Medicines Regulation: Introduction

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Department of Essential Medicines and Pharmaceutical Policies

Medicines Regulation: Introduction

Dr Lembit RägoCoordinator

Quality Assurance and Safety: MedicinesEssential Medicines and Pharmacutical Policies

World Health OrganizationGeneva, Switzerland

E-mail: [email protected]

EMP Technical Briefing Seminar, November 2011

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Medicines work in WHO HQ – in Health Systems and Services Cluster (HSS), ADG Dr Carissa Etienne

Department of Essential Medicines and Pharmaceutical Polices (EMP) Two teams

Medicines Access and Rational Use (MAR) Quality Assurance and Safety: Medicines (QSM)

Two units (teams) attached to Director's office Medicines Information and Evidence for Policy (MIE) Medicine Programme Coordination (MPC)

Collaboration with other clusters/departments/units Vaccines and biologicals (IVB/QSS) – vaccines prequalification Essential Health Technology (EHT) - diagnostics prequalification Disease oriented programs Other programs …

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Active collaboration with other international, regional and national organizations

UN family & international organizations: UNICEF, UNFPA, UNIDO etc. MSF

Regional Council of Europe/EDQM EMA/EU NEPAD

Professional and scientific FIP, IUPHAR, ISPE

National NMRAs

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QSM Technical Programmes

Regulatory Support International Nonproprietary Names (INNs) Quality Assurance Anticounterfeiting Safety (Pharmacovigilance) Prequalification of Medicines Quality Assurance and Safety of Blood Products and

Related Biologicals

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Why medicines are special category of products?

Consumers, patients and health care workers have limited capacity to judge there

SAFETY QUALITY EFFICACY

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SmellAppearance Taste

Usual perceptions may not help in Making judgements about medicines …

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Are all medicines safe, effective and meet quality criteria?

No, they are not

Some are safe, but not effective or necessarily meet the quality criteria

Some may be effective, meet quality criteria but are not safe

Some meet quality criteria but are not necessarily safe or have any efficacy

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Quality - Safety

Some safety parameters are determined by quality

Some safety parameters are determined by the intrinsic properties of active pharmaceutical ingredient(s)

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What type of medicines we have?

Originator products Multisource (generic) products

KEY – INTERCHANGEABILITY, more important THERAPEUTIC INTERCHNGEABILITY

ALL LITERATURE IS BASED ON ORGINATORS No interchangeability – NEED FOR NEW SAFETY and

EFFICACY DATA, NEW BOOKS HAVE TO BE WRITTEN

Other type of products Biological products including vaccines and blood products "Biosimilars" Radiopharmaceuticals Traditional medicines

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What type of regulations exist and how they differ?

For innovator products proof of QUALITY, SAFETY and EFFICACY is needed

For multisource products QUALITY, safety and efficacy data is referred to the originator providing only evidence about interchangeability (bioequivalence, clinical testing, in limited cases dissolution data)

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Regulations: Global vs National

National regulations still differ a lot

What is ICH and what it is not?

Regional harmonization initiatives

Do global norms exist for generics?

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In book: Drug Benefits and Risks, Chapter 6, 2008

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Main regulatory functions

Table 1. Principal medicines regulatory functions• Licensing of the manufacture, import, export, distribution,promotion and advertising of medicines• Assessing the safety, efficacy and quality of medicines,and issuing marketing authorization for individualproducts• Inspecting and surveillance of manufacturers, importers,wholesalers and dispensers of medicines• Controlling and monitoring the quality of medicineson the market• Controlling promotion and advertising of medicines• Monitoring safety of marketed medicines includingcollecting and analysing adverse reaction reports• Providing independent information on medicines toprofessionals and the public

Source: WHO Policy Perspectives on Medicines no 7, 2003.

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Is quality of medicines a problem?

Yes, a HUGE problem

If we would have the same compliance with norms and quality in aircraft industry Globally

approximately 25% planes would not take off the grounds

10% would crash and kill the people (treatment failure = killing in some cases; toxicity etc. )

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Quality can not be assessed, tested or inspected into the product, BUT

It has to be built into it!!

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Shifting the regulatory paradigm during the history (1)

From elementary quality requirements to safety and efficacy

From quality control of finished product to control of quality of manufacturing (inspection)

From quality control of finished product and inspection of manufacturing sites in general to more understanding the processes and product specific processes involved

From rigid limits to agreed upon beforehand set of limits which can be used by manufacturers in a more flexible manner (ICH QbD)

…..

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Shifting the regulatory paradigm during the history (2)

Increased role of science – new molecules, new advanced therapies and combination therapies (device + medicine, etc)

From national to international – not a single regulator today can work meaningfully in isolation and not using other regulators experience/knowledge/information

Increasing need to decide what regulatory functions to fulfil nationally and what expertise/capacities to build nationally

Increasing need for harmonization, collaboration and cooperation The future in medicines regulation is in effective collaboration

and cooperation

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Science and medicines regulation (example): European Medicines Agency (EMA) - First Scientific Workshop On Nanomedicines, 2-3 September 2010, London

Emerging therapies such as nonomedicines give rise to questions on the appropriateness of current regulatory frameworks, the relevance and adequacy of existing requirements and guidelines, and on the availability of adequate expertise to regulators.

Scientific challenges arise from the limitations of current testing methods and the reliability of novel ones, because of the 'nanosize' and the unique behaviour of such nano-systems in biological structures.

Examples of approved nonomedicines: the anti-neoplastic agent Caelyx includes stealth liposomes of doxorubicine

hydrochloride; the antineoplastic agent Mepact contains mifamurtide in multilamellar liposomes; the antineoplastic agent Abraxane contains paclitaxel nanoparticles bound to human

serum albumin; the immunosuppressant Rapamune contains sirolimus particles in nanocrystal

colloidal dispersion.

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Medicines Regulation: Introduction

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