Poll of the week: "Is Genzyme morally corrupt?"

Jeremy Laurance. Neurologists appeal to health secretary over withdrawal of drug for MS patients. BMJ 2012; 345 doi: http://dx.doi.org/10.1136/bmj.e7449 (Published 2 November 2012)

..... Three neurologists have called on health secretary Jeremy Hunt to intervene after a drug company withdrew off label use of its drug alemtuzumab for patients with multiple sclerosis ahead of trial results into its efficacy.

The doctors said they are disappointed by the actions of Genzyme, a subsidiary of the multinational drug company Sanofi. One called the company “morally corrupt” for disrupting the treatment of patients in favour of future profits.

Genzyme funded two trials of alemtuzumab that were published in the Lancet. These found that alemtuzumab dramatically cut the relapse rate compared with the current most effective treatment, interferon beta-1a. 

Researchers from the University of Cambridge announced that they had found the best treatment yet for multiple sclerosis, which had a “transformative” effect on patients. Alemtuzumab could be suitable for up to two thirds of the 6000 patients who are newly diagnosed each year, they said.

Alemtuzumab was developed as a treatment for leukaemia at Cambridge University in the 1970s, but was later discovered to be effective in multiple sclerosis. The first patient to receive it was treated in 1991.

Since then the drug has been owned by 13 different companies, some of whom bought it to close down its development, according to Alasdair Coles, a neurologist at the University of Cambridge who led one of the trials.

Genzyme, which acquired the drug in 2004, funded the latest trials and had been “very supportive,” Coles said. Alemtuzumab is given in two courses a year apart and currently costs around £2500 (€3100; $4000) a patient.

But the culmination of 20 years’ research by the Cambridge scientists was overshadowed by Genzyme’s decision to withdraw off label access to the drug for existing patients, pending the outcome of the trials.

The decision, announced last summer, triggered a protest by Neil Scolding of the University of Bristol, Neil Robertson of the University Hospital of Wales, and John Zajicek of the University of Plymouth who wrote to Hunt appealing to him to intervene.

In their letter the neurologists said that Genzyme’s action had “serious implications for vulnerable UK patients with MS.”

Patients who had already started treatment would “not be able to get their vital second course,” and new patients might “miss their window of therapeutic opportunity” thereby putting them at risk of “progressive, severe disability.”

When licensed, the drug’s price was expected to be “15 to 20 times greater” and its withdrawal set an “inappropriate precedent.” They added: “It shows little regard for patients whose opportunity to alter the course of their disease is time limited, and may represent an over-enthusiastic attempt by the parent company to profit from the current situation.”

Their letter concluded: “We think it important to bring these issues to your attention in the hope of protecting a vulnerable group of patients, and that a more humane approach to drug development in this area can be fostered, and that with your help steps may be taken to secure continued access to the drug for UK patients.”

Zajicek said he had personally treated 150 patients with the drug off label and estimated 400 to 500 had received it across the UK.

“Many of us think it is the best drug for patients with aggressive MS in the early stages of the disease. It’s the greedy behaviour of the drug company that upsets me. They are just trying to rebrand it and put the price up. It is morally corrupt,” he said.

A spokesperson for Genzyme said it had restricted off label use of alemtuzumab in multiple sclerosis to clinical trials because “any adverse event outside a clinical trial setting not only poses a risk to the individual patient but may complicate the regulatory process thereby delaying the availability of the drug for the MS community.”

The price of alemtuzumab would be established after it was licensed and would be subject to scrutiny by the National Institute for Health and Clinical Excellence (NICE). “We are committed as a company to engaging constructively in this process,” the spokesperson said......

"We have debated the issues raised in this article many times on this blog. Is Genzyme being greedy when both the USA and EU have put in place legislation to encourage pharma companies to repurpose drugs, such as alemtuzumab, and to develop drugs for orphan indications? There seems to be a disconnect between political policy and the public in terms of the expected consequences of this legislation. The politicians are taking a macroeconomic look at  things and have put incentives in place to encourage R&D in areas that nobody would have invested in the past. Politicians clearly want a healthy, thriving, pharma industry that is making profits and reinvesting them in R&D. Politicians also believe that this will ultimately lead to improved population health and address the problem we have with orphan diseases."

