Expanded Access Program

A continual challenge encountered by drug developers investigating therapies for serious diseases or conditions is that of facilitating access for appropriate patients to these potentially life-saving or life-improving drugs. Expanded access programs, which are sometimes termed patient access, named patient, early access or compassionate-use programs, present an opportunity to help patients and their physicians in both the domestic and foreign markets in accessing investigational drug therapies. The Aequitas Group has extensive experience and deep set knowledge on the development of expanded access programs for pharmaceutical companies. We have studied the specific requirements and features of the expanded access programs that operate in the United States and around the world. The Aequitas Group has the capability to design, administer and audit a customized expanded access programs plan for your company. What is an Expanded Access Program? Patients with serious or immediately life-threatening diseases or conditions often seek access to investigational therapies after having tried approved therapies without success. Expanded access programs enable companies to respond to requests made by patients and their treating physicians for investigational drug therapies. These programs are administered by drug manufacturers and are governed by regulatory agencies. Expanded access programs operate in the United States, European Union, Canada, Australia and several other countries. How can your company provide appropriate patients with investigational therapies? Regulatory agencies around the globe recognize the importance of providing access to potentially life-saving or life-improving investigational drug therapies for patients who are unable to participate in clinical trials and who have exhausted other options. Pharmaceutical manufacturers can set up programs that enable them to respond to these requests quickly and efficiently. The regulatory rules surrounding these programs often allow manufacturers to charge for the drug therapies and the costs surrounding their distribution. How can The Aequitas Group help? The Aequitas Group has helped companies understand the distinct features of expanded access programs and to create an efficient process and infrastructure to set up, monitor and execute these programs successfully. We have three areas of expertise: Expanded Access Analysis - Insight into available programs (including whether participation will assist in pre-market conditioning for your product) Implementation Support - Development of a strategy to support a program including decisions on in-house verses outsourcing, creation of a program and RFP Execution - Running a program, training of staff, melding of outsource and internal resources The Aequitas Group has the capability to design, administer and audit a customized expanded access programs plan for your company. We believe that customized solutions facilitate access, which can lead to improved health for patients. Furthermore, these programs often generate an atmosphere of goodwill amongst physicians and patients that directly and indirectly benefits drug developers. Case Study: Expanded Access Audit Insight A global pharmaceutical company developed a Named Patient Program in oncology to address unmet patient needs. The program provided commercialized US products for patients in other countries where the products were not commercially available. The company wished to audit the Named Patient Program’s compliance with local legal and regulatory requirements, the standard operating procedures (SOPs), as well as corporate policy for Named Patient Programs to ensure that it was continuing to meet corporate goals. Strategy The Aequitas Group worked with the client’s medical group, legal division and trade relations departments to review the SOPs for the Named Patient Program and ensure it reflected current requirements. The Aequitas Group evaluated the data management system used to administer program activity and track required documentation. Specific areas for improvement were identified, as were elements that needed to be maintained. Operational metrics were established and key performance indicators (KPIs) developed for the program. A training program was put in place for communicating and educating staff on policy and procedure compliance. Execution After analyzing the current Named Patient Program workflows and scope of activity, The Aequitas Group identified gaps in knowledge, as well as areas for improvement of program processes. The Aequitas Group updated the program SOPs and process flow diagrams to effectively and accurately describe actions and decision points, and provided oversight in training staff on procedure compliance and assistance in implementation of new practices. The Aequitas Group remains the client’s third-party auditor for all US-based Named Patient Programs. If you would like to learn more about The Aequitas Group, please contact us today.