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Revised: 22 October 2014

Use of Unapproved Medicines and Unapproved Use of Medicines

The Medicines Act 1981 permits an authorised prescriber to prescribe, administer or arrange for the administration of medicines for the treatment of a patient in his or her care. The medicine and its use may or may not be approved. The Act also permits the sale or supply of unapproved medicines to registered medical practitioners, but requires the supplier to notify the Director-General of Health. Approval is obtained when a sponsor company has sought and received Ministerial consent to the marketing of that medicine and the indications, contraindications, etc. are set out in the current data sheet.

The Code of Health and Disability Services Consumers' Rights places obligations on the provider of services. The consumer has the right to treatment of an appropriate ethical and professional standard, and the provider has the responsibility to ensure treatment, whether approved or unapproved, meets this standard. The consumer also has the right to be fully informed. If the use of a medicine is unapproved, the consumer should be so advised and the provider should be frank about the standard of support for the use and any safety concerns. The Code requires written consent for experimental use of a medicine. The unapproved use of a medicine would be considered to be experimental if there is little or equivocal documented support for the use.

Medsafe is aware that some authorised prescribers are confused about their rights and responsibilities with respect to prescribing unapproved medicines, or approved medicines for unapproved uses (such as unapproved indication, dosage or route of administration). Recent correspondence has raised concerns about the legal position of the practitioner who prescribes a medicine in a situation in which it is contraindicated, or against which there are warnings in the data sheet. Examples of these situations are the use of Depo-Medrol injection for epidural administration, and nifedipine capsules in the treatment of hypertensive crisis in pregnancy.

This article will attempt to clarify the situation with regard to the Medicines Act 1981. It will also apply the requirements of the Code of Health and Disability Services Consumers' Rights to the unapproved use of medicines. At the end of the article are four scenarios which illustrate the points made and describe correct procedure. The reader may find the material more accessible if the scenarios are read first.

Use of medicines regulated by the Medicines Act

The Medicines Act requires the Minister's consent to be given before a medicine is allowed to be marketed. An application with the necessary supporting documentation must be submitted. The Minister's consent is notified in the New Zealand Gazette, at which time the medicine, along with a set of indications, dosage instructions and route(s) of administration, is regarded as being approved. Proposed changes, including new indications and changes to the data sheet, also have to be applied for.

Because of this requirement for seeking and obtaining consent, it follows that there will be medicines that may be effective and safe, and approved in other countries, but do not have approval in New Zealand. There will also be other medicines that have been approved with a particular set of indications, but for which there are other recognised indications not applied for in New Zealand. Some unapproved medicines may be used for rare diseases, for which there are few or no treatments approved in this country.

Hence, the need to provide for access to unapproved medicines was recognised when the Medicines Act was formulated. Section 25 of the Act permits authorised prescribers to procure, administer and arrange the administration of an unapproved medicine. Section 29 permits the sale or supply of an unapproved medicine to a medical practitioner for the treatment under that medical practitioner’s care provided the Director-General of Health is notified of that supply. Both sections of the Act need further explanation.

Section 25 of the Medicines Act permits use of unapproved medicines

The terms of section 25 are inclusive and permissive, allowing the authorised prescriber to "procure the sale or supply of any medicine" for a particular patient in his or her care. "Any medicine" includes approved and unapproved medicines. The authorised prescriber must always be working within his / her scope of practice when exercising this right.

"Procure the sale or supply" refers to obtaining the medicine through the usual channels such as a pharmacy or a pharmaceutical company, and it also permits the authorised prescriber to use other means of obtaining a medicine such as importation. However, section 25 does not envisage bulk purchase by the authorised prescriber. The use is to be for the treatment of a particular patient in the care of that or another authorised prescriber.

It is worth mentioning, at this point, the use of approved medicines for unapproved uses. Section 25 permits an authorised prescriber to use any medicine (approved or unapproved) for the treatment of a particular patient in his or her care. The Act puts no restriction on the use of a medicine, even in a situation in which it is contraindicated. However, whether the authorised prescriber uses approved or unapproved medicines, he or she must provide care of an adequate professional and ethical standard (see the discussion of the Code of Health and Disability Consumers' Rights later in this article).

