Public Private Partnership Issues in Communicable Diseases Dr Gill Samuels Executive Director Science Policy & Scientific Affairs Pfizer Global Research & Development Sandwich Laboratories UK Dr Gill Samuels Executive Director Science Policy & Scientific Affairs Pfizer Global Research & Development Sandwich Laboratories UK P P Agenda Global Healthcare Challenge Global Healthcare Challenge Features of Pharmaceutical Industry R&D Features of Pharmaceutical Industry R&D The Challenge of Infectious Disease The Challenge of Infectious Disease The Therapeutic Gap The Therapeutic Gap Bridging the Gap Bridging the Gap Features of PPPs Features of PPPs Metrics of Success Metrics of Success Recommendations/conclusions Recommendations/conclusions P P Agenda Global Healthcare Challenge Global Healthcare Challenge Features of Pharmaceutical Industry R&D Features of Pharmaceutical Industry R&D The Challenge of Infectious Disease The Challenge of Infectious Disease The Therapeutic Gap The Therapeutic Gap Bridging the Gap Bridging the Gap Features of PPPs Features of PPPs Metrics of Success Metrics of Success Recommendations/conclusions Recommendations/conclusions P P Changes in global causes of death 1990 to 2020 P P Agenda Global Healthcare Challenge Global Healthcare Challenge Features of Pharmaceutical Industry R&D Features of Pharmaceutical Industry R&D The Challenge of Infectious Disease The Challenge of Infectious Disease The Therapeutic Gap The Therapeutic Gap Bridging the Gap Bridging the Gap Features of PPPs Features of PPPs Metrics of Success Metrics of Success Recommendations/conclusions Recommendations/conclusions P P The Principal Contributors to the Biomedical Knowledge Base World-wide Biomedical Knowledge Base Government-Funded Basic Research For example: National Institutes of Health Centre National de la Recherch Scientifique (France) Academic Universities and Teaching Hospitals The Medical Research Council (UK) Privately Funded Basic Research For example: Academic Universities and Teaching Hospitals Wellcome Foundation Howard Hughes Medical Institute Weizmann Institute of Science (Israel) Research-Based Pharmaceutical Companies New Treatments for Disease Applied Research by Research-Based Pharmaceutical Companies P P Select Disease Target can we do it in the laboratory? does it have a high unmet medical need? return on investment? can we test in patients? P P Drug Discovery and Development in cerebro in silico in vitro in homo in vivo P P Administrative Support Analytical Chemistry Animal Health Anti-infective Disease Bacteriology Behavioral Sciences Biochemistry Biology Biometrics Cardiology Cardiovascular Science Clinical Research Communication Computer Science Cytogenetics Developmental Planning DNA Sequencing Diabetology Document Preparation Dosage Form Development Drug Absorption Drug Degradation Drug Delivery Electrical Engineering Electron Microscopy Electrophysiology Environmental Health & Safety Employee Resources Endocrinology Enzymology Facilities Maintenance Fermentation Finance Formulation Gastroenterology Graphic Design Histomorphology Intestinal Permeability Law Library Science Medical Services Mechanical Engineering Medicinal Chemistry Molecular Biology Molecular Genetics Molecular Models Natural Products Neurobiology Neurochemistry Neurology Neurophysiology Obesity Oncology Organic Chemistry Pathology Peptide Chemistry Pharmacokinetics Pharmacology Photochemistry Physical Chemistry Physiology Phytochemistry Planning Powder Flow Process Development Project Management Protein Chemistry Psychiatry Public Relations Pulmonary Physiology Radiochemistry Radiology Robotics Spectroscopy Statistics Sterile Manufacturing Tabletting Taxonomy Technical Information Toxicology Transdermal Drug Delivery Veterinary Science Virology X-ray Spectroscopy Pharmaceutical R&D - a Multi-Disciplinary Team Over 100 DifferentDisciplines Working Together P P Driven by Medical Need hypertension g.i. (acid disorders) arthritis (NSAIDs) bacterial infection sedation analgesia angina lipid lowering asthma anxiety depression osteoporosis prostate hypertrophy diabetes type 2 male pattern baldness acne influenza obesity dementia arthritis (disease modifying) artherosclerosis periph. vasc. disease oral peptide delivery AIDS vaccine urinary incontinence stroke/MI prophylaxis cancers allergies herpes hemophilia chlamydia infection emesis epilepsy migraine diabetes type 1 endometriosis thrombosis fungal infection heart failure chronic bronchitis schizophrenia Parkinsons psoriasis drug/alcohol abuse sexual dysfunction wound healing arrythmias cirrhosis hepatitis Gauchers irritable bowel syndrome Crohns disease ulcerative colitis unstable angina AIDS multiple sclerosis emphysema cystic fibrosis transplant rejection septic shock PatientPopulation HighMediumLow Low Medium High Medical Need Source: Lehman Brothers P P New Medicine Timeline Discovery & Development Process Years and $ Million Discovery Exploratory Development Full Development Phase I Phase II Phase III IdeaDrug years years 7- 9 years 2- 4 years P P Complex Disease Targets Too Long in Body Adverse Reactions Poor Absorption Low Levels in Body Not Effective Enough Not Sufficiently Selective Side Effects Unsafe Unstable Competition Impractical To Make Attrition Most Compounds Do Not Become Medicines