UK Buyers Club To Get Lower Cost Versions Of Vertex’s CF Drugs
Executive Summary
A buyers club in the UK is planning to import generic versions of Vertex’s cystic fibrosis drugs that are not available on the NHS. Some commentators think such clubs may become more common.
The Cystic Fibrosis Buyers Club, set up in the UK by parents of children with the condition, is hoping to help some patients import generic versions of Vertex’s cystic fibrosis drug Orkambi (ivacaftor/lumacaftor) from Latin America. Health advocates hope the move could lead to a compulsory license for Orkambi and believe that buyers clubs could be used more frequently in response to medicine prices that prohibit access to medicines.
Three years after UK health technology assessment body NICE initially rejected Orkambi, claiming that £105,000 was too high a price to justify the drug’s benefits, there is still no agreement on how to make the product available to patients via the National Health Service. Talks between NHS England and Vertex have dragged on, with the company seemingly reluctant to accept an “improved” offer from NHS England. (Also see "NHS England Offers ‘Improved’ Orkambi Deal, But Vertex Lukewarm" - Pink Sheet, 24 May, 2019.). On the table have been not just Orkambi, but also Vertex’s Symkevi (tezacaftor/ivacaftor) and Kalydeco (ivacaftor).
For some patients, the CF Buyers club may offer hope by negotiating on behalf of its members a lower price from overseas manufacturers of generic versions of ivacaftor/lumacaftor. The generic versions are unlicensed in the UK.
The CF Buyers Club emphasizes on its website that though it aims to negotiate a better price, patients will purchase products directly from the manufacturer and not the club itself. The overseas manufacturer will also deliver medicines to the patient’s door.
The more members the club has, the better chance it has of securing a lower price. “A Buyers club has negotiating power based on numbers so please spread the word – the more buyers, the lower the price available for everyone,” it said in a blog post.
According to the club, one supplier is currently able to provide ivacftor for six-year olds and older and ivacaftor-lumacftor for ages 12 and over. The company expects regulatory approval of ivacaftor-lumacftor for patients aged six to 11 year old in September, said the club. It is also developing a generic version of tezacftor and hopes to launch a version of Symkevi in the first half of 2020.
The CF Buyers Club has negotiated a sliding price scale that will see the price fall if more people buy through the club. Prices range from £18,000 to £23,000 per patient per year. Media reports have revealed the company to be Gador, which is based in Argentina where Vertex has not secured patent products.
How It Is Possible
According to the UK’s drugs regulator, the MHRA, the move is possible because personal importation is exempt from licensing requirements. “There is no legal objection to patients importing drugs – licensed or unlicensed into the UK,” it told the Pink Sheet. Onward sale and supply is prohibited, however.
To qualify for personal use, the medicine must be for the individual seeking the product or for someone in that person’s immediate family or household, the MHRA said. Supplies of up to three months are permitted in these circumstances, though the regulator warns that customs authorities may step in when large quantities of the product are involved or if there are suspicions that the product is not being bought for personal use.
There is no requirement for individuals to notify the MHRA of any imports of medicines for personal use; the agency therefore does not issue any form of authorization, licence or certificate in such instances. However, it also said that “any buying group” that supplies an individual patient will require an appropriate licence from the MHRA.
The MHRA also warns patients that it cannot be certain about the safety, quality or efficacy of drugs that do not have marketing authorization in the UK. “Our advice is always that licenced medicines should be used over unlicensed ones.”
The club reassures patients that the drug is safe. It says that the company already supplies other medicines to the EU and the US and its labs have been inspected by the US Food and Drug Administration and other stringent authorities. In addition, the company has a World Health Organization certification for good manufacturing practice, according to the club.
Compulsory License?
According to the lawyer and public health activist Ellen ‘t Hoen, the club faces few obstacles in terms of the rules and regulations governing personal use exemptions. However, she added that the procurement would still be on a small sale. The solution, she said, was for the government to issue a crown use license to allow the NHS to procure on a bigger scale from generics suppliers.
Campaigners have indeed called for the UK government to issue a crown use license to allow generic imports or local production, but the government has so far dismissed the idea, in part because of a lack of generic alternatives. But according to ‘t Hoen, the club has debunked this. “Now we know that not to be true. Therefore, there is no reason for the UK government to not make Crown Use and allow generic CF treatments on a large scale and pay for it through the NHS at a price much lower than Vertex demands from the NHS,” she said.
The CF Buyers Club recognizes that even generic versions of the drugs will be unaffordable for most and has also called for a crown use license. It also suggests that through research exemptions, the generics could be used in a large-scale clinical trial investigating the wider impact of the product on patients and carers.
The club is unlikely to prove a sustainable solution to the access issue, said Jaume Vidal, senior policy advisor at Health Action International, an NGO that advocates for access to affordable medicines. However, it could prove “a turning point in making the government feel the pressure of public opinion to fulfil their duty to protect and promote public health.”
Buyers Clubs A Fixture?
Famously buyers clubs in the 1980s helped patients access HIV drugs. But more recently, such clubs have helped provide access to versions of expensive curative hepatis C drugs, including Gilead’s Sovaldi (sofosbuvir). Clubs have also helped patients secure access to Truvada for PReP for preventing HIV/AIDS.
Some commentators believe that buyers clubs will become more common in the future as high medicine prices continue to prohibit access. “When a medicine is essential and available elsewhere people will explore ways to gain access to it,” said ‘t Hoen.
“Given the high prices of patented medicines, it seems more than likely that we will see this again,” Vidal told the Pink Sheet.
Vidal described the CF Buyers club as a “sad indictment of a broken system that relies on market exclusivity and ends up charging excessive prices that [forces parents] to take these measures.” He criticized governments for failing to take opportunities to tackle excessive pricing. Vidal pointed to the UK’s refusal to back a resolution on making medicine prices more transparent at the recent World Health Assembly. (Also see "More Drug Price Disclosure In Store As WHO Delegates Pass Landmark Resolution" - Pink Sheet, 29 May, 2019.) .
Vertex Response
‘T Hoen believes that Vertex will take some sort of action, possibly through applying pressure on patients and the government. The company may threaten not to bring its innovations to the UK market, or it may threaten not to support research in the country. It could even seek support from the US government to exert pressure.
Vertex declined to comment on whether it would take any action. However, in a statement it said it remained in ongoing discussions with NHS England. It also said it would continue to provide free access to the most seriously ill patients. Already more than 800 such patients in England have benefited from this program.
The company has spent 20 years and billions of dollars on research and development to “do what was once thought impossible – developing medicines that treat the underlying cause of CF. Hundreds of Vertex scientists have designed, created and tested over 1 million unique molecules – each time involving painstaking work – to discover and develop treatments for people living with CF,” Vertex said.
“Companies who claim to be able to produce a product similar to Orkambi have not had to bear the cost of drug discovery and development and would be compromising intellectual property covering this medicine,” it said.