Section 29 requires notification of sale or supply of unapproved medicines

Section 29 of the Act permits the sale or supply to medical practitioners of medicines that have not been approved, and requires the "person" who sells or supplies the medicine to notify the Director-General of Health of that sale or supply in writing naming the medical practitioner and the patient, describing the medicine and the date and place of sale or supply, and the number of packs supplied.

No notification is required for an unapproved use of an approved medicine, nor is notification required if an authorised practitioner imports a medicine to treat his or her patient. However, if an unapproved medicine is sold or supplied to a medical practitioner, that sale or supply should be notified. If the supply is from one authorised prescriber to another, the supplying authorised prescriber is encouraged to notify the supply, but notification of supply is not mandatory in this case.

It should be noted that section 29 specifies only medical practitioners. This means that if other authorised prescribers wish to procure unapproved medicines their sources are limited to other authorised prescribers or to direct importation.

On occasions a pharmacist working in a pharmacy may be involved in the supply of an unapproved medicine as the medical practitioner's agent. If the pharmacy has imported the medicine, it is the pharmacist's responsibility to ensure that the details of supply are sent to Medsafe. If the medicine has been obtained from a distributor for an identified patient then that distributor should be given all information required to be held, according to section 29.

Anyone who imports an unapproved medicine for supply to a medical practitioner under Section 29 (other than a hospital or pharmacy) should ensure that they hold a licence to sell medicines by wholesale. They should also ensure that they hold the product specifications and certificates of analysis for each batch imported as required by Section 42.

Section 29 supply will be audited as part of the annual audit for a Wholesale licence.

Section 29 Notification Form (Microsoft Word document, 26 KB, 1 page)

Patient should be advised of the forwarding of information under section 29

Note that under the Health Information Privacy Code, Rule 3, the medical practitioner must advise the patient that information about supply of the medicine will be forwarded to Medsafe and recorded on a database as a requirement of the Medicines Act.

Consumers' rights spelt out in the Code

The Code of Health and Disability Services Consumers' Rights, which was introduced in July 1996, specifies certain rights of consumers of health and disability services and as a result places obligations on the practitioner. The Code covers the right to treatment of an appropriate ethical and professional standard, the right to be fully informed about condition and treatment options, and the right to make an informed choice.

Right to services of an appropriate standard

Right 4 of the Code refers to the right to services of an appropriate standard. This right includes:

2) Every consumer has the right to have services provided that comply with legal, professional, ethical, and other relevant standards.

4) Every consumer has the right to have services provided in a manner that minimises the potential harm to, and optimises the quality of life of, that consumer.

This right covers the professional standard of the service provided. Whether the medicine or use of the medicine is approved or unapproved, the practitioner must approach the decision for its administration in a professional, scientific manner which includes weighing the expected benefits and risks.

This right also applies to the situation where a patient requests the prescription of an unapproved medicine, about which the practitioner knows nothing. In such cases, the practitioner must seek to be adequately informed before assisting the patient to obtain supplies of the medicine. Only then will the practitioner be in a position to comply with Rights 6 and 7, which are discussed below.

It is conceivable that the practitioner will be unable to find sufficient information to convince him or her of either the efficacy or the safety of a medicine requested by a patient. The decision must then be made whether or not to assist the patient to obtain the medicine and conduct a clinical trial of one patient. In anticipation of such situations the practitioner needs to pre-determine what are his or her minimum criteria for regarding a medicine as a treatment option, and also what will be his or her standard of patient monitoring to minimise harm should the decision be to proceed with treatment in situations where documentation is severely limited.

Right to be fully informed

Right 6 of the Code covers the right to be fully informed.

1) Every consumer has the right to the information that a reasonable consumer, in that consumer's circumstances, would expect to receive, including...

b) An explanation of the options available, including an assessment of the expected risks, side effects, benefits, and cost of each option;...

For an unapproved medicine or unapproved use, the consumer should be advised of the unapproved status. The consumer should also be advised of the degree and standard of the support for the use of the medicine, and of any safety concerns, or warnings or contraindications regarding its use in their particular condition. In using the phrase "Right to be fully informed", the Code requires frank disclosure of information including information that may dissuade the consumer from agreeing to use of the medicine.

The following are examples of the information that should be conveyed, as appropriate:

With regard to the first point above, it is worth noting that a medicine only obtains approval if there has been an application, with adequate supporting documentation, from a sponsor company to the Director-General of Health. There may be adequate data supporting the use of a medicine, or supporting a different indication or dosage regimen, but if no company has submitted an application there can be no marketing approval in this country.

