Medical School for Actuaries June 12, 2013 Baltimore, Maryland Developments in the Treatment of Conditions Treated with Specialty Mediations (Cancer, MS, RA, Hemophilia) Mark S. Matusik, PharmD
Developments in the Treatment of Conditions Treated with Specialty Medications Mark S. Matusik, PharmD Director, Clinical Consulting Walgreen Co. Agenda Specialty Pharmacy Definition and Trends Developments in: Multiple Sclerosis Rheumatoid Arthritis Oncology Hemophilia Hepatitis C 2 1
Specialty Pharmacy Definition (Academy of Managed Care Pharmacy AMCP) 1. Specialty pharmaceuticals are typically large, unstable, protein-based molecules, produced through a biotechnology process and 2. Require a difficult or unusual process of delivery to the patient or 3. Require patient management prior to or following administration. High cost is not enough to define a product as a specialty medication! 3 Specialty Drug Spending to Jump 67% by 2015 4 Source: Express Scripts: Prescription Drug Trends Report 2013 2
5 Source: Express Scripts: Prescription Drug Trends Report 2013 Multiple Sclerosis 3
Multiple Sclerosis Established medications ABCs of MS Avonex, Betaseron, Copaxoneone Rebif, Tysabri Gilenya (fingolimod) and Aubagio (teriflunomide) 1 st oral medications Plegridy (peginterferon beta-1a) Newer version of Avonex SQ vs. IM Pegylated allows for greater drug exposure 7 Multiple Sclerosis Tecfidera (dimethyl fumarate) Oral BID dosing FDA Approved March 27, 2013 Dramatic uptake by prescribers EU launch delayed until later in 2013 Lemtrada (alemtuzumab) Infused medication 5 consecutive days, then 3 days one year later Previously marketed as Campath to treat CLL Voluntarily withdrawn from US and EU markets to optimize drug for use in MS. 8 4
Multiple Sclerosis 9 Rheumatoid Arthritis 5
Rheumatoid Arthritis $34,164 = Avg cost of care annually $18,098 = Specialty medications 70+% through pharmacy benefit Cost for current therapies ~$1,500/month 11 Source: Prime Therapeutics 2013 (data from 2010) Rheumatoid Arthritis Established Medications Enbrel, Humira, Kineret, Orencia, Cimzia, ia Simponi (golimumab) New indication ulcerative colitis (May, 2013) Actemra (tocilizumab) - New SQ form vs. IV Xeljanz (tofacitinib) New class Janus Kinase (JAK) inhibitor Also being studied for Chronic Bowel Disease 12 6
Hemophilia Hemophilia A Existing therapies Clotting Factor VIII Annual costs $60,000 - $100,000 Eloctate (rviii F c fusion protein) New class of clotting agents Covalent bond = more stable Longer dosing interval = less frequent injections Reduce injections by 50-100 per year rviii-singlechain Phase III trial begun Mfr = CSL Behring 14 7
Hemophilia B N9-GP (glycopegylated recombinant Factor IX) Phase III trial completed Prophylactic tx reduced risk of bleed by >90% 99% of occurring bleeds stopped with single dose Regulatory submission in 2015 15 Oncology 8
Multiple Myeloma 21,700 patients diagnosed with MM 10,710 deaths from MM Symptoms = Fatique, bone pain, compression fractures End organ damage = hypercalcemia, anemia, renal dysfunction, bone lesions Median survival prior to 1960s = < 1 year Source: American Cancer Society 2012 17 Multiple Myeloma Revlimid (lenalidomide) Oral medication Analogue of thalidomide Pomalyst (pomalidomide) FDA approved 02/13 Doubled survival rate for many patients More potent oral medication Velcade (bortezomib) IV or SQ administration Kyprolis (carfilzomib) IV administration 18 9
Multiple Myeloma Other medications in Phase III trials Elotuzumab (AbbVie) Daratumumab (Janssen Biotech) Panobinostat (Novartis) 19 Companion Diagnostics FDA Approves Two Advanced Melanoma Treatments, Genetic Test 20 Source: Wall Street Journal 5/15/13 10
