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Herceptin (trastuzumab) provisionally approved

Media Release

23 March 2006

Herceptin (trastuzumab) provisionally approved

The cancer medicine Herceptin has been provisionally approved for the treatment of some women with early stage breast cancer.

The announcement was made today by the Medicines and Medical Devices Safety Authority (Medsafe).

The provisional consent limits the use of Herceptin to treat women with early breast cancer who test positive for the HER2 gene once they have had surgery and completed their adjuvant (additional) chemotherapy.

Due to concerns that use of Herceptin may be associated with heart damage, the provisional consent also limits the treatment to those women who have a normal heart function before treatment starts and requires women using Herceptin to have their heart function checked by echocardiogram every three months during treatment.

Medsafe's principal technical specialist, Dr Stewart Jessamine, says Medsafe is the first medicines regulatory authority to assess and approve Herceptin as a treatment for primary breast cancer.

The decision to approve Herceptin follows a positive recommendation from the Medicines Assessment Advisory Committee.

"The decision to give provisional consent means that Medsafe and the committee are satisfied that the benefits of treatment outweigh the risks at this stage. This decision has been made on the basis of limited data, and further data examining the overall safety and effectiveness of Herceptin in the long term is required before Herceptin can be considered for full consent," Dr Jessamine says.

As part of the approval process, the Medicines Assessment Advisory Committee considered an interim analysis of clinical trial results including the pivotal HERA study, and other published data. It concluded that treatment with Herceptin significantly reduced the recurrence rate of breast cancer compared to women who received no additional treatment.

The HERA study found that after 12 months, the recurrence of HER2 breast cancer was reduced by 9% after treatment with Herceptin (86% disease-free on Herceptin + standard adjuvant chemotherapy vs. 77% disease-free following standard adjuvant chemotherapy).

Another study includes a small number of women who have been followed up for up to four years. This study has reported that treatment with Herceptin in addition to standard adjuvant chemotherapy reduced the recurrence of HER2 breast cancer by 18% (85% disease-free on Herceptin+standard adjuvant chemotherapy vs. 67% disease-free on standard adjuvant chemotherapy).

Answering the question about whether preventing breast cancer recurrence also prevents premature death is more difficult at this stage.
One study found a 4% increase in survival rates at four years (91% survival of those treated with Herceptin at 4 years compared to 87% for those with standard treatment). However this study examined data at four years for less than 200 patients, which limits our confidence in the results. The range of survival rates from this study could be as low as 0.6% increase in survival or as high as 9%. However, the pivotal HERA study showed no significant difference in survival rates for those treated with Herceptin at 2 years follow-up.

In addition to demonstrating benefits, it was noted by the committee that research also demonstrated that use of Herceptin was associated with significant side effects. Of most concern was the finding that up to 3% of women developed severe heart failure while being treated with Herceptin.

"Due to these concerns the committee recommended that careful monitoring of heart function be undertaken as a condition of the provisional approval", Dr Jessamine says.

"Before the committee is able to consider an application for full approval of Herceptin the manufacturer has been asked to provide the committee with further safety and efficacy data as it becomes available, including data on long-term survival after treatment."

The Medicines Assessment Advisory Committee (MAAC) is a Ministerial advisory committee consisting of clinicians and experts in a range of specialties.

The members of the committee assess data on the safety and efficacy of medicines independently of Medsafe. Dr Jessamine wished to take this opportunity to publicly thank the committee for the professionalism they bring to the task, and the time they invest in evaluating medicines applications.

"I believe that the skills we apply when considering the safety and efficacy of medicines are up there with the rest of the world, and that it is good to see that a small regulator, such as Medsafe, can in the right circumstances be the first to assess and come to a conclusion about the safety of a new medicine, such as Herceptin."


Questions and Answers

What is the Medicines Assessment Advisory Committee?

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 Committee. The Committee's members 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.

What is the role of Medsafe in the medicines approval process?

Medsafe (the New Zealand Medicines and Medical Devices Safety Authority) is a regulatory arm of the Ministry of Health which is responsible for administering the Medicines Act 1981 (the Act). The Act sets out 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:

- Safety and quality of the ingredients

- Appropriateness of the manufacturing process and the quality systems which support it

- Quality of the finished product

- Packaging and labelling

- Studies to demonstrate the safety and efficacy of the medicine.