The use of medicines in certain groups, such as infants and small children, pregnant women, and frail or elderly patients can be a dilemma for the practitioner. Usually trials have not been conducted in these groups for a number of reasons including ethical ones. Therefore, the sponsor company is unwilling and unable to recommend such use in its data sheet and this becomes an unapproved indication.

Right to give informed consent

Right 7 refers to the right to make an informed choice and give informed consent:

6) Where informed consent to a health care procedure is required, it must be in writing if...

b) The procedure is experimental;...

The use of an unapproved medicine, or the unapproved use of a medicine, will not always be experimental, but in some circumstances the requirement to obtain written consent will apply. These would include situations where:

Prescribers need to take responsibility for thinking through the issues, deciding in each situation whether the use is experimental or not, and taking the necessary action. If the use is not judged to be experimental the consumer still has the right to make an informed choice and give informed consent to the treatment.

Note that the obtaining of written consent does not mean that the requirements of the Code have been complied with. The obtaining of informed consent is a process which involves effective communication, frank information disclosure and freely given consent. It also involves careful investigation of the clinical condition of the patient and maintaining a current knowledge of treatment options.

The following four scenarios illustrate what is permitted under the Medicines Act and what is required by both the Medicines Act and the Code of Health and Disability Services Consumers' Rights.

Scenario 1: Unapproved medicine

A patient comes to a medical practitioner’s surgery complaining of insomnia and requesting melatonin. The patient claims that melatonin has been helpful in the past and says that it can be obtained from Company X in Auckland. The practitioner knows that melatonin1 is not approved and that it has recently been scheduled a prescription medicine. He has no knowledge of the safety or efficacy of melatonin. The practitioner is obliged to use the means available to him to obtain unbiased information on the efficacy and safety of melatonin in the treatment of insomnia, and be assured that it may benefit and not harm the patient. He is then able to decide whether he wishes on scientific grounds to assist the patient to obtain melatonin. He should discuss with the patient the information obtained, as well as the nature of the patient’s insomnia, and other medical and non-medical treatment options.

If, after a full and frank discussion, the decision of both the medical practitioner and the patient is to use melatonin, the practitioner has three further steps to take before obtaining supplies. He should decide whether melatonin for insomnia should be regarded as experimental, necessitating signed consent. If it is considered experimental he should agree with the patient on a suitable procedure for monitoring for safety and efficacy. Finally, he should advise the patient that it is a requirement under the Medicines Act for the information about the supply, including the patient's name, to be forwarded to Medsafe and be stored in a database.

The practitioner then contacts Company X and requests melatonin for his patient. If the medicine is procured from Company X, the company must report the supply to the Director-General.

Scenario 2: Unapproved medicine

A patient who has recently returned from two years working in Canada has been taking Medicine A for rheumatoid arthritis and has found it to be more effective in easing the pain and improving mobility than any other medicine he has used to date. He would like to continue using this medicine. His general practitioner checks the MIMS New Ethicals Catalogue and cannot find either the medicine or the manufacturer listed. She contacts Medsafe (or searches the ‘Product/Application database’ function on the Medsafe website and is informed that it is not available in New Zealand and no application for consent has been received for this medicine.

Through the medical school library the practitioner is able to obtain some information on the efficacy and safety of Medicine A in the management of rheumatoid arthritis. She also finds that Medicine A has been available in the United Kingdom, France and Canada for about 20 years. The general practitioner also contacts a rheumatologist, who advises that she was aware of the effective use of Medicine A when she was working in the UK for a year.

After this process the general practitioner is persuaded that Medicine A could be a useful and safe medicine for this purpose, and she agrees to write on behalf of her patient to the manufacturer at the address on the bottle supplied by the patient. She also discusses the treatment with another general practitioner who has a patient with difficult to manage rheumatoid arthritis. This colleague requests some of the medicine for his patient.

When the supplies of the medicine arrive, the first medical practitioner supplies one bottle to her patient and another to her colleague. She reports to the Director-General of Health the supply of the medicine to the other practitioner, including the name of the practitioner, name of the other practitioner’s patient and the date and place of supply.

