By John Thynne
Producer, BBC Panorama
We have heard much of the hype surrounding the use of Herceptin for early stage breast cancer but very little has been heard from scientists questioning just how effective the drug is.
Sunday 5 February 2006
22:15 GMT, BBC One
Join the interactive debate after the programme on BBC News 24 and online at
The strongest indication that Herceptin was going to be more than just another drug came in May 2005 at the American Society of Clinical Oncology (Asco) conference in Florida.
This annual meeting of Asco attracts the top scientists in the world in this field of medicine and provides an opportunity to present the latest advances in the treatment of cancer.
When the results of the Herceptin Adjuvant trial (Hera), a major international study on the use of Herceptin in early stage breast cancer, were announced, the audience of scientists gasped in astonishment.
What the trial data reported was that Herceptin could reduce the risk of recurrence in suitable patients by as much as 52%.
This was not a marginal advance; rather, in the words of Professor Ian Smith, the lead investigator in the trials in the UK: "This is the biggest treatment development in breast cancer, in terms of the magnitude of its effect, for at least the last 25 years, perhaps as big an anything we've seen."
This early enthusiasm for the drug was quickly picked up by the media who fanned the flames of publicity by labelling it a "wonder drug".
Stories quickly followed of women who had been denied funding for the drug and who were forced to contemplate selling their houses to pay for it.
Politicians followed where their constituents led.
The government's position swiftly moved from allowing the normal procedures for funding drugs on the NHS to follow their usual course, to ensuring the process was fast-tracked and finally advising local health trusts that they should not deny the drug to patients on the grounds of cost alone.
Hype over hope
It was at this point that the first indication came from some in the medical world that the hype may have overtaken the hope.
The Lancet, Britain's long-established and highly-regarded medical journal, questioned just how much we really knew about Herceptin.
What really mattered was how many lives the drug saved
An editorial raised concerns about the amount of data that existed and what it revealed, about the way the trials had been stopped early, and, most importantly, about the safety of the drug.
It concluded by saying: "The best that can be said about Herceptin's efficacy and safety is that the available evidence is insufficient to make reliable judgements."
Outside Britain, others had begun to question the use of statistics.
While the trials revealed that the risk of recurrence of breast cancer within one year was reduced by an impressive 52%, this gave, it was claimed, a rather skewed impression of the drug's efficacy.
What really mattered was how many lives the drug saved.
This survival data, which is normally collected over at least five years, showed no statistically significant improvement at the one year stage.
Professor Ian Smith has since conceded that on the key question of survival, the proven benefit of the drug is currently only marginal.
Re-analysing the figures
Earlier this year, the clinical director of the National Institute for Clinical Excellence (Nice) joined the debate over statistics in an article for Lancet Oncology.
Re-analysing the data from the Hera trial, the article revealed that you would have to treat 18 patients to save one life.
It stated: "For every 100 suitable patients prescribed trastuzumab (Herceptin), 94 will have been exposed to the side effects without any benefit, at a cost of £400,000 per recurrence prevented."
Cost-effectiveness, of course, lies at the heart of the debate.
If the drug were proven to be highly cost-effective, there would be less concern about the issue of funding it.
The job of evaluating new treatments' cost-effectiveness (and hence whether or not they can be provided on the NHS) is performed in England by Nice and it can only act once a drug is licensed for use.
Licensing, a vital procedure to ensure the safety of drugs, is performed by the European Agency for the Evaluation of Medicinal Products (EMEA), and this body in turn can only act once the manufacturer of a drug submits an application for a licence.
And herein lies the problem: Roche, who manufacture the drug, have not yet submitted an application for a licence.
Revolutionising cancer treatment
The remarkable rise of Herceptin, from relative obscurity to must-have 'wonder drug' in the space of a few months, points to a larger problem looming for the NHS.
As drugs become increasingly focussed on smaller patient populations, their cost will inevitably rise
Herceptin is merely the most prominent of a new generation of drugs, called monoclonal antibodies, which are going to revolutionise the treatment of cancer.
These drugs work more efficiently than traditional treatments because they target the individual cancers they are treating far more effectively.
But they will not work for everybody.
Herceptin, for example, only works with a particular form of breast cancer that affects about a quarter of all breast cancer patients.
As drugs become increasingly focussed on smaller patient populations, their cost will inevitably rise, and the cost for the nation for all such drugs could be overwhelming.
The bill for Herceptin alone has been put at £100m per year, or about a quarter of the annual cancer drug bill for the whole country.
And this is just for one drug, for one particular type of cancer.
Panorama's "Wanting the Wonder Drug" will be broadcast on Sunday, 5 February, 2006 at 22:15 GMT on BBC One and online at bbc.co.uk/panorama, where it is also available on demand following the initial transmission.
And have your say in a special interactive programme on BBC News 24 at 23.00GMT on Sunday immediately after Panorama.
Reporter: Betsan Powys
Producer: John Thynne
Assistant Producers: Patrick Barrie
Deputy Editors: Andrew Bell, Frank Simmonds
Editor: Mike Robinson