Most Compounds Do Not Become Medicines P P Attrition in the R & D Process Idea Drug Years ~100 Discovery Approaches Phase I Phase II Phase III Discovery Exploratory Development Full Development 1,000 Screening Hits 12 Candidates 7,000,000 Compounds Screened 1 Product 6 Candidates P P Agenda Global Healthcare Challenge Global Healthcare Challenge Features of Pharmaceutical Industry R&D Features of Pharmaceutical Industry R&D The Challenge of Infectious Disease The Challenge of Infectious Disease The Therapeutic Gap The Therapeutic Gap Bridging the Gap Bridging the Gap Features of PPPs Features of PPPs Metrics of Success Metrics of Success Recommendations/conclusions Recommendations/conclusions P P Medicines Developed for Tropical Diseases DiseaseDrug African Trypanosomiasiseflornithine Chagas Diseasebenznidazole, nifutrimox Cysticercisisalbendazole, praziquantel Fascioliasistriclabendazole Giardia, moebiasissecnidazole, tinidazole Giardia, Helminths & Amoebiasisnitazoxanide Helminthiasisalbendazole, flubendazole, levamisole mebendazole, pyrantel pamoate Hydatidalbendazole Leishmaniasisamphotericin B Leprosyclofazimine, ofloxacin Lymphatic Filariasisalbendazole, ivermectin MalariaAmodiaquine, atovaquone/proguanil halofantrine, co-artemether mefloquine, pyrimethamine, sulfadoxine/ pyrimethamine Onchocerciasisivermectin Schistosomiasismetriphonate, niridazole, oxaminiquine praziquantel Strongyloidiasisivermectin TBvarious Trachomaazithromycin Source: FIIM-IPFMA, December 2000 Source: FIIM-IPFMA, December 2000 f P P Agenda Global Healthcare Challenge Global Healthcare Challenge Features of Pharmaceutical Industry R&D Features of Pharmaceutical Industry R&D The Challenge of Infectious Disease The Challenge of Infectious Disease The Therapeutic Gap The Therapeutic Gap Bridging the Gap Bridging the Gap Features of PPPs Features of PPPs Metrics of Success Metrics of Success Recommendations/conclusions Recommendations/conclusions P P Infectious Disease Burden in Developing Countries Every infectious disease is different -for some, products exist and are accessible -for many, products exist, but problems with access, affordability or acquired drug resistance -for some, R&D underway, but has yet to deliver new medicines -in other, limited R&D because insufficient science base -for some doubts on ROI to support ongoing R&D P P Agenda Global Healthcare Challenge Global Healthcare Challenge Features of Pharmaceutical Industry R&D Features of Pharmaceutical Industry R&D The Challenge of Infectious Disease The Challenge of Infectious Disease The Therapeutic Gap The Therapeutic Gap Bridging the Gap Bridging the Gap Features of PPPs Features of PPPs Metrics of Success Metrics of Success Recommendations/conclusions Recommendations/conclusions P P Defining Medical Need Mortality/Morbidity Mortality/Morbidity Disease trends Disease trends Cost to society Cost to society Percentage accessible to treatment Percentage accessible to treatment Availability of non-drug interventions Availability of non-drug interventions Limitations of existing products Limitations of existing products Alternative potential interventions Alternative potential interventions Scientific challenge Scientific challenge Extent of current industry engagement Extent of current industry engagement P P Agenda Global Healthcare Challenge Global Healthcare Challenge Features of Pharmaceutical Industry R&D Features of Pharmaceutical Industry R&D The Challenge of Infectious Disease The Challenge of Infectious Disease The Therapeutic Gap The Therapeutic Gap Bridging the Gap Bridging the Gap Features of PPPs Features of PPPs Metrics of Success Metrics of Success Recommendations/conclusions Recommendations/conclusions P P Types of PPPs Generate basic knowledge/research Generate basic knowledge/research Product discovery and development Product discovery and development Improvement of access to health products Improvement of access to health products Global co-ordination mechanisms, including funding vehicles Global co-ordination mechanisms, including funding vehicles Health Service strengthening Health Service strengthening Public education and advocacy Public education and advocacy Regulation, quality assurance and standards Regulation, quality assurance and standards P P Features of R&D - PPPS Use some private-sector approaches to attack research and development challenges Use some private-sector approaches to attack research and development challenges Target one or more neglected diseases Target one or more neglected diseases Use or intend to use variants of the portfolio management approach Use or intend to use variants of the portfolio management approach Primary objective is public health rather than a commercial goal Primary objective is public health rather than a commercial goal Are focused on developing products specifically suited for use in developing countries Are focused on developing products specifically suited for use in developing countries P P Agenda Global Healthcare Challenge Global Healthcare Challenge Features of Pharmaceutical Industry R&D Features of Pharmaceutical Industry R&D The Challenge of Infectious Disease The Challenge of Infectious Disease The Therapeutic Gap The Therapeutic Gap Bridging the Gap Bridging the Gap Features of PPPs Features of PPPs Metrics of Success