Regulatory Science

The information proposed in this section provides key elements on the regulation of medicines for Rare Diseases in human and provides support in the:
Health Technology Assessment,
Drug repurposing,
Preclinical suties,
Early access support,
Clinical Development,
Marketing Authorization,
Ethic and legal issues,
Regulatory Agencies.

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  2. Regulatory Science
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Magisterial hospital preparations – hospital exemptions

Published by IRDiRC

Advanced TherapyATMPsEarly Access SupportHospital ExemptionRegulatory Affairs

Article 28 of the ATMP Regulation also empowers Member States to permit the manufacturing and use of certain non- routine produced advanced therapies that have not been authorised by the Commission under certain conditions (so-called “Hospital Exemption”) outside the scope of the Medicinal Product Directive 2001/23.

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Joint EMA-HTA Scientific Advice

Published by IRDiRC

EUnetHTAHTA BodiesRegulatory AffairsScientific Advice

The European Medicines Agency (EMA) offers consultations in parallel with the European Network for Health Technology Assessment (EUnetHTA) as of July 2017. This aims to allow medicine developers to obtain feedback from regulators and health technology assessment (HTA) bodies on their evidence-generation plans to support decision-making on marketing authorisation and reimbursement of new medicines at the same time.

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Advanced Therapy Medicinal Products (ATMPs) Classification

Published by IRDiRC

Advanced TherapyATMPs ClassificationEarly Access SupportEuropean UnionRegulatory Affairs

Companies can consult the European Medicines Agency (EMA) to determine whether a medicine they are developing is an advanced therapy medicinal product (ATMP). This is an opportunity for ATMP developers to receive confirmation that a medicine, which is based on genes, cells or tissues, meets the scientific criteria for defining an ATMP. The procedure can help developers to clarify the applicable regulatory framework since the beginning of the product development.

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EU Compassionate Use Programs

Published by IRDiRC

Clinical DevelopmentCompassionate UseEarly Access SupportEMA Compassionate UseEuropean UnionOrphan DrugRegulatory AffairsResearch and Drug Development

Compassionate use is a treatment option that allows the use of an unauthorised medicine. Under strict conditions, products in development can be made available to groups of patients who have a disease with no satisfactory authorised therapies and who cannot enter clinical trials.

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National Scientific Advice with HTA bodies

Published by IRDiRC

HTA BodiesHTA NationalNational Scientific AdviceRegulatory SciencesReimbursement

Regional and national HTA bodies in Europe, provide recommendations on medicines and other health technologies that can be financed or reimbursed by the healthcare system in a particular Member State or region. The assessment criteria used by HTA bodies differ between Member States, in accordance with regional and national legislation.

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National programs for early access

Published by IRDiRC

Clinical DevelopmentCompassionate UseRegulatory Sciences

Sometimes called “compassionate use”, expanded access is a potential pathway for a patient with an immediately life-threatening condition or serious disease or condition to gain access to an investigational medical product (drug, biologic, or medical device) for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available

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