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European Patients’ Academy on Therapeutic Innovation http://www.eupati.eu Fact Sheet: Summary of regulatory concepts and legislation, and the role of patient organisations Type of products Definition of the product Medicines for the treatment of rare diseases (life-threatening or chronically debilitating conditions that affect no more than 5 in 10,000 people in EU) Orphan medicines Key aspects of regulatory pathway for Marketing Authorisation (MA) application and approval Dedicated scientific committees Specific role of patient organisations European Medicines Agency (EMA) centralised procedure: mandatory COMP (EMA – Committee for Orphan Medicinal Products): assesses the Orphan Designation application Members of the COMP Application for Orphan Designation as a pre-requisite before MA application Incentives from EMA: scientific & technical support; fees reduction for small & medium enterprises (SMEs); extension of market exclusivity 1 European Patients’ Academy on Therapeutic Innovation http://www.eupati.eu Advancedtherapy medicinal products (ATMPs) Complex medicines made from genes and cells or tissues (different from conventional medicines made from chemicals or proteins) They propose novel approaches for treating diseases and injuries (and not only genetic diseases) and are from the most advanced scientific researches Medicines for children from birth to less than 18 years Paediatrics medicines EMA centralised procedure: mandatory CAT (EMA -Committee for Advanced Therapies): assess the MA application Members of the CAT PDCO (EMA – Paediatric Committee): assess the PIP and issue PIP waiver Members of the CAT Beyond quality, safety, and efficacy, assessment may also consider criteria due to the nature of the product: i.e., environmental risk Full traceability is required Incentives from EMA: fees reduction EMA centralised procedure Submission of a proposal of paediatric investigation plan (PIP) as pre-requisite before application at EMA level (mandatory for all new medicinal products) Application of MA must contain results and information required in the PIP – except when PIP has been waived when a product targets diseases not affecting children 2 European Patients’ Academy on Therapeutic Innovation http://www.eupati.eu Incentives from EMA : scientific consultation & extension of patent exclusivity Medicines developed to be the same as a medicine already authorised ‘the reference medicine’ Generic medicines Biosimilar medicines Contain the same active substances and used to treat the same diseases at the same dose EMA centralised procedure (under conditions) or national and other procedures No specific committee Application dossier: based on the reference medicine data and on the bioequivalence data of the generic compared to the reference But may differ on inactive ingredients, name, appearance, taste MA only granted after the period of exclusivity of the reference medicine has expired Medicines that are developed to be similar to biological medicines already authorised (biological medicines are complex medicines made or derived from a biological source/living cells and may contain blood and blood components, gene therapies, tissues, and proteins. EMA centralised procedure or national/ other procedures MA only granted after the period of exclusivity of the biological medicine (as reference) has expired No specific committee but BMWP (EMA – Working Party on Biosimilar Medicinal Products): provide recommendations to the CHMP and liaise with interesting parties Part of interested parties in liaison with BMWP 3