Disregarded DISEASES: THE ORPHANEST DISEASES OF ALL

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Yamey, Brit. Med. J. 2002. What is a disregarded ailment?. . Unbalanced impact on LMI nations Gap in consideration from worldwide R

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Dismissed DISEASES: THE ORPHANEST DISEASES OF ALL Arrigo Schieppati, MD Clincal Research Center for Rare Diseases Mario Negri Institute Bergamo, Italy

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What is an ignored malady? Lopsided impact on LMI nations Gap in consideration from worldwide R&D Shortage of sheltered, compelling medicines Yamey, Brit. Med. J. 2002

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Operational meaning of dismissed infections From the U.S. Vagrant Drug Act Any sickness that either: influences under 200,000 people in the United States OR for which there is no sensible desire that the cost of creating and making accessible in the U.S. a treatment...can be recuperated from offers of the treatment.

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Neglected infections Disease Death Incidence Tubercolosis 3 Million 7.4 Million Malaria 2 Million 300-500 M HIV 1,5 M 3,1 Tripanosoma 150,000 200,000 Leishmaniasis 80,000 2 Million

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Neglected ailment: uncertain issues Tubercolosis  Resistance, no consistence Malaria  Resistance, no new medications farmaci HIV  Cost of treatment Tripanosomiasis  Only old, dangerous medication accessible Leishmaniasia  Old drugs, temperamental circulation

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Examples – Lymphatic Filariasis 40 million individuals for all time incapacitated or deformed by the sickness. 1/3 of cases in India, 1/3 in Africa, the rest scattered around LMI areas Worms are transmitted by mosquito chomp. Most evident indication is elephantitis. Crippling and trashing illness Treatment choices are constrained. www.who.int

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Sleeping Sickness Between 300-500K passings every year in Africa A neighborhood ailment – commonness as high as 20-half in a few regions Causes torment, migraines in its first stage, extreme neurological ailment – perplexity, rest cycle interruption, and so forth. Without treatment – lethal. Illustrations – African Trypanosomiasis www.who.int

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No money, no cure. The medication piece of the overall industry

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RICHEST POOREST

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The 90/10 crevice 90% of the infected individuals advantage from under 10% of overall biomedical research Only 1% of 1400 new medications which achieved the market amid the most recent 25 years are given to treat dismissed ailments

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The exploration hole Pecoul, PLoS Med. 2004

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Area New Drugs 1975-2000 Nervous System 211 (15%) Cardiovascular 179 (12.8% Cancer 111 (8%) Respiratory disease 89 (6.4%) Infectious disease 224 (16.1%) HIV/AIDS 26 (1.9%) Tubercolosis 3 (0.2%) Tropical diseases 13 (0.9%) Malaria 4 Other drugs 579 (41.6%) Totale 1393 Troullier et al, Lancet 2002

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R&D of new medications R is (generally) showcase - autonomous D is (to an expansive degree) advertise subordinate D costs > 10 x R costs

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Hence the ignored maladies No market, No new solutions Even when some examination is done, improvement is to a great extent lacking Vaccine are additionally dismissed (3% of the medication advertise)

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The scene of R&D for disregarded sicknesses Push and force motivators Push: coordinate financing or assistance of innovative work (stipends) Pull: guarantee downstream rewards by sorting out a business opportunity for possible finished results (licenses) Public-private organizations (PPPs) – scratch components for push, give subsidizing and pipeline administration Funding vehicles and propel buy contracts – essential cases of draw

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A Breakthrough in R&D for Neglected Diseases The scene of disregarded illness tranquilize advancement has changed drastically amid the previous five years, on account of the arrangement since 2000 of new pharmaceutical industry ignored ailment foundations, and the formation of new medication advancement open private association Moran et al. PLOS Sept 8, 2005

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A Breakthrough in R&D for Neglected Diseases: New Ways to Get the Drugs We Need 63 ND medicate ventures 3 new industry ND foundations 18 ND tranquilize extends in clinical trial and 2 in enlistment Translates into around 8-9 new medications by 2020 Moran et al. PLOS Sept 8, 2005

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New Ways to Get the Drugs We Need Small scale business Multinationa non revenue driven WHO In PPP alone 45% 25% 63 ventures Moran et al. PLOS Sept 8, 2005

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About DNDI In 2003, seven associations from around the globe united to set up DNDi: five open part establishments (Oswaldo Cruz Foundation from Brazil, Indian Council for Medical Research, Kenya Medical Research Institute, the Ministry of Health of Malaysia and France's Pasteur Institute) A helpful association, Médecins sans Frontières A universal research association, the UNDP/World Bank/WHO's Special Program for Research and Training in Tropical Disease

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DNDi doesn't direct research and logical work to create drugs itself Instead, it benefits from existing, divided R&D limit, particularly in the creating scene, and supplements it with extra aptitude as required.

