When novelty is not enough: Assuring adoption, access, as well as approval within the clinical development process Michael F . Murphy M.D. Ph.D. Chief Medical and Scientific Officer, Worldwide Clinical Trials Research and Development Editor, American Health and Drug Benefits™ Teaching Faculty, Center for Experimental Pharmacology and Therapeutics Harvard-MIT Division of Health Sciences and Technology
Disclosures Michael F. Murphy M.D. Ph.D. Chief Medical and Scientific Officer, Worldwide Clinical Trials Research and Development Editor, American Health and Drug Benefits™ Teaching Faculty, Center for Experimental Pharmacology and Therapeutics Harvard-MIT Division of Health Sciences and Technology Acadia Coronis GB Sciences Neumentum RMJH Rx AgeneBio Cortexyme Gideon Richter Neuralstem Roche Amygdala Cristcot Global Blood Therapeutics NeuroRx R-Pharm Aptinyx Curadim Immunova Neurotrope Rubius ARC Cytogel Incys Novus Sanofi Genzyme Argenx Diurnal Isothrive Orchard Sinopia ArQule Dynacure Medivir PanX Spruce AudioCure Eisai Momenta Philip Morris Int. Suven Aurin EMS Navinta Praxis X4 Frequency BioXcel NeoTrope Rhythm Therapeutics
“I have time to ask three questions– how’s your gallbladder, how’s your teeth, and do you have good outpatient pharmacy coverage” Interventional Cardiologist Birmingham, AL Prior to PCI
We Will Discuss • The “other” stakeholders • Starting at the End • Looking beyond the obvious • Competing on value, not novelty
The “Other Stakeholders” Academia Children Insured Assisted Living Care Providers Women Consumers Uninsured Nursing Homes Financial Markets Seniors Intermediaries Physicians Labs Patients Bio Tech Employers Nurses Global Regulation Regional Variation Med Tech CMS, State Medicaid Governments Unions Products & Medicare -Medicaid Services Disease Management Basic Researchers States Insurers Pharma Neither orderly nor rational, multiple Devices stakeholders impact R&D with conflicting data needs
Start with the End in Mind “ CMS coverage policies for Biologics : defining a Lagrangian point for cost, quality, and access ” “ Capnography in procedural anesthesia: at the edge of a “perfect storm ” “ Relieving nasal sym ptom s : uncommon excellence in a common clinical condition” Risk stratification “Case Study: Insu-Nectin (NV 1008) for Type 2 Personalized Medicine Diabetes” Orphan Disease Indications “Pharmaceutical R&D Strategy and the Transition to Personalized Healthcare Planning ” “ Pharm acogenom ics and Drug Development: A Raises questions about validity of drug Stakeholder Perspective” formularies and reimbursement “ Post-Approval Developm ent Options in COPD A Case Study mechanisms to control access to in Value-Based Healthcare Systems” therapeutics “ The Metabolic Syndrom e and Atypical Antipsychotic Medications: Risk Factors, Surveillance Methods, and Healthcare Implications” Can this inform a development program? “ Medical Claim s Data Can Inform Coverage Decisions in Managed Care Health Plans” - Angina in Patients with Coronary Artery Disease in a US Managed Care Setting David B. Nash M.D., MBA Michael F. Murphy M.D., Ph.D. Editor-In Chief, Dean Jefferson School “ Rarity, Disease Heterogeneity, and A Pathw ay Research & Development Editor of Population Health for Estim ating Econom ic Burden
Essenti tial C Component t of O f Overall I Innovati tion Value Assuring adoption, payer coverage, and premium pricing Improved Impact How Result Features & Benefits Quality Market Attractiveness Improved patient ? Clinical Impact outcomes Fit Within Evidence-based Company Innovation Value Strategic Value Initiatives Reduces Economic ? downstream Impact Cost of Goods Intellectual costs Advantage Property Position Ability to Leverage Core Competencies Data during development creates a “value” proposition to inform technology investment decisions Used with permission of Boston Healthcare Associates, Inc. Confidential - Used with Permission, Boston Healthcare Associates
What if the Indication was “Fatigue”?
