ig infusions to reconfirm diagnosis and demonstrate a definitive need for the treatment. Patients voiced their concerns to IDF and throughout the PI community.

We worked quickly to engage leadership at HCSC and the individual health plans to articulate the challenges our patients were facing. HCSC was eager to learn about our concerns and offered us a single point of contact who could evaluate the new information and insights and effect evidence-based policy change throughout the organization where and when it was needed.

In turn, IDF offered HCSC access to top clinical experts on primary immunodeficiency diseases who relayed accurate, updated guidelines for definitive diagnosis and clarified how critical it is for patients to be diagnosed properly and have uninterrupted access to treatment. We also explained the nuances of Ig therapy and how different products are not interchangeable. Ig is a highly complex biologic derived from human plasma — patients can respond differently to the same product.

Working with our medical experts, we assisted HCSC and its affiliates in amending their policies so patients with PI would have access to essential Ig treatment without delay. The updated policies matched the highest standards of care for patients needing immunoglobulin replacement for immunodeficiency while still allowing HCSC to manage its costs. Once the new policies were in place, we stayed in touch with HCSC to relay feedback from our patients and iron out a few issues in the administration of the policies.

IDF also made certain to close the loop with our patient community and communicate with our constituencies, crediting HCSC for working with us to create a positive outcome.

HCSC was pleased with the outcome and stressed that they deliberately put a focus on collaboration and appreciated the direct connection to clinical experts and patient advocates we provided. This approach helped them ensure medical policies were transparent, provider friendly, and met the latest standards in evidence-based care.

Proactively Seeking Collaboration

While large payers have mechanisms in place to monitor their policies, rare disease communities can play an important role in helping insurers avoid unintended consequences for patients. Patient groups can sound a warning when patients are being negatively impacted because a policy is not working as it should.

Many health insurers have been willing to collaborate when unanticipated problems arise, but even better for insurers and patients is to have payers and rare disease groups work together proactively when policies are being created.

Obtaining an accurate diagnosis and providing the optimal route of administration, appropriate dose, and site of care for each patient requires a high level of disease-specific knowledge.

While most payers strive to offer policies based on evidence-based medicine, that evidence is constantly evolving. Rare disease groups such as IDF can make experts available to ensure that the latest information is taken into account when formulating or modifying policies. IDF was able to provide this resource in our collaborative discussions with payers in the form of access to top-tier medical experts in PI.

This collaboration netted positive outcomes for all parties, and can serve as a model of how rare disease groups can interact with payers.

Insurers and rare disease advocates can’t serve the needs of patients, particularly medically vulnerable patients, in isolation. By working together, however, they can have a powerful impact on the entire health care ecosystem — payers, providers, and patients. It is an approach we hope will be replicated again and again when new policies are created or existing policies are amended.