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The part of multinational pharma organizations There are right now 32 ND medicate extend by MPC 4 organizations have ND divisions GSK, Norvartis, AstraZeneca, Sanofi-Aventis They are chipping away at a not-revenue driven premise

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Key figures the restored enthusiasm of MPC in ND Early contribution in R&D Involvement of open accomplices in clinical improvement stage R&D costs sponsored by open parntership Distribution of medications to not-at benefit costs

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Longer-term business contemplations for MPC being included in ND sedate advancement Minimizing the hazard to their notoriety originating from developing open weight on organizations over their inability to address creating nation needs Corporate social duty and moral concerns Strategic contemplations

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This recharged action initiated to a great extent without noteworthy new government motivators and for the most part without open intercession 80% of PPP medication advancement action is financed through private charity, while the business foundations are to a great extent self-subsidizing

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Where the cash originates from... Contributor Total subsidizing ($) % of aggregate Bill and Melissa Gates F. 158,757,717 58 MSF 29,738,133 11 Rockfeller F. 20,300,000 7.5 Wellcome Trust 2,827,504 1.1 SUBTOTAL 211,623,354 78.5 US Gov. 16,000,000 5.9 UK Gov. 10,909,468 4.1 Netherlands Gov. 10,489,255 3.9 Swiss Gov. 4,422,285 1.6 EU Commission 1,554,150 0.6 SUBTOTAL 43,585,077 16,2

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Nearly 50% of all PPP ventures (49%) and the greater part of industry collaborating ventures (63%) are in the "leap forward" class, contrasted with just 8% of medications created by industry working alone under the pre-2000 model.

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Health result The PPP approach conveyed the best wellbeing results for creating nation patients. Ivermectin : divided the worldwide weight of onchocerciasis in the vicinity of 1990 and 2000 Praziquantel : control schistosomiasis in Brazil, the Mahgreb, the Middle East, China, and the Philippines Coartem tablets mark augmentation for pediatric utilize: first protected, powerful, reasonable new hostile to malarial for a long time in Africa

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Government activities are still in the line of taking huge organizations back to the field Instead, the PPP approach is probably going to give better outcomes and ought to be upheld

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Armenia Moldova Nepal China Cuba Maroco India Nicaragua Philippines Nigeria Indonesia Bolivia Australia South Africa Paraguay Established projects Programs being developed

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Chronic Disease Outreach Program in Australia Tiwi Islands Wadeye Borroloola Broome Naiuyu Bega, Kalgoorlie Soweto, South Africa Chennai, India

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Bergamo BOLIVIA ITALY La Paz 'MARIO NEGRI' PER L'AMERICA LATINA El projecto de Enfermedades Renales en Bolivia Population: 8.328.700 CNS: 2,255,510 Life anticipation: 62 yrs Income for each month: 70 $ ESRD: 650 pt/yr Dialysis: 30/yr Transplant: 8/yr Identify subjects with renal ailment and give the premise to building a local and across the nation program of counteractive action of ailment movement to ESRD

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KIDNEY DISEASE SCREENING PROGRAM IN NEPAL Screening Team Community Screening at Dharan: 3218 individuals > 20 years old Dipstick proteinuria Demographic profile BP estimation Smoking and way of life Random glucose Follow up : At Renal Disease Prevention Clinic of Bisheswar Prasad Koirala Institute of Health Sciences (BPKIHS)

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THE PILOT EXPERIENCE OF ISN - WHO COOPERATION IN MOROCCO An epidemiological - mediation contemplate ISN-COMGAN Res Com ( M. De Broe ) WHO - Morocco ( R. Ben Ammar ) Morocco Ministry of Health Morocco Nephrology Society ( M. Benghanem Gharbi ) Khemisset ( country ) 25,000 individuals to be screened El Jadida ( industrialized )

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