Compulsory License On Table In UK For Orkambi
Executive Summary
The UK government will consider all options to improve access to Vertex’s cystic fibrosis treatment, including compulsory licensing.
UK parliamentarians have again discussed how to get Vertex’s cystic Fibrosis drug Orkambi (ivacaftor/lumacaftor) to patients as talks between NHS England and the company stagger on with no conclusion. Seema Kennedy, the parliamentary under-secretary for health and social care, said the government would now start looking at all options on the table, including a compulsory license.
“The ideal thing is to get a deal, and deals have been done with other pharmaceutical companies,” Kennedy said. Echoing previous criticism of Vertex, she called the firm an “outlier” and accused it of refusing to accept the official health technology assessment process as conducted by NICE and of putting forward unacceptable demands. (Also see "‘Extreme Outlier’ Vertex Slammed For Orkambi Pricing Approach In England" - Pink Sheet, 8 Mar, 2019.)
“Unless Vertex changes its approach and behaves responsibly, I have a moral obligation to look at these other options. Of course NHS England and NICE will carry on the negotiations, because a negotiated outcome is the desired option. However, I have no alternative but to look at these other options on the table,” said Kennedy.
The parliamentary debate came earlier this week after another petition demanding access to Orkambi and other CF treatments amassed more than 100,000 signatures. In 2016, NICE first rejected Orkambi, saying it was too expensive to justify the benefits. Negotiations between NHS England and Vertex have been ongoing ever since.
“Unless Vertex changes its approach and behaves responsibly, I have a moral obligation to look at these other options.” - Seema Kennedy MP
Patients and their carers are becoming increasingly frustrated at the fact there is an effective treatment option that is not accessible. Last week, a buyers club appealed for CF patients to get in touch and explore the possibility of buying a generic version of Orkambi from Argentina’s Gador. (Also see "UK Buyers Club To Get Lower Cost Versions Of Vertex’s CF Drugs " - Pink Sheet, 6 Jun, 2019.)
Vertex again came under heavy fire at Monday’s debate from members of parliament, several of whom demanded that the government pursue a compulsory license. Earlier this year MPs debated whether issuing a crown use license could prove a reasonable solution to the impasses. The then under-secretary for health, Steve Brine, said this was not a viable solution because there was no generic version of Orkambi. (Also see "UK Has Sought Advice On Compulsory Licensing For Vertex’ Orkambi " - Pink Sheet, 5 Feb, 2019.)
At Monday’s debate, Labour MP Kerry McArthy said this was clearly not the case as a generic version was evidently available in Argentina. Fellow Labour MP Clive Lewis also called on the government to consider a compulsory license.
McArthy also suggested that the government should fund a large-scale trial of the generic version that would allow the NHS to circumvent intellectual property rights laws through exploiting research exemptions. “A trial could be set up in anticipation of a Crown use licence, providing data necessary to support regulatory approval of the generic version of Orkambi,” she said.
The generic could also be accessed by providing personalized health budgets that would help patients meet the cost of the drug, said McArthy.
There were words of caution from the floor about restricting a company’s intellectual property rights. Conservative MP Paul Scully warned that options such as compulsory licensing and a buyers club could hit innovation further down the line. “Yes, we need those weapons in our armoury for the negotiations, but there is always the possible unintended consequence of fallout—the risk for future research into rare diseases of all sorts,” Scully said.
Sarah Wollaston, chair of the House of Commons health select committee, which is investigating the lack of access to Orkambi, suggested a different approach. “Unfortunately, the gap is huge between what Vertex continues to demand for the drugs and what the NHS is offering based on recommendations from NICE,” Wollaston said. The solution could be similar to arrangements in Scotland, where an “interim price” has been agreed until further research is conducted on the impact of the drug.
Other MPs called for Vertex to be transparent in its pricing and to publish what it was offering. “The NHS has made its proposals public. That big pharmaceutical firm must do the same if it is to have any chance of having the public’s confidence that it is behaving in an ethical way. Failing to do so only helps to build the case against the continuation of the marketized pharmaceuticals industry in its current form,” said Labour MP Laura Smith.
Meanwhile, other MPs said the NICE system had to change to keep up with drug development.
Vertex said it welcomed the debate and that it shared “the urgency of the speakers to find a solution to access as soon as possible.” It said it remains committed to negotiations and that following a meeting on 7 June, its “intensive work continues on a daily basis.”
“In the absence of an access agreement, Vertex will continue to provide free medicines to patients who are the most seriously ill, based on objective clinical criteria, through our compassionate use programme. Since its inception, this programme has supported more than 800 of the sickest CF patients in England and over 1,000 patients across the UK.”