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Letters to the Editor
Managing the risks of thalidomide
From Mr F. G. Astbury
As president of Thalidomide UK, which represents the interests of thalidomide victims in the UK, I wish to respond to the article by David Thomson and Toby Capstick (PJ, 14 February, p190).
Thalidomide UK has provided the European Agency for the Evaluation of Medicinal Products with a detailed and confidential consultation on Pharmion’s application for a product licence. For the benefit of PJ readers, this letter presents an overview of some of our concerns.
Thalidomide UK firmly recognises that thalidomide is of major benefit in the treatment of multiple myeloma. It is not opposed to a risk management programme in principle. However, it has severe reservations as to the programme adopted by Pharmion — the Pharmion Risk Management Programme (PRMP) based on Celgene’s STEPS programme used in the US.
· In the first instance, PRMP is ill-suited to UK prescribing practice. From our discussions with oncologists and pharmacists, it is clear that the time-consuming and laborious inputs required by Pharmion from prescriber, patient and pharmacist could deter these groups from using thalidomide. We have particular concern over the intrusive nature of the disclosures that patients are required to give, not least of all in view of data protection considerations and concerns over patient confidentiality.
· Secondly, Thalidomide UK questions the ability of Pharmion to manage the PRMP at all. As reported in the national press, in December 2003 we were able to obtain thalidomide over the internet from Pharmion by registering as a bogus pharmacy in the PRMP. We understand that Pharmion claims to have since amended its procedures to close this loophole. Nonetheless, we strongly believe that such a major lapse severely compromises the company’s ability to control thalidomide distribution.
· Thirdly, we have had adverse comments from several pharmacists at children’s hospitals that, whereas the previous suppliers Penn Pharmaceuticals had a 25mg capsule available, Pharmion only markets a 50mg presentation, which is not appropriate for younger children and increases the administration risk.
· Our final concern is over the price currently being charged by Pharmion, which has increased 400 per cent since it took over supply from Penn Pharmaceuticals in July 2003. This clearly places an extra burden on the NHS and, once again from our discussions with oncologists and hospital pharmacists, could well prove a deterrent to it being prescribed for patients suffering from multiple myeloma.
In light of these and other concerns, Thalidomide UK does not support the PRMP nor does it support Pharmion’s current application for a product licence for thalidomide.
Patrick J. Mahaffy, president and chief executive officer, Pharmion Corporation, replies:
Pharmion is pleased that Thalidomide UK acknowledges the benefit that thalidomide can bring to patients with serious diseases and that it also endorses the need for a stringent safety system. However its letter raises a number of points that Pharmion would like to address.
In response to the first point pertaining to the suitability of the Pharmion Risk Management Programme (PRMP), Mr Astbury is correct that the PRMP is based on Celgene’s STEPS programme in the US. Pharmion wishes to point out that this system has proved highly effective and was a critical factor in the FDA approving Celgene’s licence application. Furthermore, Thalidomide Pharmion has now been licensed in Australia and New Zealand. In all cases the licence was granted on the condition that the Pharmion PRMP system was in place.
Although local health care systems vary between countries the aims of the PRMP remain the same — to protect patients and health care professionals and to ensure that the risks of a thalidomide baby being born are as minimal as possible. Pharmion acknowledges that the PRMP system is detailed and requires a high level of commitment by health care workers and by Pharmion. Health professionals accept the importance of a strict programme to ensure that a vital medical treatment is used safely and the terrible events of the 1960s are never repeated. The system has been in use in Europe for over eight months, many thousands of patients and health care professionals have registered and the system has been accepted and successfully implemented in many hospitals through out Europe.
In response to Mr Astbury’s comment regarding the management of the PRMP system, Pharmion acknowledges there have been several attempts by Thalidomide UK to register on the PRMP fraudulently and obtain thalidomide. In one instance thalidomide was successfully obtained and this has resulted in increased security around the PRMP system. The programme has been designed as a safeguard and information system for patients receiving thalidomide; it was not designed with the thought that individuals would try to break into the system fraudulently for the purposes of obtaining thalidomide. Pharmion has regularly involved Thalidomide UK in plans regarding licensing and risk management. As thalidomide is still easily obtainable from other sources which are selling the drug without any safety procedures, Pharmion is surprised that Thalidomide UK has decided to target the only company seeking to license and monitor the use of thalidomide.
Thirdly Mr Astbury mentioned that Pharmion does not currently offer a 25mg capsule of Thalidomide Pharmion. The company would like to point out that this presentation is not currently available because it is still awaiting EMEA approval. It is normal procedure for a limited range of doses to be developed before approval due to associated manufacturing costs. It is the company’s intention to extend the product range should a marketing authorisation be granted.
Finally, the company would like to respond to Mr Astbury’s concerns about the cost of Thalidomide Pharmion. At present thalidomide is unlicensed in Europe and is being provided by numerous companies with hugely varying degrees of quality control and safety systems. Pharmion is the only company that has applied for a formal marketing authorisation to distribute thalidomide and this licensing application is associated with many benefits for patients and professionals. These benefits are not possible, however, without investment from the company. With licensed thalidomide, patients can be confident that the product they are taking is manufactured to the highest level and there is no risk of variability between batches. All pack details, package inserts and information for Thalidomide Pharmion will comply with current EU regulations and are provided to each market in local language. The manufacturing of Thalidomide Pharmion is also audited by the regulator to ensure full compliance with Good Manufacturing Practice. Under the PRMP all reported side effects are recorded and compiled. Furthermore, Pharmion is committed to providing education to clinicians treating myeloma patients about the merits of thalidomide. Patients themselves will be able to get a full information pack on the product in all EU countries and in their local language. The company is also funding a succession of clinical trials to expand understanding of how the product works, moderate its side effects and look at new indications. No other supplier in Europe is currently investing in thalidomide safety and research to this extent.
Since the early stages of its plan to license Thalidomide Pharmion, Pharmion has maintained transparent and two way communications with the EMEA, patients’ associations, nursing groups and thalidomide victim groups throughout Europe to ensure the correct information is being provided to patients and all the necessary precautions to avoid pregnancy are in place. It is determined to ensure that Pharmion achieves a balance between the need to control safe access to thalidomide and the requirements of medical practitioners treating genuine patients.
With thalidomide still readily available throughout Europe from many unlicensed suppliers and even over the internet, it is unfortunate that Thalidomide UK is focusing its attention on Pharmion and the PRMP when the company fully welcomes its support and has made every effort to ensure it is informed about Pharmion’s activities. Pharmion believes that thalidomide should be a licensed and regulated product in Europe, as it is elsewhere in the world and that all interested parties should work together to bring this product under control as soon as possible.