Metrics of Success Recommendations/conclusions Recommendations/conclusions P P How to Maximise Chance of Success Clearly defined mission with a well articulated goal Clearly defined mission with a well articulated goal Adequate financing for the initial phases of mission and projection of total financing required to meet the end goal Adequate financing for the initial phases of mission and projection of total financing required to meet the end goal Top management team with access to the best science and a track-record in product development Top management team with access to the best science and a track-record in product development Plan which identifies the steps to be taken, by whom and when, in order to achieve the mission Plan which identifies the steps to be taken, by whom and when, in order to achieve the mission Real collaboration from partners with the expertise required Real collaboration from partners with the expertise required Active and independent oversight from an experienced board Active and independent oversight from an experienced board Robust portfolio with rigorous portfolio management processes Robust portfolio with rigorous portfolio management processes P P Indirect and Direct Measures of Success Direct fund raising success Direct fund raising success Early pipeline successes Early pipeline successes New attention to some neglected diseases by some major pharmaceutical companies and selected biotechnology firms New attention to some neglected diseases by some major pharmaceutical companies and selected biotechnology firms Early advocacy and education successes Early advocacy and education successes Development of some infrastructure including clinical trial networks Development of some infrastructure including clinical trial networks New collaborations between the North and South and some emphasis on capacity building New collaborations between the North and South and some emphasis on capacity building Development of a cadre of management talent, able and willing to apply private-sector models to public-sector neglected-disease challenges Development of a cadre of management talent, able and willing to apply private-sector models to public-sector neglected-disease challenges P P The Long Road to a New Medicine P P Agenda Global Healthcare Challenge Global Healthcare Challenge Features of Pharmaceutical Industry R&D Features of Pharmaceutical Industry R&D The Challenge of Infectious Disease The Challenge of Infectious Disease The Therapeutic Gap The Therapeutic Gap Bridging the Gap Bridging the Gap Features of PPPs Features of PPPs Metrics of Success Metrics of Success Recommendations/conclusions Recommendations/conclusions P P Recommendations/Conclusions Partnerships should be neither over-competitive with each other, nor monopolistic Partnerships should be neither over-competitive with each other, nor monopolistic A sense of common purpose and direction is needed that facilitates cross-linkages and synergies where appropriate A sense of common purpose and direction is needed that facilitates cross-linkages and synergies where appropriate The product profiles being sought by PPPs should be in line with global health need The product profiles being sought by PPPs should be in line with global health need Exploratory research, and early product R&D translational research needs to be supported to ensure that product R&D pipelines are maintained Exploratory research, and early product R&D translational research needs to be supported to ensure that product R&D pipelines are maintained Appropriate mechanisms need to be in place to ensure the continued and sustainable production and distribution of useful new products Appropriate mechanisms need to be in place to ensure the continued and sustainable production and distribution of useful new products P P Recommendations/Conclusions cont.d Appropriate downstream research implementation research needs to be undertaken to optimise product use and provide evidence for policy Appropriate downstream research implementation research needs to be undertaken to optimise product use and provide evidence for policy Conflicts of interest should be minimised Conflicts of interest should be minimised Capacity building and capacity utilisation in developing countries should be integrated into product R&D activities Capacity building and capacity utilisation in developing countries should be integrated into product R&D activities There should be strong stakeholdership from developing countries within the context of product R&D, product use and product delivery There should be strong stakeholdership from developing countries within the context of product R&D, product use and product delivery P P The Millennium Development Goals Eradicate extreme poverty and hunger Eradicate extreme poverty and hunger Achieve universal primary education Achieve universal primary education Promote gender equality and empower women Promote gender equality and empower women Reduce child mortality Reduce child mortality Improve maternal health Improve maternal health Combat HIV/AIDs, Malaria and other diseases Combat HIV/AIDs, Malaria and other diseases Ensure environmental sustainability Ensure environmental sustainability Build a global partnership for development Build a global partnership for development