"From my perspective the problem lies in the public response to this legislation, i.e. when companies invest and take measures to recoup their investment we cry foul and call them morally corrupt. Are we correct in doing this? Clearly, both the industry and the politicians need a forum to discuss these events in the open so that public understand their implications. When it comes to excessive profits, at least outside the USA, governments' have policies in place to bring down the price of drugs; this is NICE's remit. The NHS only pays for drugs that are deemed to be cost-effective. Therefore, before we start criticising Genzyme, and the Pharma Industry, we need to wait and see what NICE has to say about the cost-effectiveness of alemtuzumab for the treatment of RRMS. More worrying that NICE is what will the EMA have to say about Alemtuzumab? Will the EMA limit the prescribing of alemtuzumab to highly-active MSers, or only for those MSers who have failed first and second line treatments? The EMA does not make decisions on price but risk:benefits. I am acutely aware from my experience with cladribine that you cannot take the EMA for granted. The alemtuzumab development programme was done to provide data on the risk:benefit ratio of using alemtuzumab as early as possible in MS. I am very worried, not for myself but for MSers, that the EMA won't allow us this option."

"One thing that Genzyme do have a responsibility for is to ensure that all the MSers who have been treated with alemtuzumab off-license and need further courses get speedy and appropriate access to the drug. To the best of my knowledge the company has reached out to the neurology community to allow this to happen."

"Some readers of this blog feel we don't need pharma and that we could simply develop drugs in our academic institutions and the NHS. This is simply not possible, or at least not possible at a scale and intensity that is required to address the medically unmet needs of the world. Mouse Doctor and I, are currently involved in a programme to develop and commercialise a small molecule for the treatment of spasticity for MSers. Let me tell you the resources and infrastructure simply don't exist within academia and the NHS to do this with in any reasonable time span, or at a level that is needed to feed the insatiable appetite of people with chronic diseases have for both disease-modifying drugs and symptomatic treatments. Just look at how frustrated MSers are with the lack of drugs for progressive MS. Who do you think has the resources to develop and test drugs in progressive MS? The risks associated with drug development are also so large, both financially and medico-legally, that the NHS would not be able to take them on. In addition, academia and the NHS don't have the necessary in-house skills to do this. The Big Pharma paradigm has ensured that most of these skills now reside in the private sector and are very expensive. The Pharma industry is one of the most high-tech industries around and is therefore a very expensive enterprise. Skills cost money! I suspect this is why developed countries, the UK included, are all trying to lure and retain viable pharma hubs within their borders. The sad and unfortunate thing is that the UK is not doing very well at this at the moment, with a large number of Pharma companies disinvesting."

Other related posts on this blog:

Discontinuation of licensed supplies of alemtuzumab or campath
20 Aug 2012
This means that alemtuzumab will no longer be available as a licensed product in the UK once existing supplies run out. This action is not being taken for any reasons related to product safety, efficacy or supply, but as part of ...
22 Aug 2012
Alemtuzumab remains in the news. Sanofi pulls Campath to clear way for higher-priced Lemtrada FiercePharma. Genzyme has been developing Campath-slash-Lemtrada for MS, hoping to become a big player in that disease ...
27 Aug 2012
Cambridge, MA – August 27, 2012 – Genzyme has announced that it has received a "Refuse to File" letter from the U.S. Food and Drug Administration (FDA) in response to the supplemental Biologics License Application ...
21 Aug 2012
Alemtuzumab is being pulled in the US as well. (Reuters) - Sanofi's rare disease unit Genzyme is pulling leukaemia drug Campath to prepare for its launch under a different dosage and as a multiple sclerosis treatment that ...

01 Nov 2012
Maybe more risks with alemtuzumab will emerge later, but the mayor risks with tysabri are well-documented- PML if you're JCVe+, and if you decide to come off it IRIS, and now these recent cases of fulminant MS in MSers ...
01 Nov 2012
Alemtuzumab for patients with relapsing multiple sclerosis after disease-modifying therapy: a randomised controlled phase 3 trial. The Lancet, Early Online Publication, 1 November 2012 doi:10.1016/S0140-6736(12)61768-1.
28 Oct 2012
"The aim of this presentation was to present the phase 3 trial data on Alemtuzumab in early RRMS as a therapeutic strategy that probably works by depleting circulating lymphocytes. The slides may be a little complicated as ...

28 Aug 2012
The graph below compares the search volume index of alemtuzumab to CCSVI. To make the graphs readable I had to do a log conversion of the search volumes, which are given by week. To do the log conversion I simply ...
05 Sep 2012
"Yesterday's post on the withdrawal of alemtuzumab to prevent off-license use of the oncology version of alemtuzumab (Mabcampath) resulted in a flurry of discussion and criticism. With some of the latter occurring off-line.
21 Aug 2012
"Yesterday's post on the withdrawal of alemtuzumab to prevent off-license use of the oncology version of alemtuzumab (Mabcampath) resulted in a flurry of discussion and criticism. With some of the latter occurring off-line.
28 Aug 2012
Genzyme must resubmit Lemtrada application to FDA Boston Herald Sanofi subsidiary, Genzyme Corp. of Cambridge, said today it has received a refuse to file letter from the U.S. Food and Drug Administration for approval of ...

CoI: multiple