Customized Patient Care through Patient Support Programs for Rare/Orphan Drugs. Sandra Anderson August 2015



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Customized Patient Care through Patient Support Programs for Rare/Orphan Drugs Sandra Anderson August 2015

Why Orphan Drugs are Different Difficult to diagnose Population size Unique patient and caregiver needs Cost Reimbursement challenges Wait times for Specialist Large amount of stakeholders involved to get patient on therapy There is no clear path to get Orphan Drugs Funded 2

A Typical Journey of a Patient with a Common Disease Insert visual of patient with high blood pressure- ie asymptomatic disease Insert visual of a patient with asthmaa symptomatic disease Physician Office

The Patient Journey in Rare Disease Prescription Enrolment in PSP Demographics, clinical history PSP Introduction Coverage information is captured Services explained Onboarding SA submissions Explore Reimbursement coverage Journey to Dx Patient set up to receive treatment Coverage is secured Injection/Infusion Life time Services? Max Copay? Call review Coverage decision Rx $ Infusio/Injectionn Continued Care Calls Determine status Coach patient Maintenance Annual Renewals

Why are orphan Drugs Different? Different reimbursement routes across Canada. Public reimbursement/private reimbursement 50/50 Traditionally very high cost medication requires additional administrative work ie. Special authorization Increase in private payer restriction calls for strong private payer strategy Small patient populations may have regional bias

The Patient Perspective The high drug cost, and out-ofpocket component, (copayment or deductible) can be unmanageable for many patients (especially for Orphan drugs ~hundreds of 000s/year) Most income-based public plans such as Trillium in Ontario require patient contributions in the form of copayments/deductibles (~3-5% of household income) With complex therapies, the primary concerns for patients are access, convenience and the level of customer service Private plans vary: range from 100% coverage to 50% coverage, though the most commonly seen co-payments are set between 0 and 20%

Case Study: Patient Access Barriers Defined Condition and Therapy Patient Healthcare System Source: Berry SA, et al. Genet Med 2013:15(8):591 599 Social and Economic

Needs for Orphan Rare Disease PSPs High Touch Require specific administration or monitoring requirements before and ordering administration High Support Education, counselling, compliance monitoring, and drug information Special Distribution Special handling techniques; and / or require special handling, have special shipping requirements or need complex reconstitution Customized Patient Assistance Clinical, Diet and Lobbying/Financial Support to assist with access to drug (i.e. co-pay assistance)

Customized Patient Support Programs Nurse Case Manager One point of contact to deliver quality care at every touch-point Update on Patient Progress Innomar Nurse Case Manager Enrolment Enrolment: Entered in CRM, NCM assigned, patient contacted Reimbursement: Case Manager confirms coverage with the patient and sets up financial assistance Infusion Nurse completes pre-infusion checklist Patient Experience ***** Drug Dispensed, shipped to clinic) Specialty Pharmacy Reimbursement Infusion Scheduled Pre Infusion Checklist (Program) Infusion Scheduled: Clinic chosen, Fax to physician, Pre-infusion checklist, Reminder call, Drug order Specialty Pharmacy: Deferral of copay, Drug Order (Direct through Database), Warehouse sends drug to pharmacy, Drug delivered to Clinic Infusion: Nurse confirms Drug is onsite, Preinfusion assessment by nurse, Medical clarification needed?, Infusion, PIR Patient experience touch-point

PSP Case Manager, RN Case Manager / Reimbursement Specialist interacts with key stakeholders and customers - Highly experienced in Orphan/Rare products and motivated by offering best in class patient care Regular and customized updates on patient progress START: Enrolment - Experienced in coordinating patients therapy regimens - The Case Manager role is the one point of contact for the patient and their HCP Pre & Post clinical assessment follow-up Coordinates reimbursement & Co-pay - Experience working with Specialist s offices Schedules and coordinates patient infusions

Extensive Knowledge and experience in Rare Disorders Case Study 1 Challenge: Patient was hospitalized with multiple organ failure. The medication was still under review by the Province. Solution Public EAP requested Patient Advocacy: Lobby on behalf of patient; media releases; Canada wide petition; add pressure to decision makers; weekly telephone conferences with stakeholders Outcome Provincial compassionate program obtained to start and then successfully gained full approval from the province Patient is still on therapy!

Extensive Knowledge and experience in Rare Disorders Case Study 2 Challenge: Patient had a major stroke and hospitalized for over 2 years. Patient is in need of drug to prevent future thrombotic events, hospital refused to pay out of their budget. Solution Public/Private Requested that private payer cover medication (even though patient was hospitalized) Concierge (FCM) went to the physician s office to complete paperwork Provided private payer clinical evidence to support patient stability and improvement Outcome Gained approval through private payer for a period of one year after demonstrating clinical need

Conclusions > Rare diseases present unique challenges for: Diagnosis Testing Reimbursement Financial assistance Ongoing treatment Monitoring

Goal: Manufactures should leverage the PSP to demonstrate real world effectiveness value to gain access to payers Value to Payers Patient Support Program Demonstrate Real World Effectiveness and Value Health Outcomes Research Our ultimate objective is to build a build a different framework for Orphan Drugs which will shorten the timelines to access 14

Thank-you

Capitalize on PSP to generate real world data Purpose: To decrease reimbursement and commercial timelines pre-launch NOC post-launch SAP Patient Support Programs Objectives Allow patients to access commercial therapy Assist in the navigation of multidisciplinary services Coordinate patient logistic services (F/A, Pharmacy, Distribution, Nursing) Post Marketing Surveillance and Real World Data - Objectives To track outcomes in real world setting To illustrate and prove value of drug Clinical Research To prove clinical efficacy and safety (Phase I III) Reimbursement Objectives To gain coverage and funding with payers To gain coverage on behalf of patients 16