AMSUS THE SOCIETY OF FEDERAL HEALTH PROFESSIONALS Cracking the Code On Gene Therapy Steve Miller, MD Chief Medical Officer Nov. 27, 2018
Disclosures Presenter(s) has the following interest to disclose: Full-time employee of Express Scripts Board of Directors, Medibeacon Chairman of the Board, SureScripts ACE/PESG and AMSUS staff have no interest to disclose. This continuing education activity is managed and accredited by AffinityCE/Professional Education Services Group in cooperation with AMSUS. ACE/PESG, AMSUS, and all accrediting organizations do not support or endorse any product or service mentioned in this activity. 2
Learning objectives 1 Participants will understand the developing gene therapy market; which diseases, market size and implications for providers and plan sponsors. 2 Participant will be introduced to innovative approaches to fund treatments for small populations with very expensive, often one-time therapies. 3 Participant will understand the actions they need to take today to be prepared for the future. 3
Our costliest conflict 4
Which was more deadly? 5
A magic bullet 6
Drug spend and specialty share are growing Specialty Traditional 37% 2016 52% 2019 63% 48% Note: Data is actual for 2016, projected for 2019 Source: Express Scripts research 7
40 35 Novel FDA approvals continue to increase 30 25 20 15 10 5 0 Traditional Specialty Source: U.S. Food & Drug Administration 8
New scientific approaches will lead to more novel, but costly medications Conjugated Monoclonal Antibody Pipeline Products Using Gene-based Mechanisms 188 DNA & RNA Therapeutics 173 Cell Therapy (CAR-T) 529 Gene Therapy 202 Sources: Analysis Group, PhRMA 9
Rare disease therapies are much more costly Growth of rare disease FDA approvals (%) Average cost to treat a patient annually Source: Express Scripts data 54% increase in the cost of treatment over 4 years 10
RARE CONDITION PREVALENCE More common than you think 7,000 known rare conditions 1 with more identified all the time 1. Global Genes: Rare Disease Impact Report 2. American Diabetes Association 11
Gene augmentation therapy Functioning gene Cell with non-functioning gene Cell functioning normally 12
Gene inhibition therapy Blocking gene X X Cell containing faulty gene New gene product blocks faulty gene Cell functioning normally 13
Transfection: The process of inserting genes 14
Gene therapy poses unique challenges 4,000 diseases linked to gene disorders High cost: $375k-$1.5M per patient Single administration Very small patient populations Durability periods vary American healthcare system is ill equipped for this model 15
Fewer patients lead to higher prices $1,600,000 Estimated cost of therapy $1,400,000 $1,200,000 $1,000,000 $800,000 $600,000 $400,000 $200,000 $0 Glybera <10 patients Luxturna 600 patients Strimvelis <20 patients Kymriah 300 patients Yescarta 7,500 patients
Critical questions need to be addressed Clinical Utilization/Eligibility Who will manage? Centers of Excellence Where will patients go for treatment? Follow up How will patients be monitored? Risk management Who will collect and manage outcomes? 17
First U.S. CAR-T therapies approved for cancers Cost: $475,000 Treats lymphoblastic leukemia Lethal blood and bone-marrow cancer Affects children and young adults Cost: $373,000 Treats large B-cell lymphoma Aggressive non-hodgkin lymphoma Indicated after other treatments fail 18
Luxturna TM : Therapy with novel payer programs brings hope for inherited retinal dystrophies Outcomes-based rebates Innovative contracting model Payments over time 19
Specialized patient care enables risk sharing and improves health outcomes ONE-ON-ONE PATIENT SUPPORT IN-HOME NURSING SERVICES SAFE, APPROPRIATE MEDICATION USE 20
New payment models under consideration Pooled Risk Lump Sum Pooled Risk With Performance Guarantees Periodic Payments The right solution will enable collaboration among manufacturers, payers, patients and policymakers
Learning objectives 1 Participants will understand the developing gene therapy market; which diseases, market size and implications for providers and plan sponsors. 2 Participant will be introduced to innovative approaches to fund treatments for small populations with very expensive, often one-time therapies. 3 Participant will understand the actions they need to take today to be prepared for the future.
CE/CME Credit If you would like to receive continuing education credit for this activity, please visit: http://amsus.cds.pesgce.com Hurry, CE Certificates will only be available for 30 DAYS after this event! 23