Gaps in Access to Patient Care for Patients with Rare Hematological Disorders
Authors: Lachance Renee , Stewart Douglas A. , Worthington Katie , Mistry Beena , Yunger Simon , Garnica Jose M , Knight Shelagh , Milliken Debbie 
Published: October 2019

Abstract
Abstract 4752

Background
In Canada, gaps exist in access to therapy for patients with rare hematological disorders. Although a growing body of evidence exists for these rare disorders, they remain off-label as they are not Health Canada approved and as a result, limited drug funding options are available through Canadian funding bodies. For patients suffering with Warm Autoimmune Hemolytic Anemia (AIHA), Cold Agglutinin Disease (CAD), Cryoglobulinemia, Graft versus Host Disease (GvHD), Idiopathic Thrombocytopenic Purpura (ITP), Thrombotic Thrombocytopenic Purpura (TTP) and Waldenstrom's, challenges exist for physicians to access appropriate treatment for these patients. With no formalized public or private reimbursement available for these off-label indications, patients must pay for costly treatments out-of-pocket, or be left untreated.
In response to this unmet medical need, Roche Canada initiated a patient assistance program to ease the burden for physicians and their patients seeking limited treatment options for these rare disorders. Roche Canada's Patient Assistance Program (RPAP), managed by a 3rd party, assists patients with timely access to treatment through reimbursement navigation, financial assistance and private infusion clinics. To address the gap in patient care, Roche Canada piloted a non-promoted Compassionate Use (CU) program to provide access to therapy for patients with rare hematological disorders enrolled in RPAP after all public and private funding options have been exhausted. At the discretion of a 3rd party adjudication panel, the CU program provides rituximab on a case-by-case basis to patients with the above stated rare hematological disorders, where substantial evidence exists to suggest its safety and efficacy (Garvey B, BJH, 2008). All participating physicians enter a contractual agreement acknowledging the off-label use of rituximab. Response outcome data was not collected or required for the program.

Objective
This observational study compares, across two consecutive years, physician enrollment of patients in RPAP and patients' subsequent access to therapy in an environment with and without a compassionate use program.

Methods
An analysis of anonymized data from RPAP and the CU program was conducted, comparing 1 year of RPAP without the CU program, May 2009-April 2010(Y1), to 1 year of RPAP with the CU program, May 2010-April 2011(Y2). Patients were assessed by enrollment, indication, reimbursement coverage type, reimbursement approval, and access to rituximab.

Results
Total enrollment into RPAP over the 2 year period was 218 patients. 65 (30%) patients were enrolled in Y1, while 153 (70%) patients were enrolled in Y2, indicating a 153% (88 patient) increase in enrollment. Of the 218 patients enrolled, 154 (71%) subsequently received rituximab therapy. In Y1, 36 of 65 enrolled patients received rituximab therapy (55%), while 118 of the 153 patients enrolled in Y2 received rituximab therapy (77%). This indicates a 228% (82 patient) increase in access to therapy. Table 1 illustrates the number of patients who received access to therapy after enrollment in RPAP, and the type of reimbursement they received. Table 2 illustrates the enrollment and access to therapy by year and disease area.

Conclusion
The pilot compassionate program has highlighted that an unmet medical need exists for patients with rare hematological disorders. There remains a need for additional regulatory and provincial programs to help address the gaps in care for these patients.

Disclosures:
Lachance: Hoffman-La Roche Limited: Employment. Off Label Use: Rituximab is a chimeric mouse/human monoclonal antibody that binds specifically to the transmembrane antigen CD20. Rituxan is indicated in Canada for Non-Hodgkin's Lymphoma (NHL), Chronic Lymphocytic Leukemia (CLL) and Rheumatoid Arthritis (RA). This abstract describes the use of rituximab in the following rare hematological disorders: Warm Autoimmune Hemolytic Anemia (AIHA), Cold Agglutinin Disease (CAD), Cryoglobulinemia, Graft versus Host Disease (GvHD), Idiopathic Thrombocytopenic Purpura (ITP), Thrombotic Thrombocytopenic Purpura (TTP) and Waldenstrom's. Stewart:Hoffmann-La Roche Limited: Honoraria. Worthington:Hoffmann-La Roche Limited: Employment. Mistry:Hoffmann-La Roche Limited: Employment. Yunger:Hoffmann-La Roche Limited: Employment. Garnica:Hoffmann-La Roche Limited: Employment. Knight:Hoffmann-La Roche Limited: Employment. Milliken:Hoffmann-La Roche Limited: Employment.