- The medicines assessment process involves:

- Submission by the sponsor of an application comprising data to address the safety, quality and efficacy issues.

- Evaluation of the data

- Correspondence between the regulator and the sponsor company (often iterative) to seek answers to technical queries and request additional data to address any issues.

How are medicines approved under the Medicines Act 1981?

Full consent: Medicines can be given full consent under Section 21 of the Act when all of the application requirements have been met.

Provisional consent: 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 data set available demonstrates that the risk: benefit profile of the medicine is acceptable.

Herceptin received provisional approval for the treatment of patients with metastatic breast cancer in 2001.

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 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.

Medsafe considers applications for consent to market medicines independently of PHARMAC (who are responsible for making decisions about which medicines to fund). The data and criteria considered by Medsafe and PHARMAC often differ when performing an evaluation. Medsafe considers only the safety, quality and efficacy of a medicine, it does not consider product cost or cost-comparison with other medicines in its deliberations. PHARMAC examines cost-utility and cost effectiveness of a medicine compared to other medicines that produce the same or similar effect, to determine how treatment with a medicine might impact on the health of New Zealanders and the pharmaceutical and overall health budget before coming to a decision about funding.

What approval has been sought for Herceptin?

In November 2005 Medsafe received an application to licence Herceptin for the treatment of primary breast cancer. This application requested extension of the provisional approval for Herceptin granted by Medsafe in 2001 for the treatment of patients with metastatic breast cancer to include treatment of patients in women following surgery and who have completed chemotherapy.

Information about Herceptin, its currently approved uses and side effects is available on the Medsafe website:

What happened after the Medicines Assessment Advisory Committee made its recommendations to Medsafe?

Medsafe considered the Committee's recommendation and in the first instance contacted the medicine's manufacturer about the outcome. In addition, the minutes of the meeting were rapidly ratified by the committee and then sent to the Minister of Health delegate for consideration and decision making.

What is Herceptin and what is HER2 positive breast cancer?

Herceptin or trastuzumab is a _cancer treatment which targets the HER2 protein found on the cells of some breast cancers. HER2 positive cancers tend to be more aggressive than other cancers that are HER2 negative (or don't have the protein). Herceptin has been used in trials to treat some HER2 positive breast cancers following, and in addition to, completion of standard breast cancer treatments: surgery, radiotherapy and or chemotherapy.

What are the studies of Herceptin which have been used by the MAAC in its consideration?

Early results have been reported from three large trials. One of these trials is a large European and worldwide trial called HERA and two other trials are being run in America. The trials have all looked at giving Herceptin to women who have completed standard treatments of surgery, radiation and or chemotherapy. The trials have been looking at whether this combined treatment reduces the risk of breast cancer recurring compared to the use of surgery and chemotherapy on its own.

The pivotal HERA (HERceptin Adjuvant Trial) is a long-term randomised clinical trial which has been investigating the use of Herceptin for 1 or 2 years versus no treatment (observation) following a range of surgery, radiation and chemotherapies. An interim analysis comparing the first year of the trial was published in the New England Medical Journal last year (October).

Another American study, the National Surgical Adjuvant Breast and Bowel Project trial B-31, has also been looking into the use of Herceptin for up to 4 years in a small number of women compared to a group who has no treatment following standard treatments of surgery, radiotherapy and or chemotherapy. The most recent analysis of this study was also published in the New England Medical Journal last year (October).

These are very early trial results and new data and information will need to be considered as it comes to hand, including long-term survival data.

Further information is available at:

What does adjuvant mean?

Adjuvant means in addition to and in combination with a range of other treatments. For example the standard treatment for cancer includes a combination of surgery, and or adjuvant radiation and chemotherapy treatments.

What is an echocardiogram?

It is a test used to examine the heart by using ultrasound. It allows clear images of the heart to be seen in a two-dimensional view, including viewing the heart chambers, valves and major blood vessels and enables problems to be detected. It can be used to calculate the volume of blood pumped out of the heart with each heart beat, (Left Ventricular Ejection Fraction), and so measure heart function.

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