Named patient programs should always be considered an exception and not a general rule to treat a patient
Dr Martin J Hug
With the overwhelming amount of medical information delivered at the fingertips of patients and health professionals it is tempting to explore treatment options outside the range of licensed drugs. Ever so often a pharmacist is confronted with questions like:”Ive heard about this new drug that does miracles in patients suffering from…- can you get me that drug?” Naturally, the pharmacist feels obligated to fulfil this wish but soon finds out that the “revolutionary” drug is not as easily available as initially anticipated. One of many reasons could be that the drug in question is not yet approved in the country of residence. Almost every drug on the market today has passed a strictly regulated procedure of preclinical and clinical trials that eventually lead to marketing approval. Despite the fact that the results of the clinical testing are often published years before the drug is commonly available, both physician and patient may anticipate that a licensed product that meets the medical need is already available. Sometimes even after the health authorities have approved a medicinal product the actual launch may be delayed thus keeping the licensed drug out of the market. A named patient treatment program may be one way out of this dilemma. Pharmacists can bridge the gap between the treating physician and the manufacturers in this process.
Even though the term “named patient treatment” appears so often in the literature there is neither a precise definition nor a general applicable procedure that describes the distribution or supply of an unlicensed product for the treatment of an individual patient. That very patient is most likely in a desperate, life threatening position and is seeking any help available. What can be done? We have to keep in mind that prior to receiving approval anywhere in the world manufacturers will test a drug within clinical trials. In such a case the safest option for both the patient and the treating physician would be to get the patient enrolled into a clinical trial. This is of course easier said than done since clinical trials are usually assigned to certain centres that may not be anywhere near to the location the patient lives or maybe not even in the same country. Even if the patient happens to be treated in a hospital that is participating in a trial involving the respective drug, he/she may still not meet the criteria for inclusion. Moreover, if the patient is successfully enrolled in a randomised clinical trial he still has a 50% chance to receive a placebo instead of the possibly active compound. In a collaborative effort drug manufacturers and health authorities have made it possible to give patients access to unlicensed drugs via so called “compassionate use programs.” The latter are similar to clinical trials, are registered as such and extensive briefing of both patient and physician are mandatory. Hence the term “compassionate” also implies that the drug is supplied to the patient free of charge. In return the manufacturer receives additional data on the safety and efficacy of the drug. Pharmacists can use their broad knowledge of the market and their relationship to the manufacturers to facilitate access to such programs.
Sometimes however, drugs are already approved but in a different country than the one the patient lives in. On the background of globalisation one might believe that drugs should be available everywhere for everyone. But each country has its own regulations that provide a legal and ethical way to allow access to patients who need these medicinal products. Unfortunately the legal framework is neither clearly understood on the national nor the international level.
Pharmacists are best advised to familiarise themselves with the legal implications that come with the use of unlicensed drugs. Any health care professional residing in the EU should be aware that “No medicinal product may be placed on the market of a Member State unless a marketing authorisation has been issued by the competent authorities of that Member State in accordance to this directive”. Any exemption from that rule must meet the requirements of article 83 of Regulation (EC) No 726/2004. Unauthorised medicinal products may thereby be made “available for compassionate reasons to a group of patients with a chronically or seriously debilitating disease or whose disease is considered to be life-threatening, and who can not be treated satisfactorily by an authorised medicinal product. The medicinal product concerned must either be the subject of an application for a marketing authorisation in accordance with Article 6 of this Regulation or must be undergoing clinical trials.” If meeting these requirements wasn’t a tall order already, one should keep in mind that making unlicensed medicinal products available either on a named patient basis or to cohorts of patients is mainly governed by individual EU member States legislation.
It is apparent that if a manufacturer is willing to implement a named patient treatment program he is best advised to collaborate with healthcare professionals that already have expertise in this field. However, both EU and national regulations seem to apply to the manufacturer than to the health care professionals.
Using a drug that has not been authorised by a national or European procedure is not without risks. The origin of the drug may not be easy to trace back especially if it is shipped from overseas. Many novel drugs require being stored at low temperatures, which mandates appropriate measures such as the use of temperature loggers. The manufacturer has to establish ways to secure that the supply chain remains uninterrupted. Has the drug already received market authorisation elsewhere it is not unlikely that criminals replace the original shipment with counterfeit products. Due to the lack of an appropriate sample in hand the pharmacist receiving the unlicensed drug may not be able to distinguish the counterfeit from the original drug. Keeping in mind that the use of unlicensed drugs is only justified when the patient suffers from a severe possibly life threatening disease it is also mandatory to keep shipping times and the paperwork involved at a minimum. It is therefore worthwhile collaborating with companies that provide safe, reliable and fast logistics.
Even though named patient treatment requires a lot of documentation, the information provided with the drug itself is sometimes very limited. Pharmacists often have to struggle with unclear instructions on the preparation of the applicable form; lacking data on the stability of reconstituted solutions and limited knowledge of side-effects and adverse events complicate the use of the drug. In some cases even a simple product information leaflet translated into the respective national language or at least into proper English is missing. One can only imagine the potential risks that result from improper preparation and administration of an unlicensed drug all the while little is known about the effect that drug may have on the individual patient.
Hospital pharmacists are responsible for the safe and appropriate use of medicines no matter whether these are licensed or not. To maintain the highest degree of safety standard operating procedures (SOP) on how to deal with unlicensed drugs should be implemented for each hospital. Due to the differences in national legislations a respective European wide SOP is far from being established as yet. Nevertheless the national and European societies of healthcare professionals should aim to collaborate on this issue helping to further eliminate risk factors in named patient treatments.
Pharmacists are also advocates for the patients and their treating physicians. They are in charge to deliver the requested information as reliable and quickly as possible. Network based solutions that provide fast access to information on named patient treatment programs can facilitate both access to and safety of unlicensed drugs. As an example, the German Association of Hospital pharmacists (ADKA) provides an email based network (“ADKA-intern”) restricted to its members. Here each participant can submit an inquiry on the status of approval and availability of a given drug. Due to the large number of members registered to this network most of the inquiries are getting answered within one or two days. Interestingly, questions on unlicensed drugs make up a significant share of all the inquiries submitted to the network. The service is yet on the stage of an emailing list but one could conceive converting it into a database with structured query functions that could promote the flow of information even further.
Named patient programs should always be considered an exception and not a general rule to treat a patient. Despite the risks involved when using an unlicensed drug pharmacists should be ready to face this option and become an aide to both the patient and the treating physician in this process, thus making named patient treatment a benefit and not a hazard.
1. Article 6, Directive 2001/83/EC of the European Parliament and of the Council of 6 November 2001 on the community code relating to medicinal products for human use. OJL- 311,28/11/2004:67-128.
2. Regulation (EC) No 726/2004 of the European Parliament and of the Council of 31 March 2004 laying down Community procedures for the authorisation and supervision of medicinal products for human and veterinary use and establishing a European Medicines Agency. OJL 136,30/4/2004:1-33.