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From Bottlenecks to Solutions: Clinical Trials

R x. D. From Bottlenecks to Solutions: Clinical Trials. Brendan M Buckley European Centre for Clinical Trials in Rare Diseases University College Cork National University of Ireland. Orphan Medicines: Concept. Orphan medicines are for Prevention, treatment or diagnosis, of

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From Bottlenecks to Solutions: Clinical Trials

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  1. Rx D From Bottlenecks to Solutions:Clinical Trials Brendan M Buckley European Centre for Clinical Trials in Rare Diseases University College Cork National University of Ireland.

  2. Orphan Medicines: Concept • Orphan medicines are for • Prevention, treatment or diagnosis, of • Life-threatening or very serious conditions • With a prevalence < 5 / 10 000 persons in EU • For which no satisfactory methods exist • Or for which significant benefit may be provided over existing treatments The European Orphan Drugs Regulation 1999

  3. The Reality of Orphan Drugs • The 1983 US Orphan Drug Act resulted in about 250 approved products, helping over 11 million patients. • Each additional orphan drug approval is estimated to have prevented, on average, more than 200 US deaths in the subsequent year (NBER Working Paper No. 8677) • Any one orphan drug in EU may help from 1 to 175,000 people.

  4. Orphan Drugs in the EU EU Centralised procedure at EMEA • Orphan designation by COMP (founded 2000) • 165 designated Orphan products* • First Marketing Authorisation August 2001 • 11 productswith Marketing Authorisation available to patients* *17th October 2003

  5. Magic is Not the Treatment for Disease

  6. Clinical Development of Orphan Medicines Patients with rare diseases need medicines that are • as safe • as effective • of the same quality as any commonly used medicine Clinical trials

  7. Apply for Orphan drug status Grant Orphan drug status Develop Orphan drug Safe Effective Marketing authorisation The Ideal Orphan Drug Process Industry EMEA COMP Industry EMEA CPMP Patients Benefit Appropriate use of good drug

  8. Industry Apply for Orphan drug status EMEA COMP Grant Orphan drug status Clinical Trial Data None / Insufficient / Poor quality ? Develop Orphan drug Industry Safe Effective Marketing authorisation The Usual Orphan Drug Process EMEA CPMP Patients Benefit Appropriate use of good drug

  9. Furberg & Furberg

  10. A Clinical Trial A Prospective study of an intervention Designed to measure the impact of a treatment Compared with a control treatment On a future possible outcome.

  11. The Clinical Trial Process • Design a workable protocol • Obtain Ethical and Regulatory approval • Recruit the right investigators • Recruit the right subjects • As fast as possible !

  12. The Clinical Trial Process • Conduct the study to highest quality standards of Good Clinical Practice • Monitor (quality assure) the study efficiently At the least financial cost !

  13. The Clinical Trial Process • Collect the data • Process the data • Interprete the data expertly • Present the data to the Regulators (EMEA, FDA etc.) • Communicate the results to the scientific community • Communicate the results to patients and carers As fast as possible !

  14. % Orphan Drugs 80 60 40 20 0 Small Medium Large Companies Challenges of Trials in Rare Diseases forIndustry • Orphan companies are often small • with limited geographic reach • with poor access to patients • lack trials experience • with limited regulatory experience

  15. Challenges of Trials in Rare Diseases • for Expert Physicians • Few patients per centre • Lack of funding • Lack of trials expertise • Multi-state Europe with diverse medical systems discourage development of major referral centres

  16. Challenges of Trials in Rare Diseases • for Patients • Few good trials • Patients’ participation wasted in poor trials • Ineffective channels for research funding (proposed studies too small) • Disincentive for companies to invest because of difficulties in doing trials

  17. Challenges of Trials in Rare Diseases • forRegulatory Agencies • Few adequate clinical trials for risk-benefit assessment • Poor regulatory compliance • Uncertain adherence to Good Clinical Practice • by industry • by doctors • Challenge of biostatistical study design for small cohort studies

  18. A Challenge for Europe • In contrast to the USA • Fragmentation • Multiple Languages • Multiple Cultures • Multiple Medical systems • Cross-border reimbursement difficulties • National rather than European Centres of Excellence • Pathetic level of direct Funding of research

  19. A Further Challenge • Pharmaceutical Research & Development activity has decreased in Europe, partly due to lack of government support • European patients risk being at “the back of the line” for new medicines • - The future depends on a long-sighted and creative partnership between Industry, Academia, Patients and European Governments

  20. Opening the Bottlenecks Possible Roles for Patient Organisations Pan-European Centres of Excellence • Encourage their development • Lobby for easier trans-national patient access to them • Support patients who travel from abroad to them

  21. Opening the Bottlenecks Possible Roles for Patient Organisations Act as True Stakeholders in the Clinical Trial Process: • Help ask the questions to be answered by trials • Give ethical guidance to Ethics Committees • Promote recruitment to good trials • Discourage recruitment to poor trials • Ensure publication of trial results

  22. Opening the Bottlenecks Some Roles for Academia • Facilitate clinical trials in rare diseases on a not-for profit basis. • Search for efficiencies in new methods for design of clinical trials. • Take a European view !

  23. Opening the Bottlenecks Some Roles for Academia Maximise effectiveness of rare disease trials • Effective multinational recruitment • Efficient use of scarce patient resources • Coordination of multiple study designs to facilitate meta-analyses • Long term open-label extension studies and structured post-marketing surveillance • Coordinated biobank use.

  24. European Institute for Clinical Trials in Rare Diseases A Pan-European Coalition between Academic Institutions and Patient Organisations to facilitate and accelerate orphan drug development in Europe in consultation with Regulatory Authorities.

  25. European Centre European Centre European Centre European Centre European Centre European Centre European Institute for Clinical Trials in Rare Diseases European Centre European Centre European Centre European Centre European Centre European Centre Patient Organisations

  26. Rx D European Centre for Clinical Trials in Rare Diseases University College Cork National University of Ireland. www.ucc.ie / ctc brenbuck@ucc.ie

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