Revised: 13 May 2013
January 2006 (updated February 2007 and February 2010)
The attached papers have been released by Medsafe to provide information about the regulatory approval process for the MeNZB vaccine produced by Chiron.
The data being released includes:
Medsafe (New Zealand Medicines and Medical Devices Safety Authority) is a business unit of the Ministry of Health and is the regulator responsible for administering the Medicines Act 1981 (the Act). The Act establishes a pre-market evaluation and approval system for medicines that is designed to ensure that new medicines meet the required standards.
The evaluation of medicines is based on internationally agreed standards which cover all aspects of the medicine including:
The medicines assessment process involves:
Two types of medicine approval are available under the Medicines Act 1981.
Medicines can be given full consent under Section 21 of the Act when all of the application requirements have been met.
Medicines can be given a provisional consent under Section 23 of the Act. This occurs in situations where there is a clinical need for the medicine and when the available data set, though less comprehensive than that required for full approval under Section 21, demonstrates that the risk: benefit profile of the medicine is acceptable.
Approval under Section 23 is valid for two years, but can be renewed, or converted to a full consent provided that the sponsor provides additional data justifying this approach. It is also possible to place conditions or restrictions on the use or supply of a medicine that is given provisional consent.
When assessing the "likely benefits" of medicines for approval under Section 23 it is not always possible to evaluate clinical trials measuring outcomes, such as "number of patients cured" or infections prevented, as this information may not yet be available. Medsafe therefore considers data from investigations such as blood tests that measure the effect of the medicine on the underlying cause of the disease. These tests are often called surrogate markers of effect, the level of HIV virus in the blood is an example of a surrogate marker for HIV/AIDS, where if a medicine lowers the level of virus, it is likely to indicate the medicine will be beneficial in the treatment of this disease. The measurement of protective antibodies in the blood following vaccination is another example of an acceptable surrogate marker of efficacy. The use of these surrogate markers allows medicines to become available on the basis of smaller amounts of information.
When considering approval under Section 23 of the Act, the risks and benefits of using the medicine are assessed against the risks and benefits of using available alternative products and the risks of denying access to the medicine. Approving a product under Section 23 allows a product to be used in the community at an earlier stage than would be possible if it was necessary to wait until further data were available to satisfy the data requirements for full consent. Section 23 is therefore a useful tool to allow early access to a treatment where other options are limited, or absent, such as a vaccine to manage an epidemic.
In the past, a number of products such as the HIV/AIDS medications were first approved under Section 23 when they initially became available, and have since obtained full consent as even more data demonstrating safety and efficacy became available.
MeNZB was approved under Section 23 of the Medicines Act 1981. The safety monitoring programme which has been implemented as part of the meningococcal B immunisation strategy was an important consideration during the approval process.
Further information on the safety monitoring programme for the MeNZB vaccine is available on the meningococcal B immunisation web site (http://www.moh.govt.nz/meningococcal).
The process involved input from two expert advisory committees, the United Kingdom medicines regulator and the UK-based laboratory for biological standards. The roles of these bodies are described below.
The Medicines Assessment Advisory Committee (MAAC) is an expert advisory committee established under the Medicines Act 1981 to provide advice to the Minister of Health. The Committee's role is to assess the safety, quality and efficacy of new medicines and advise whether the products meet the required standards. Medsafe provides secretarial services to the MAAC. The members of the MAAC have a broad range of expertise including clinical pharmacology, psychiatry, oncology, general practice and biostatistics. The Committee uses both Medsafe and International guidelines as the basis for their assessment process. Further details on the regulatory requirements for medicines are available in the "Regulatory Information" section of the Medsafe website.
The Vaccine Subcommittee (VSC) is a subcommittee of the MAAC. The VSC includes two members of the MAAC as well as four other clinicians with expertise in paediatrics, infectious diseases and general practice. Its role is to assess the safety, quality and efficacy of new vaccines and advise whether they should be approved for use. The VSC provides a written report of its considerations to the MAAC. The MAAC may either ratify or reject the VSC's report and recommendation. In November 2002, MAAC had agreed that the Chairman could ratify VSC decisions in certain circumstances. The application for approval of MeNZB was of such importance that the VSC recommendations were discussed by the whole Committee.
The Medicines and Healthcare Products Regulatory Agency (MHRA) is the medicines regulatory authority for the United Kingdom and performs a similar role to Medsafe. The MHRA has extensive experience in evaluating medicines and vaccines and in the past five years evaluated and approved a meningococcal vaccine to intervene in an epidemic of meningitis in the United Kingdom. The MHRA applies the same standards for evaluation as Medsafe and due to their experience was contracted by Medsafe to evaluate the manufacturing data contained in the MeNZB application and provide advice to Medsafe. The MHRA provided Medsafe with written reports on the safety and quality of the vaccine based on its evaluation of the original data set and the additional data submitted by Chiron in response to questions raised during the evaluation process.
Based in the UK, the National Institute for Biological Standards and Control (NIBSC) is a multidisciplinary scientific establishment which has a national and international role in the "standardisation and control" of biological substances used in medicines. Such substances include bacterial and viral vaccines against diseases such as meningitis, diphtheria and polio as well as products, such as clotting factors, derived from human blood.
The UK Health Department funds the NIBSC and the quality and validity of the control work undertaken at NIBSC is accredited by the United Kingdom Accreditation Service (UKAS) to comply with the International Standard BS EN ISO/IEC 17025.
The Institute is also a World Health Organisation International Laboratory for Biological Standards.
NIBSC provided peer review comments to MHRA on their evaluation report as well as scientific advice to both MHRA and Medsafe on aspects of the MeNZB vaccine.