Innovative patient support programs and data-driven
solutions are improving access to life-saving rare disease therapies in Canada.
Patients with rare diseases face significant barriers in accessing innovative therapies. The high cost of these drugs presents challenges to public and private drug programs, as well as the provincial and territorial health care systems. Timely access to these therapies is necessary to potentially save lives and vastly improve the quality of life for patients and their caregivers.
Pharmaceutical companies face significant challenges in developing and commercializing treatments for rare diseases. Of the 7,000 known rare diseases, only 5 per cent have a known effective treatment. By definition, rare diseases affect a smaller patient population, which makes research and development of effective treatments difficult. Among other challenges, randomized, controlled clinical trials are often infeasible due to small sample sizes, and there are fewer specialist clinicians who have the necessary expertise to conduct them. These limitations make evaluations of these treatments for health technology agencies difficult, as there is likely to be limited clinical data available and more uncertainty in the economic valuation, particularly due to the high cost of drugs for rare diseases.
Supporting patients with rare diseases
Many Canadians diagnosed with rare diseases rely on treatments that are not approved by Health Canada through Health Canada’s Special Access Program (SAP). The SAP has become an important pathway for patients to access new therapies. Bayshore Specialty Rx works with many manufacturers and specialist clinicians to leverage the national network of pharmacy and nursing support services to support SAP patients with rare diseases.
Manufacturer-supported patient support programs (PSPs) like those provided through Bayshore Specialty Rx represent another critical pathway for patients with rare diseases to obtain compassionate care for innovative medications that are approved by Health Canada but not yet funded through private or public drug programs. Due to the limitations of clinical data, health technology agencies are increasingly accepting value-based and outcome-based submissions to improve timely access. PSPs supported by Bayshore offer an interesting opportunity for manufacturers to gather data to support outcomes-based submissions, particularly for drugs for rare diseases due to their high touchpoints with patients and caregivers, along with medical staff, throughout the complex patient journey. These touchpoints often include information on adherence, safety, reimbursement, and utilization. In more robust PSP offerings, nationwide linkage of Bayshore Pharmacy and Bayshore PSP databases can be used to support decision-making.
Utilizing real-world data and advanced analytics
Bayshore implementation teams collaborate with manufacturers to help identify evidence gaps and design PSPs that capture high-quality data for future submissions to private and public drug plans. The collection of outcome measures to support reimbursement negotiations can be targeted to address private or public drug plans at the launch of a PSP. For example, private drug plans have been interested in quality of life, particularly presenteeism and absenteeism. Bayshore has been able to implement omnichannel solutions to collect quality of life surveys at defined treatment touchpoints to help support manufacturer in payer negotiations. Adopting advanced technology within PSPs is critical to support the collection of enrolment, baseline, and outcome measures (for example, with MyBayshoreCares, Gateway, Alida, and other technology). The collection of data in a real-world setting, coupled with advanced analytics and linkage with external economic utilization measures, can be powerful tools to inform commercial strategy for manufacturers and support reimbursement decisions.
To learn more, visit bayshorespecialtyrx.ca
