07/04/2011. Phase I 15% Phase IV 20% Phase II 34% Phase I. Phase IV 17% 22% Phase III 22% Phase II 39% Phase III 31%

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1 La Ricerca clinica in Italia: il punto di vista della SIFO XII CONGRESSO NAZIONALE SSFA CROCEVIA DELLO SVILUPPO DEL FARMACO Roberta Joppi SIFO Dipartimento Farmaceutico - Verona Agenda Farmaci in sviluppo: il contesto internazionale e nazionale La ricerca clinica non-profit Le infrastrutture a sostegno della ricerca: un bisogno inevaso Quali regole per la ricerca clinica 1

2 Drugs in development According to the Pharmaceutical Research and Manufacturers of America (PhRMA) more than 2,900 new molecules are currently under development. Emerging medicines include: 300 potential drugs for rare diseases nearly 800 possible treatments for cancer 277 new medicines for heart disease and stroke 109 new drugs to fight and prevent HIV/AIDS [ Accessed Nov. 2010] Drugs in development Phase IV 17% Phase I 22% Total World: 28,533 Phase III 22% Phase II 39% Phase IV 20% Phase I 15% Total EU: 8,315 Phase III 31% Phase II 34% ClinicalTrials.gov. 29/03/2011 2

3 Il Registro italiano Fase IV 11% Bioeq./biodisp. 2% Fase II 37% Fase III 50% conclusi 53% in corso 47% Totale studi: (n) [ accesso del 16/11/2010 ] RCT multicentrici (Anni ) 2009) Nazionali 28% Tot.= Internazionali coord. EU 40% Internazionali coord. Italiano 8% Internazionali coord. Altro 24% OsSC, AIFA - La Sperimentazione Clinica dei Medicinali in Italia 9 Rapporto Anno