Melanoma Metastatic Melanoma Two oral treatments Tafinlar (dabrafenib) and Mekinist (trametinib) both marketed by GlaxoSmithKline. Advance Melanoma Nivolumab and lambrolizumab, ormk-3475 shrunk tumors 30-52% in early trials 21 Companion Diagnostics Genentech unit of Roche developed test to detect t epidermal growth factor receptor gene mutation Those who test positive are more likely to respond to Tarceva (erlotinib), manufactured by Roche 22 11
Companion Diagnostics A risk-based framework is under development that will ensure the diagnostics used in cancer treatment will provide medical professionals with a critical baseline for confidence in the test they order 23 Source: Pharma Times 6/6/13 Hepatitis C 12
Hepatitis C 2.7 to 3.9 Million people affected in U.S. 12,000+ deaths annually More people die from viral hepatitis infection than from HIV infection Everyone born between 1945 and 1965 should be tested for Hepatitis C (CDC Aug 2012) 25 26 Source: Express Scripts: Prescription Drug Trends Report 2013 13
Hepatitis C Treatment based on Genotype Genotype 1 (75% of cases) Requires triple therapy Interferon + ribavirin + protease inhibitor Genotypes 2 and 3 (25% of cases) Interferon + ribavirin (no protease inhibitor) Goal of therapy: Maintain a sustained virologic response (SVR) i.e. no detectable virus in the blood 27 Hepatitis C Victrelis (boceprevir) Oral protease inhibitor 4 tablets 3 times daily with food Food enhances exposure by 65% Incivek (telaprevir) Oral protease inhibitor 2 tablets 3 times daily with food Food enhances exposure by 237% High fat meal (330%) Low fat meal (117%) 28 14
Hepatitis C Cost of triple therapy: Early responders = $43,000 to $57,000+ Late responders = $90,000+ Non responders (e.g. Cirrhosis) = $106,000+ AWP cost per tablet Victrelis = $14.99 (12/day) Incivek = $109.67 (6/day) 29 Hepatitis C Future treatments in Phase III trials Protease Inhibitors Simeprevir Faldaprevir Vaniprevir NS5A Inhibitor Daclatasvir NS5B Polymerase Inhibitor - Sofosbuvir FDA approval anticipated Fall 2013 30 15
What lies ahead Hepatitis C Multiple drug approvals Mixing and Matching of combinations that have not been investigated All oral regimens Elimination of interferon and associated side effects 31 Summary 16
Overall Pipeline Large number of drugs in development Cancer, ~320 drugs CNS disorders, ~140 drugs Infectious diseases, ~80 drugs Diabetes, ~80 drugs Respiratory disorders, ~75 drugs Rheumatoid arthritis, ~70 drugs Cardiovascular (CV) disease, ~65 drugs. 33 Summary Growing Focus on Specialty and Oral Drugs Current drug pipeline is booming with SpRxs 907 specialty medicines and vaccines targeting over 100 conditions Monoclonal antibodies (N = 338) Vaccines (N = 250) MS, Inflammatory Conditions (e.g. RA) and Oncology will drive the trends Oral medications will become more popular Control of treatment will move to consumer Controlling costs will remain a major focus for payer (e.g. MCOs, employers, government) 34 Source: Pharmaceutical Research and Manufacturers of America (PhRMA) 17
Nomenclature for SpRx mab = monoclonal antibody zu = humanized monoclonal antibody y( (trastuzumab) mo = murine (mouse) proteins (tositumomab) mu = fully human (not murine!) proteins (adalimumab) tu = cancer indications/tumor (gemtuzumab) li = inflammatory conditions (natalizumab, efalizumab) ci = cardiovascular related indication or mechanism (abiciximab) xi = chimera proteins (infliximab, rituximab) tinib = targeted therapy (erlotinib, lapatinib, sunitinib) 35 18