Drugs for Rare Disorders A CASE FOR NATIONAL PHARMACARE THAT LEAVES NO ONE BEHIND
Disclosures WIDOWER WHOSE SPOUSE DIED WITHOUT A DIAGNOSIS FATHER OF AN ADULT CHILD WITH A RARE DISORDER CO-FOUNDER & PRESIDENT, CANADIAN PKU AND ALLIED DISORDERS CO-FOUNDER & TRUSTEE, GLOBAL ASSOCIATION FOR PKU BOARD CHAIR, BEST MEDICINES COALITION (BMC) CO-CHAIR, DISABILITY TAX FAIRNESS ALLIANCE ADVISOR, SICKLE CELL DISEASE ASSOCIATION OF CANADA PAST CHAIR, CANADIAN ORGANIZATION FOR RARE DISORDERS (CORD) RECEIVED HONOURARIA TO ADVISE BIOMARIN PHARMACEUTICAL, INC. developer of drugs for PKU and other rare and ultra-rare disorders ADVISOR TO APTATEK BIOSCIENCES, INC. Developing Biosensing devices, starting with PKU SELF CARE
A Plea: Leave No One Behind Plan Pharmacare for Not-yet-met Patient Needs DEDICATED TO MARITA ANNETTE ADAMS, DOD JULY 4, 2014 DEATH CERTIFICATE: CAUSE OF DEATH: NYD (NOT YET DIAGNOSED) AUTOPSY: FRONTOTEMPORAL DEGENERATION (FTD) & ALS
3 Big Questions for National Pharmacare 1. How many people are covered? Universal or not 2. How many drugs are covered? 17,000 DINs, or 8,000, or 2,000 or 125 The fewer, the less equitable for patients with rare disorders 3. Who pays? eliminate the 22% spend on out-of-pocket costs for Canadians address those patients who go without medicines government as first payer or backup payer National Pharmacare won t work without a major investment of new federal tax dollars
Drugs for Rare Disorders (DRDs) A Canadian Reality +7,000 rare diseases: affect 1 in 12 - nearly 3 million Canadians as patients +90% of RDs have no treatments that change course of disease Except symptom management and palliative care 60% of DRDs approved in USA and EU are approved in Canada- the gap most approved up to six years later than in USA & EU the lag A survey of patients with rare disorders by CORD in 2015 revealed that 2 in 5 patients do not have private drug plans or those with private plans are not fully covered Nearly a third said their prescription drugs were not covered by their drug plan 1/3: costs and/or co-pays, deductibles & caps made their drugs inaccessible
CORD s RARE DISEASE STRATEGY (2015) 1. Improving early detection and prevention 2. Providing timely, equitable and evidence-informed care 3. Enhancing community support 4. Providing sustainable access to promising therapies 5. Promoting innovative research
Goal Sustainable access to promising therapies: 6 recommendations by CORD Implement a regulatory framework for DRDs Explore adaptive designs of clinical trials for market authorization and post-approval phases Enhance role of patients in market authorization process and post-market evidence-generation provide resources to support the participation of rare disease patient groups in this process Establish a separate, more flexible health technology assessment process tailored to the specific attributes of drugs for rare disorder patients Increase support to assist rare disease patient groups to engage in health technology assessment, including in preparing patient input submissions Develop a consistent funding approach to ensure timely and equitable patient access to DRDs
Conventional HTA: Appropriate or Not for DRDs? DRDs often have higher costs for individual patients as R&D investments have to be recouped from a small market worldwide impossible, in most circumstances, for DRDs to meet conventional cost-effectiveness thresholds The clinical and economic evidence is more limited for DRDs given the rarity of the disease and the small patient populations Rareness of disease requires acceptance of more uncertainty Ethical and necessary to accept a higher cost per Quality Adjusted Life Year (QALY) and Incremental Cost Effectiveness Ratio (ICER) for DRDs
Approaches to Expanding Universal Coverage: What Might Work for DRDs? 1. Age Incrementalism 2. Common Drugs/Essential Medications List 3. Improving Catastrophic Drug insurance 4. Disease/Therapeutic Category Incrementalism 5. Big Bang
Expanding Pharmacare by Age Most provinces & territories have drug programs that provide coverage to residents over the age of 65, with each province having its own formulary. Ontario, as its opening initiative in expanding pharmacare, has started with age incrementalism and is now committing to cover full prescriptions costs for those younger than 25 years of age, and in March 2018 Budget proposing to eliminate co-pays for seniors in 2019. Unless DRDs have defined regulatory pathway and an HTA process and reimbursement negotiation processes appropriate for DRDs. expanding access by age to currently funded formularies does nothing for patients with rare diseases. If national pharmcare, looks like current F/P/T drug programs, we do not want it!