4 Drugs approved by the EMA ATC code ACTIVE PRINCIPLE TRADE NAME INDICATION Development in Italy Liraglutide Victoza Phase II Type 2 diabetes mellitus Prucalopride Resolor Symptomatic treatment of chronic constipation - A - Alimentary tract and Saxagliptin Onglyza Type 2 diabetes mellitus Phase III metabolism Repaglinide Enyglid Type 2 diabetes mellitus Phase III Tocofersolan Vedrop Vitamin E deficiency - Epoetin theta Biopoin/Eporatio Treatment of symptomatic anaemia associated with chronic renal failure - B - Blood and blood Prevention of atherothrombotic events in patients with Acute Coronary Syndrome forming organs Prasugrel Efient (PCI intervention) Phase III Romiplostim * Nplate Chronic immune thrombocytopenic purpura Phase II C - Cardiovascular Dronedarone Multaq Atrial Fibrillation Phase III system Tolvaptan Samsca Syndrome of inappropriate antidiuretic hormone secretion Phase II Bazedoxifene Conbriza Osteoporosis in postmenopausal women Phase III G- Genito-urinary Follitropin beta Fertavid Infertility - system and sex Lasofoxifene Fablyn Osteoporosis in postmenopausal women Phase III hormones Sildenafil Vizarsin Erectile dysfunction Phase III Ulipristal ellaone Emergency contraception - Aztreonam lysine * Cayston Pulmonary infections due to Pseudomonas aeruginosa in cystic fibrosis Phase III J - Anti-infectives for Human hepatitis B systemic use immunoglobulin Zutectra Prevention of hepatitis B virus - Canakinumab * Ilaris Cryopyrin-Associated Periodic Syndromes - Catumaxomab Removab Malignant ascites in patients with EpCAM-positive carcinomas - Certolizumab pegol Cimzia Active rheumatoid arthritis Phase III Degarelix Firmagon Advanced hormone-dependent prostate cancer Phase III Everolimus * Afinitor Advanced renal cell carcinoma Phase II Gefitinib Iressa Locally advanced or metastatic non-small cell lung cancer Phase II L - Antineoplastic and Golimumab Simponi Active rheumatoid arthritis Phase III immunomodulating Mifamurtide * Mepact High-grade resectable non-metastatic osteosarcoma - agents Plerixafor * Mozobil Mobilisation of haematopoietic stem cells Phase III Rilonacept * Arcalyst Cryopyrin-Associated Periodic Syndromes - (Regeneron) Tacrolimus Modigraf Prophylaxis of transplant rejection Phase II Tocilizumab RoActemra Active rheumatoid arthritis Phase III Ustekinumab Stelara Moderate to severe plaque psoriasis Phase IV Vinflunine Javlor Advanced or metastatic transitional cell carcinoma of the urothelial tract Phase II Autologous cartilage cells M - Musculo-skeletal expanded ex vivo ChondroCelect Cartilage defects of the femoral condyle of the knee - system Eptotermin alfa Opgenra Spondylolisthesis - Agomelatine Thymanax/Valdoxan Major depression Phase III Amifampridine Firdapse (Zenas) Lambert-Eaton myasthenic - * syndrome Caffeine citrate * Nymusa (Peyona) Primary apnoea of premature newborns - N - Nervous system Capsaicin Qutenza Peripheral neuropathic pain in non-diabetic adults - Eslicarbazepine acetate Exalief/Zebinix Epilepsy Phase III Rivastigmine Nimvastid Mild to moderately severe Alzheimer s dementia Phase III Hirobriz Breezhaler/ R - Respiratory system Onbrez Breezhaler/ Indacaterol Oslif Breezhaler Chronic obstructive pulmonary disease. Phase II V- Various Sevelamer carbonate Renvela Hyperphosphataemia in patients receiving haemodialysis or peritoneal dialysis. Phase II A - Alimentary tract and Sitagliptin Ristaben Type 2 diabetes mellitus Phase III metabolism Velaglucerase alfa * Vpriv Type 1 Gaucher disease. - Conestat alfa Ruconest Hereditary angioedema - B - Blood and blood Eltrombopag * Revolade Chronic immune thrombocytopenic purpura Phase III forming organs Ticagrelor Possia Prevention of atherothrombotic events in adult patients with ACS Phase III Regadenoson Rapiscan Pharmacological stress agent for radionuclide myocardial perfusion imaging - C - Cardiovascular Telmisartan Tolura Essential hypertension Phase III system Vernakalant Brinavess Atrial Fibrillation Phase III G - Genito-urinary Corifollitropin alfa Elonva Infertility - system and sex Silodosin Silodyx/Urorec Benign prostatic hyperplasia. Phase III L - Antineoplastic and Ofatumumab * Arzerra Refractory chronic lymphocytic leukaemia Phase II immunomodulating Pazopanib Votrient Advanced renal cell carcinoma Phase III agents Musculo-skeletal Thiotepa * Tepadina Conditioning treatment haematopoietic progenitor cell transplantation - M - system Denosumab Prolia Osteoporosis in postmenopausal women Phse III N - Nervous system Asenapine Sycrest Moderate to severe manic episodes Phase III R - Respiratory system Roflumilast Daxas Chronic obstructive pulmonary disease. Phase III S- Sensory organs Dexamethasone Ozurdex Macular oedema - V- Various Besilesomab Scintimun Scintigraphic imaging in adults with suspected osteomyelitis. - * Orphan Medicinal Products (Total: 13) Clinical trials on cardiovascular diseases in the World TOT: 9,816 Phase IV 23% Phase I 16% Phase III 29% Phase II 32% Completed 48% Ongoing 45% Phase III trials (2842) Discontinued or early terminated 7% ClinicalTrials.gov, December

5 I Cardiovascolari in Italia Tot. = 780 Fase IV 17% Biod/bioeq. 2% Fase II 22% Fase III 59% Conclusi 63% In corso 37% Pubblicati 12,6% [accesso del 22/03/2011] Dabigatran per la prevenzione dell Ictus in pazienti con Fibrillazione Atriale: sviluppato in Fase III [accesso del 22/03/2011] 5