Scenario 3: Unapproved indication and route of administration

A man visits a pain clinic with long term disabling back pain. The specialist he sees at the clinic believes that an epidural injection of Depo-Medrol may ease the patient’s pain, and restore a degree of quality of life. She has successfully treated a number of back pain sufferers with Depo-Medrol in the past 12 months, and so have her colleagues at the same clinic. She believes that she is able to administer the injection with accuracy and skill.

However, she is aware that epidural Depo-Medrol is not an approved treatment for back pain, and the manufacturer has included a warning in the data sheet stating that "Depo-Medrol should not be administered by any route other than those listed under Indications." In addition, she is aware that the studies examining the efficacy of epidural steroid injections in the management of back pain are not well-controlled and not all show significant gains in pain relief. She also knows that some New Zealand back pain sufferers have claimed that they have been more seriously disabled following treatment with an epidural corticosteroid injection.

The specialist presents epidural Depo-Medrol to the patient as a possible treatment option. She mentions that it is not an approved treatment, that the manufacturer of the medicine warns against this use, that the evidence for its efficacy in clinical trials is not well-supported, and that some recipients treated at other clinics believed they were more seriously disabled following this treatment. She also explains that in her experience it is a valuable option for the treatment of back pain and that she has administered it successfully in the past. The patient and the specialist discuss why the manufacturer may warn against this use and why other patients may claim they have been further disabled by it. The specialist gives the patient a leaflet that discusses this and other treatments for lower back pain and invites the patient to consider the options and return to the clinic the following week.

When the patient returns, he asks a few questions and then advises the specialist he wishes to have the epidural Depo-Medrol injection. Because the documented evidence of efficacy is equivocal, the manufacturer warns against use in unapproved indications, and some patients consider they have been seriously disabled by treatment, the specialist considers the treatment should be regarded as experimental under the Code, and explains this to the patient with reasons. She asks the patient to sign a form indicating he has agreed to treatment with epidural Depo-Medrol.

Scenario 4: Unapproved indication

A patient is admitted for the placement of a coronary artery stent. The specialist routinely prescribes ticlopidine and aspirin for antiplatelet therapy after this procedure. Ticlopidine is an approved medicine, but it is not approved for antiplatelet therapy following stenting2 (ticlopidine [Ticlid] is now approved for this indication). A recent large scale well-designed study demonstrated that ticlopidine and aspirin were an optimum treatment option in this indication. The specialist and his colleagues had carefully discussed the merits of the study and other knowledge they had of the treatment options, together with other views expressed in letters to the editor, and had decided to use ticlopidine and aspirin.

Before the procedure the specialist discusses the need for anticoagulant or antiplatelet therapy and the treatment options with the patient. He explains that ticlopidine is not approved for this indication but recent data have shown it to be an effective treatment with better long term outcomes than other options. He briefly discusses the nature of the data. The specialist also explains the need for biweekly blood monitoring because of a risk of bone marrow suppression, and advises the patient to contact her general practitioner at the first sign of any infection.

The patient agrees that the combination of aspirin and ticlopidine appears to be the best choice. She is not invited to sign a consent form for ticlopidine treatment because the specialist has decided that the evidence for efficacy and safety is substantial and its use cannot be regarded as experimental.

This article has been prepared in consultation with the Health and Disability Commissioner. The reader should note that the Health and Disability Commissioner does not give advance rulings on interpretation and application of the Code, and hence it is not possible to say in advance that a particular practice is or is not in breach of the Code. This provision is to ensure that each complaint is considered impartially and with an open mind.

Disclaimer: Mention of the therapies in the scenarios described in this article should not be regarded as implied endorsement on the part of Medsafe.

Medsafe accepts no responsibility or liability in respect of the actions or omissions of any person arising from or as a consequence of any statement in this article.

  1. This statement was accurate at the time this article was first published in April 1998. However, in 2011, melatonin (Circadin) was approved for the short term treatment of primary insomnia, characterised by poor quality of sleep, in patients over 55 years of age. The scenario continues to aptly illustrate the point even though it no longer applies to melatonin.
  2. This statement was accurate at the time this article was first published in April 1998. However, in 1999 ticlopidine (Ticlid) was approved for antiplatelet therapy following coronary artery stenting. The scenario continues to aptly illustrate the point even though it no longer applies to this indication of ticlopidine.
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