Expanding Pharmacare via an Essential Medicines List (EML) An Essential Medicines List, as proposed by some, is a basic formulary for primary care An EML, as discussed by the Canadian Labour Congress, the ON NDP and the former federal Health Minister Jane Philpott, is targeted to provide common (mostly generic) drugs for chronic conditions only needing primary care - provided to patients free regardless of age or income The main objective would be to buy as much health with as little money as possible and simplify prescribing for general practitioners There is nothing particularly equitable about this approach as it addresses a small set of diseases for which there are many treatment options An Essential Medicines List that does not include Drugs for Rare Disorders, is NOT an Essential Medicines List patients with rare disorders are left behind Essentialism is in the eye of the beholder
Expanding Pharmacare by Improving Catastrophic Drug Coverage There is no standardized nationwide catastrophic drug insurance for Canadians The 2002 Romanow Commission recommended a national program that would see reimbursement for catastrophic prescription drug costs that surpassed certain thresholds of household income As many rare disorders require a lifetime of treatment, subjecting patients to a lifetime of outof-pocket expenditures before catastrophic support kicks in, could further marginalize those with rare disorders A national program that improves catastrophic coverage of drugs to be helpful for patients with rare disorders -- would require an extensive and frequently updated list of DRDs
Expanding Pharmacare by Therapeutic Area Example: in Western Canada all cancer patients, regardless of age or income, have the costs of outpatient drugs covered by governments Example: F/P/T Inherited Metabolic Diseases (IMD) programs which cover the full cost of certain therapeutic products for specific IMDs such as PKU a small minority of patients are subject to dollar caps by a few provinces This approach offers the most potential for patients with rare disorders How will this work: Managed Access Programs? Separate Formulary for DRDs? Pilot projects? That is what we are here to talk about today
BMC: What is Needed to Meet Patient Needs UNIVERSALITY: Prescription medications have become an essential part of healthcare, with their potential to improve patient outcomes and save lives while bringing economic value to the health care system. All Canadians have a right to access medically necessary medications. NATIONAL SCOPE : Equitable access to medicines must be national in scope, regardless of whether there is central administration or the current structure of federal/provincial/territorial health care delivery. BROAD INCLUSIVITY: Pharmacare must be comprehensive and inclusive of all chronic, episodic, or acute conditions, regardless of incidence. THERAPEUTIC OPTIONS: Canadians have a right to a secure supply of a full array of approved medications, including both long-standing compounds and more recently approved advancements. The right medication at the right time" must be a central premise, enabling patients to benefit from those medications most appropriate for each. Pharmacare must offer a range of medicines, which provide therapeutic options, so that treatment is not limited to a narrow selection of drugs within a particular class, since individual even small variations affect patient response and outcomes. TIMELY ACCESS: Extended wait times to receive medically necessary pharmaceutical treatments are unacceptable. Access delayed is access denied and so leaders must ensure that efficiency and timeliness is embedded within any pharmacare framework including the review, assessment and reimbursement processes. COLLABORATION: Canadians need significant pharmaceutical policy reform that involves full consultation with all stakeholders and careful consideration of options and implications. While this process is underway, decision-makers and drug program leaders have a responsibility to continue to use current policy, program, and budgetary options to address specific pharmacare challenges, which directly affect the lives of patients.