Thought leaders gather to examine challenges facing patients with primary immunodeficiencies

Thought leaders from the medical, scientific and patient advocacy communities gathered in New York and London for CSL Behring's Key Issues Dialogue–Immunoglobulin to examine challenges facing patients with primary immunodeficiencies. They found common ground between the US and Europe on access-to-care issues such as early diagnosis and treatment of primary immunodeficiency disease (PID) and explored possible ways of improving patient care.

The Think Zebra! campaign from the Immune Deficiency Foundation (IDF) put the challenge of early diagnosis in perspective. "In medical school, physicians are taught that when you hear hoof beats, think horses," Marcia Boyle, Dialogue participant and president and co-founder of IDF said.  "In other words, think of the most usual cause of the most usual diagnosis. Immunologists are taught when they hear hoof beats to think zebras. Look for the unusual."

The participants examined barriers to accessing immunoglobulin (Ig) therapy, which is available through subcutaneous and intravenous dosing, including challenges by some insurance plans that impede patient care. "Specialists cannot see a patient initially and then have the remaining care provided by a doctor closer to the patient's home and still be covered by insurance," said Dr. Richard A. Lewis, Professor and Associate Chairman of Neurology, Wayne State University School of Medicine.

Medicare and insurance companies sometimes determine the location of care based on criteria that do not consider the patient's best interests and that are not necessarily the most cost-effective option, according to Dr. John W. Sleasman, Robert A. Good Professor and Chief-Division of Allergy, Immunology and Rheumatology, Pediatrics Department, University of South Florida.  "The patient is required by Medicare to travel two hours by ambulance from his home to a hospital where he is administered intravenous Ig," Dr. Sleasman said about a patient in his seventies with common variable immunodeficiency. "It would be much more convenient and less costly to administer it in his home."

The problem has been further compounded by cuts to healthcare benefits. As a result, patients are experiencing longer intervals between Ig treatments and in some cases never get back to that point where they left off when they were getting infusions on a regular basis. "They [patients] go downhill, and they then end up with health changes that require treatment they didn't need before," Dr. Sleasman said.

Another challenge involves rigid guidelines for managing patients, particularly with respect to dosage. "You need guidelines but when they're too specific, insurance companies can really misuse them," Boyle said. Dr. Lewis added that in Europe, discussions about dosage restrictions are linked to the development of health technology assessments (HTA) which factor in dosage, efficacy and cost. "We have to be careful in the area of HTAs and provide data in a timely and effective way," Dr. Lewis said.

The participants agreed that Ig patients often have dramatically different needs in terms of dosing, formulation and methods of administration as they grow from infancy to adulthood. It is important for the makers of biotherapies used to treat PID to help manage patients' needs throughout their lifecycles, just as they manage the lifecycle of the products in their portfolio. Areas of common ground between the US and Europe were identified including a universal emphasis on sharing information to ensure that patients receive optimal care. Boyle said an IDF Internet survey found that patients who are connected to a patient organization receive better care than those that are not.