This is a terrible precedent... Many 'positive' RCT's suffer shortcomings in design, execution and interpretation. The low bar set by FDA and EMA nonetheless leads to approvals of drugs lacking any real proof of benefit. Forcing a local HTA to reevaluate a carefully made negative decision of this trial (see also Meirson et al Lancet Oncol. 2023 Jun;24(6):589-593.) is just worrying. This will ultimately only harm breast cancer patients and society. I fear more cases (for access to other non-effective, interventions lacking proof) will follow...
Biggest issue IMO not the drug companies. Many of the contemporary oncology trials cannot answer the question they set out to study properly because of design issues (poor control arms, inappropriate (lack of) cross-over among many other things). But 'we' allow these to be run. 'We' should not be entering patients into such trials, we should not participate. KOLs should not advocate for drugs lacking benefit, we should not take money from Pharma as personal payments, we should advocate for ethical trials studying appropriate clinical endpoints,... We should raise the bar!
Pharma invented & bought the very term, Key Opinion Leader (KOL).
It is the ambition of top academics to be so classified. I knew two KOLs in my domain & several other wannabes. The least fussy accumulated over £1M per year as a professor at a London university, and this was back in the noughties. He was very clever & he worked harder and for longer than anyone I’ve ever known. He looked a decade plus older than his years right from the beginning, and retired only recently. I always had the impression he was biased equally in favour of his pharma clients, to whom he was arguably the top KOL or #2 to the other Professor I mentioned. I think he took from them all, while secretly despising the lot of them. He was very good at fund raising for his labs and brought on several very good researchers. Unsurprisingly, they all developed with similar characteristics. In the covid era, the one I knew best & who could have helped materially in 2020 (a professor of immunology) simply went radio silent on me. We’d been friends since university & are friends no more.
Virtually nobody has clean hands. Me? I was too asleep to realise the extraordinary extent of corruption all around me in pharma, in academia and in the funding bodies (national research councils and NGOs). If I had realised it, I’d have resigned. The entire system is broken beyond fixing. The best an alert patient can do is research everything & trust no one.
I am a PhD, not a medic & oncology is not my field.
I’ll tell you this, though. I would not be bothering with “chemo” if ever I develop cancer. The “medicine” at the centre of this debacle has such a skinny benefit, if any at all, that it is unlikely a physician would be able honestly to say to an individual that “You are likely to receive meaningful, real world clinical benefits, outweighing likely unwanted effects”. Yet, if it ends up being approved in The Netherlands, specialists may have no choice but to prescribe it, because “treatment guidelines” will, in time, include it. That process, re-evaluating treatment guidelines, is the single best remunerated gig a KOL can get, informally of course, because the commercial implications of a company’s drug into the treatment guidelines as early as possible in the course of disease can be huge.
Nobody asks the taxpayer either, do we as a society agree to spend whatever it is, an incremental million Euros per patient, for a potential benefit so small that this argument arises on the method for counting the angels on the head of a pin? As spend per head of population rises, so the society grows sicker.
It all needs to come down. Regrettably that will happen only when everything comes down. It’s a sad situation. There’s corruption everywhere that there’s money, with no exceptions. Was it always so? I don’t know but I’m not well placed to know.
Yes, pharma development comes with risk, as well as potentially enormous profits of course, which Pharma, not the public, should take full responsibility for. Looks like the Dutch government has been captured, or has shot itself in the foot.
Not sure I see a problem with this. If the reason for it to be turned down is based upon a new, stricter guideline that went into effect last year, then how is this different from moving the goal posts against Eli Lily? How can any company develop any product if just before approval different criteria are set?
Great insights and coverage, thank you Sahar van Waalwilk !
This is a terrible precedent... Many 'positive' RCT's suffer shortcomings in design, execution and interpretation. The low bar set by FDA and EMA nonetheless leads to approvals of drugs lacking any real proof of benefit. Forcing a local HTA to reevaluate a carefully made negative decision of this trial (see also Meirson et al Lancet Oncol. 2023 Jun;24(6):589-593.) is just worrying. This will ultimately only harm breast cancer patients and society. I fear more cases (for access to other non-effective, interventions lacking proof) will follow...
OH MY GOD.
My exact thoughts…
This is not remotely surprising. Eli Lilly is practically a TRILLION dollar company. Would you expect anything less? sabrinalabow.substack.com
Terminally corrupt system here and abroad
Unfortunately drug companies rule ….
Biggest issue IMO not the drug companies. Many of the contemporary oncology trials cannot answer the question they set out to study properly because of design issues (poor control arms, inappropriate (lack of) cross-over among many other things). But 'we' allow these to be run. 'We' should not be entering patients into such trials, we should not participate. KOLs should not advocate for drugs lacking benefit, we should not take money from Pharma as personal payments, we should advocate for ethical trials studying appropriate clinical endpoints,... We should raise the bar!
Pharma invented & bought the very term, Key Opinion Leader (KOL).
It is the ambition of top academics to be so classified. I knew two KOLs in my domain & several other wannabes. The least fussy accumulated over £1M per year as a professor at a London university, and this was back in the noughties. He was very clever & he worked harder and for longer than anyone I’ve ever known. He looked a decade plus older than his years right from the beginning, and retired only recently. I always had the impression he was biased equally in favour of his pharma clients, to whom he was arguably the top KOL or #2 to the other Professor I mentioned. I think he took from them all, while secretly despising the lot of them. He was very good at fund raising for his labs and brought on several very good researchers. Unsurprisingly, they all developed with similar characteristics. In the covid era, the one I knew best & who could have helped materially in 2020 (a professor of immunology) simply went radio silent on me. We’d been friends since university & are friends no more.
Virtually nobody has clean hands. Me? I was too asleep to realise the extraordinary extent of corruption all around me in pharma, in academia and in the funding bodies (national research councils and NGOs). If I had realised it, I’d have resigned. The entire system is broken beyond fixing. The best an alert patient can do is research everything & trust no one.
I am a PhD, not a medic & oncology is not my field.
I’ll tell you this, though. I would not be bothering with “chemo” if ever I develop cancer. The “medicine” at the centre of this debacle has such a skinny benefit, if any at all, that it is unlikely a physician would be able honestly to say to an individual that “You are likely to receive meaningful, real world clinical benefits, outweighing likely unwanted effects”. Yet, if it ends up being approved in The Netherlands, specialists may have no choice but to prescribe it, because “treatment guidelines” will, in time, include it. That process, re-evaluating treatment guidelines, is the single best remunerated gig a KOL can get, informally of course, because the commercial implications of a company’s drug into the treatment guidelines as early as possible in the course of disease can be huge.
Nobody asks the taxpayer either, do we as a society agree to spend whatever it is, an incremental million Euros per patient, for a potential benefit so small that this argument arises on the method for counting the angels on the head of a pin? As spend per head of population rises, so the society grows sicker.
It all needs to come down. Regrettably that will happen only when everything comes down. It’s a sad situation. There’s corruption everywhere that there’s money, with no exceptions. Was it always so? I don’t know but I’m not well placed to know.
Yes, pharma development comes with risk, as well as potentially enormous profits of course, which Pharma, not the public, should take full responsibility for. Looks like the Dutch government has been captured, or has shot itself in the foot.
The Netherlands are anti-humans. Progressive leftism is in full bloom.
Worrisome.
Not sure I see a problem with this. If the reason for it to be turned down is based upon a new, stricter guideline that went into effect last year, then how is this different from moving the goal posts against Eli Lily? How can any company develop any product if just before approval different criteria are set?