Like Asterix’ and Obelix’ tiny village in Roman-occupied Gaul, the pharmacists of the Amsterdam Academic Medical Centre fought the Italian pharmaceutical company Leadiant this year. Leadiant had raised the price for a drug called CDCA, which is used to treat a rare metabolic disorder called CTX, making it 500 times more expensive than it used to be: 140 dollar per pill, up from 28 cents per pill. In response to the price hike, the Amsterdam-based pharmacists decided to start making the drug themselves, which they were allowed to do as compounding pharmacists. Initially, the Dutch Health Care Inspectorate seemed to disallow the practice, but by now, the Amsterdam AMC has been granted permission to start compounding the drug itself again, subject to certain conditions. Was 2018 the year in which society finally genuinely revolted against Big Pharma?
Unethical
‘We have to go to prices that are fair’
“It has become very clear this year that we can’t go on this way,” says Carin Uyl-de Groots, a Professor of Healthcare Evaluation at the EUR. “Some new medications are practically unaffordable, even though patients are entitled to treatment. Maybe it’s legal for prices to be set in a particular way, but maximising your profits at the expense of patients is completely unethical. We have to go to prices that are fair”
Earlier this year, Uyl-de Groot published a study in Nature Reviews Clinical Oncology, which showed that the great majority of cancer drugs are too expensive. She created an algorithm that calculates the cost price of medications. “Take this ovarian cancer drug, for instance. Our algorithm says it should cost 15,000 euros. In actual fact, it costs 126,000 euros.” She is seeking to have the calculation method applied to the pharmaceutical industry. “To this end, I’m discussing the matter with the European Parliament, among other parties. Last year, the European Parliament passed a resolution requiring pharmaceutical companies to provide greater transparency on the prices they charge for medicines.”
Standing stronger together
Europe is where true breakthroughs must be brought about, Uyl-de Groot believes. “At present, countries are negotiating on an individual basis. As a result, they are not aware what kind of agreements pharmaceutical companies are entering into with other countries.” Moreover, the Netherlands does not have much of a say on its own, since it only constitutes 2 per cent of the European market. Uyl-de Groot explains that a large country like Germany is more interesting to companies, meaning it gets medications sooner and at a cheaper rate. “For this reason, I’m currently in talks with Beneluxai, a group established by five countries that wish to negotiate with pharmaceutical companies together: Belgium, the Netherlands, Luxembourg, Austria and Ireland. Together we will stand stronger.”
If Uyl-de Groot had her way, a European agency would be established that was in charge of the price of medications, like EMA, the European Medicines Agency, which evaluates applications for European marketing authorisation for medicines. She feels this would be in pharmaceutical companies’ interest, as well. “Their turnover would increase, because medicines will be marketed sooner and will be used by more patients due to their lower price point.” For instance, in the example of the aforementioned ovarian cancer drug, her algorithm predicts that the pharmaceutical company’s turnover would multiply five-fold (up to €700 million), despite the reduced selling price. Prof. Uyl-de Groot is at pains to emphasise that she is not at all against pharmaceutical companies. “We simply need those medicines.”
More effective and cheaper, too
Pauline Meurs, too, is in favour of countries jointly entering into agreements on the prices they are going to pay. Meurs, a Professor of Healthcare Management, hopes that the pressure engendered by such a coordinated action will make pharmaceutical companies more innovative. Pharmaceutical companies currently have a business model based on failure, or so Meurs recently told Bruno Bruins, the Minister for Health Care, in a recommendation issued by the Public Health and Society Council (RVS). Meurs chairs this independent advisory body that issues recommendations to the Ministry of Health, Welfare and Sport. “Only 10 per cent of all medications being tested end up being marketed. That is a ridiculously low percentage. It’s high time we looked at pharmaceutical companies’ cost efficiency.”
She feels that the success rate of pharmaceutical research should be able to be raised, and that prices will benefit from this. “Pharmaceutical companies really aren’t that innovative. They all conduct standard randomised controlled trials, to determine the efficacy of medications in large groups of patients.” Many potential medicines are not rejected until these expensive and labour-intensive studies, called ‘phase-3 studies’.
Therefore, Meurs and RVS are in favour of more efficient phase-2 studies, in which the efficacy of a drug and its potential side-effects are established. “If you examine not only middle-aged men, as is the case at the moment, but also women and the elderly, it is easier to assess whether a drug will survive the expensive phase-3 trials.”
Pharma bashing
‘Biopharmaceutical start-ups can act like a gadfly to major companies’
Meurs does see innovative methods being used at small biopharmaceutical start-ups. She thinks the Ministry of Economic Affairs should do more to support these small companies. “Let them act like a gadfly to major companies. Give them incentives to keep doing so, instead of telling them the market must be allowed to run its own course. It’s all about protecting the public interest: affordable medicines.”
Meurs’ proposal played right into the hands of Hans Büller, who served as the Chairman of Erasmus MC’s Board until 2013 and has been the Managing Director of FairMedicine for the last two years. This organisation seeks to develop medications at an acceptable price point, by creating coalitions between all stakeholders, i.e., knowledge institutions, doctors, patients and, yes, pharmaceutical companies. “I’m not in favour of ‘pharma bashing’. However, I do think it’s better if multiple parties market a medicine together, rather than having pharmaceutical companies dictate the whole process.”
Rare disorders
FairMedicine’s methods are as follows. If someone working at a knowledge institution discovers a potential medication, FairMedicine will seek partners that will help him or her develop and market the medicine. A coalition made up of knowledge institutions, pharmaceutical companies, doctors and patients will perform the clinical trials and set prices, based on actual costs, with a ‘socially acceptable’ profit margin not exceeding 15 per cent.
The former paediatrician believes FairMedicine’s model will prove attractive to universities’ pharmaceutical spin-offs. They, being more socially engaged than profit-hungry, tend to focus on rare disorders that major pharmaceutical companies do not think are worth their while. “They have a completely different attitude to the matter than the real ‘crooks’ whose only intention is to obtain a monopoly on certain medicines because they can then charge a hundred times as much.”
Medicines slowly becoming more affordable
‘This was a year in which it became abundantly obvious that things really have to change’
However, Büller knows that it is hard for such small companies to actually obtain marketing authorisation for interesting drugs. “The drugs must be evaluated in expensive clinical trials. And banks don’t give loans to such small companies that employ, say, twelve people. So then we’re looking at a buy-out by a larger company that can finance the trial. It’s even become a bit of a business model for these start-ups. But it does mean they are no longer in charge of the product and the price charged for it.”
FairMedicine hopes to market its first medicine in mid-2019. Büller does not yet wish to announce what kind of drug it is. It seems that medicines are slowly getting more affordable. Büller agrees with Meurs and Uyl-de Groot that ‘this was a year in which it became abundantly obvious that things really have to change’. But, he adds, “a lot of things will still have to change for us to get where we wish to be”.