Different Prisms, Different Data • Actuary “W hat don’t they w ant?” • Employers • Epidemiology Research “Current stum bling blocks?” • Health Information Technology • Health Outcomes Research • Managed Care & Government Affairs “W hat they are w illing to • Managed Markets consider?” • Patient Advocacy • Pharmacy Benefit Design • Policy & Public Health “W hat’s prom ising given current perspectives?” • Reimbursement Policy • Research & Development • Specialty Pharmacy “How diverse are data needs?”
Payers, Purchasers, Policymakers • W hat they don’t w ant? • Paying for treatment requiring evidence that doesn’t exist • W hat do they regard as stum bling blocks? • “Big enough to care about, but small enough to do something about?” • “Will it impact overall healthcare resource utilization?” • “Any guidelines?” • “Is this a lifestyle-modifiable condition?” • “Will this prompt disease-mongering? – it’s been done before!” • W hat are they w illing to consider? • Controlled and observational studies • Outcomes (direct, indirect, HRQOL, productivity) • Universally--- “bring me in prior to approval!”
Multiple Sclerosis – “Limiting Access if Value Uncertain” Diverse Policies Specialist Rx Only Prior Authorization (+ Re-authorization) Tier Placement (~ 60% ) Require affirming benefit before and after therapy (~ 40% )
ADHD – “The Better the Data the Less the Management” Strattera will face low reimbursement from HMOs. Parma Business Week 2003 JUN 9 - (NewsRx.com & NewsRx.net) -- Decision Resources finds that Eli Lilly's Strattera will face low or nonexistent reimbursement from HMO pharmacy directors… High Interest Fatigue impact on cost drivers uncertain (Reimer et al, 2007; Wu et al, 2007; “Out of pocket expenses range Leibson et al, 2001) from $40-$230/ month across a range of possible medications…and “Interaction effects” (Page et al,2016) can substitute a generic without physician permission” Many Tools Control Utilization (Fail-First PA Pharmacist strategy, Preferred List, Co-insurance, Co-pay)
Alzheimer’s Disease – “Looking Beyond the Patient” Opportunities Mild cognitive impairment (Zhu et al, 2013) Informal care (Jönsson et al, 2006; Deb et al, 2017) Depression, resilience, distress in caregivers (Lavretsky et al, 2010; Abreu et al, 2018)
Schizophrenia – “Episodes of Care” “Off the radar” for Some Stakeholders, Many Questions by Others Fatigue entwined with Cognitive Impairment, Reduced Physical Activity? Contribution to metabolic syndrome (CV outcomes)? Poor compliance (institutionalization)? Episodes of care (utilization)?
Depression – “Interest, but not where expected” High Interest Commercial Plans Non-Psychiatric Utilization (minor somatic complaints including fatigue; Katon et al, 2001; Greenberg et al, 2015) Employers Absenteeism, Presenteeism (Druss et al, 2000; Cocker et al, 2014; Evans-Lacko and Knapp, 2016)
A Portfolio of Initiatives
The Rise of RWE
Accessible Cost Drivers in Development • If it’s after filing, it’s too late No (minimal) Additional Expenditures • Breakthrough? • Using Micro-environments (IDN) • “Novelty makes me nervous” • The "big five in the EU“ • Patients in deep end of the swimming pool? • Within study claims analysis • Piggybacking • Practice topology as a prognostic factor? • Compendia • Outcomes, not just measures? • Complementarity? Additional Expense • Budget impact vs cost effectiveness? • Piggy-back or Observational? • Longitudinal cohort, screen failure patients • Research Center (direct-to-patient call Center) • A publication package (adoption; access)
In answer to the question Systems/ Provider Perspective: What are we Currently Doing and How is it Working? “Requires an alternative definition of translational research, demonstrating the value of an intervention in the context of it’s novelty.”
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