Designing Strategies for Neglected Disease Research: What Innovations Do We Need? Julia Walsh MD MSc UCB School of Public Health jwalsh@berkeley.edu What Innovations Do We Need? Health Interventions Products & Services that promote health How do we promote R&D for New Interventions for the Major Diseases of Poor Countries? Types of Interventions 1 Health care dependent • Drugs • Diagnostics • Vaccines • Others (e.g. male circumcision, behavior change) Types of Interventions 2 Health care Independent • Environment • • • • Water, Sanitation Air pollution - Indoor & Outdoor Safety Vector Control • Behavior Change • Others? Types of Health Interventions 3 Mechanisms for Health Care Independent Interventions – Budget allocation – Tax incentives/Disincentives – Subsidies – Regulations/Laws – Information, Education, Communication – Research Myriad Possible Interventions: How set Priorities • Goal of the health system - maximize health of the population • Limits - scarce resources • Decisions and choices necessary • Best choice - maximize health with fewest resources • Therefore - must compare costs of strategies with outcomes Cost-Effectiveness Analysis Examples of Cost-Effectiveness Analysis • • • • Population 1Million people $10Million available for health investment Two Research Programs A & B both cost $10M Program A will potentially avert 10,000 deaths or $1,000/death averted ($10M/1000deaths) • Program B 25,000 deaths or $400/death averted ($10M/25,000) • Which is More Cost-effective? The Amount of Health $1Million Will Buy - Reducing Under-Five Mortality Service or Intervention Cost/ DALY DALYs per $1M spent Neonatal care Age 10-400 2.5– 100,000 2-20 40-250 50 – 500,000 4 – 24,000 8-20 50 – 100,000 <28days Vaccines Expand coverage Add new vaccines H.flu, Hep B, rotavirus Improved Rx Malaria SubSaharan Africa The Amount of Health $1Million Will Buy – HIV Prevention & Treatment Service or Intervention Cost/ DALY DALYs per $1M spent Prevention Mother to Child Transmission 50-200 5 – 20,000 STI Treatment * 10-100 10 – 100,000 ARV Treatment 10-500 2 – 100,000 Source: DCP2.org Male Circumcision Source: Fieno J; 2008 AIDS Care 0-7.50 10-40,000 The Amount of Health $1Million Will Buy – Noncommunicable Disease Service or Intervention Cost/ DALY Tobacco Tax 3-50 DALYs per US$1M spent Generic Rx Acute Myocardial Infarction + Streptokinase Bypass Surgery 40-100,000 10-25 20-330,000 +600-750 +1,300-1,600 Cervical CA Dx & Rx >25,000 <400 15-50 20-60,000 Basic Surgical Ward 70-250 4-15,000 What makes an intervention costeffective? • • • • Averts large number of deaths +/or DALYs Highly Efficacious Very Inexpensive Health care - Easy to use & distribute – – – – – – One or small # doses Heat stable No electricity, technology, maintenance, pure water Use by unskilled health workers Lightweight, easily carried to homes or rural clinics Feasible in poor countries (?regulations against use of trans fats?) • More cost-effective than the next best intervention for that condition From Yach et al., The Global Burden of Chronic Diseases, 291 JAMA 2616 (2004). For what causes of death are better interventions needed in LDC: • • • • • • • • Respiratory Infections HIV Perinatal Conditions Diarrheal Diseases TB Childhood Diseases ???? Malaria Maternal Conditions Health Care Interventions Infectious Diseases & childhood illness control usually most cost-effective Biotechnologies – – – – Diagnostic tests Vaccines Treatments Others – e.g., preventive measures • Microbicides • Female condoms Priorities for Development of New Diagnostics Gates Process: Health impact Market potential & demand Willingness to Pay – Clients, donors, governments Gates Priorities for New Diagnostics Health Impact - No DALYs! • Tuberculosis • Sexually transmitted Infections in women – In Comercial Sex Workers: Gonorrhea & Chlamydia – In antenatal care: Syphilis • Malaria in Children • Acute Lower Repiratory Tract Infections in Children – Bacterial etiology & needing antibiotics – Severe disease requiring hospitalization • HIV in infants • Stunting in children: Enteroaggregative E.coli, Giardia lamblia, Cryptosporidium parvum Source: Nature 2006; Determining the Global Health Impact of Improved Diagnostic Technologies for the Developing World Vaccines for the 21st Century: A Tool for DecisionMaking Institute of Medicine 2000 US Based Method: Cost-effectiveness assessment of health benefits vs. costs of care Assumption of availability within 20 years Results IOM Vaccines for 21st Century Level I Most Favorable –Saves $ & QALYs • Cytomegalovirus administered to 12 year olds • Influenza virus vaccine for the general population (once per person every 5 year or 20% of the population per year) • • • • • Insulin-dependent diabetes mellitus therapeutic vaccine Multiples sclerosis therapeutic vaccine Rheumatoid arthritis therapeutic vaccine Group B Strep vaccine for pregnant women and high risk Streptococcus pneumonia vaccine for infants and elderly Health Impact of Tropical Diseases • • • • • • • • • • trypanosomiasis, Chagas disease, schistosomiasis, leishmaniasis, lymphatic filariasis, onchocerciasis, intestinal nematode infections, Japanese encephalitis, dengue, and leprosy TOTAL 177,000 deaths worldwide in 2002, mostly in sub-Saharan Africa, 20 million DALYs, or 1.3% of the global burden of disease and injuries. Conclusion Many new health innovations will save lives and DALYs/QALYs Work on the big problems