6 Agenda Farmaci in sviluppo: il contesto internazionale e nazionale La ricerca clinica non-profit Le infrastrutture a sostegno della ricerca: un bisogno inevaso Quali regole per la ricerca clinica? Sperimentazioni per anno e promotore profit/no profit (67,5%) 437 (70%) (63,3%) 502 (64,4%) 516 (60,6%) PROFIT NO-PROFIT 443 (59,7%) 400 (41,5%) (37,1%) 287 (37,1%) (40,3%) (29,8%) 186 (32,6%) OsSC, AIFA - La Sperimentazione Clinica dei Medicinali in Italia 9 Rapporto Anno

7 Work Programme 2011 COOPERATION THEME 1 Health (European Commission C(2010) 4900 of 19 July 2010) STRATEGIC FRAMEWORK AND RESPONDING TO EU POLICY NEEDS The Health Theme is aligned with the fundamental objectives of EU policies: increasing innovation and competitiveness of European health-related industries and services and improving the health of European citizens. It also addresses global health issues and the socio-economic dimension in various areas of health research. In order to boost innovative drugs and health solutions in Europe the Health Theme makes a major effort into investigator-driven clinical trials in various fields. With a focus on brain-related diseases, diabetes, and cancer (incorporating life style issues and social determinants of health) the 2011 work programme addresses major health-related societal challenges. Finally, with a focus on antimicrobial drug resistance and emerging epidemics, the Health Theme continues to address global health issues of utmost importance. Attraverso la ricerca non-profit si promuovono studi clinici rilevanti allo scopo di: o colmare gap di conoscenza o migliorare la pratica clinica o rispondere a quesiti di Sanità Pubblica e di tipo regolatorio di limitato interesse commerciale Rilancio della ricerca clinica non-profit attraverso: ricostituzione della Commissione R&S dell AIFA rapporto stato di avanzamento degli studi finanziati e diffusione dei risultati relativi agli studi conclusi 7

8 Agenda Farmaci in sviluppo: il contesto internazionale e nazionale La ricerca clinica non-profit Le infrastrutture a sostegno della ricerca: un bisogno inevaso Quali regole per la ricerca clinica Clinical Research Infrastructure Research Networks Methodological support Data Management Financial Management Pharmacovigilance and drug dispensation Data Monitoring Educational Programmes European Clinical Research Infrastructures Network. 8

9 Funding of Infrastructures The Institut National de la Santé et de la Recherche Médicale (INSERM) supports infrastructures through a partnership with Hospitals corresponding to a total investment of 2M /y The UK Clinical Research Network (UKCRN) and a national framework for experimental medicine will provide much of the infrastructure required with a total new investment 100 M and 117 M over 5 years Best research for best health: a new National Health Research strategy Lo sviluppo della Ricerca Clinica profit e non- profit in Italia non può prescindere da: investimenti in INFRASTRUTTURE del SSN valutazione dei DG rispetto alle attività di ricerca condotte nell ambito delle Aziende Sanitarie 9

10 Agenda Farmaci in sviluppo: il contesto internazionale e nazionale La ricerca clinica non-profit Le infrastrutture a sostegno della ricerca: un bisogno inevaso Quali regole per la ricerca clinica Le GOOD CLINICAL PRACTICE sono uguali per tutti (ricerca profit o no-profit) i principi devono essere osservati le procedure devono essere flessibili 10

11 Monitoring The act of overseeing the progress of a clinical trial and of ensuring that it is conducted, recorded and reported accordance with the protocol, SOPs, GCPs and the applicable regulatory requirement(s) in (ICH GCP) Site monitoring accounts for 25-30% of the costs of a phase III trial Grazie per l attenzione! 11