Scrip: industry news and insights
18 Sep 2020
A composite endpoint conceals Actemra’s lack of mortality benefit – while life-saving dexamethasone gets EMA endorsement.
A UK parliamentary committee has criticized the pharmaceutical industry for not putting medicinal cannabis products into clinical trials, saying that companies that fail to do so should be “named and shamed.”
“It is concerning that that some pharmaceutical companies are still resistant to making their products available for research,” says the House of Commons’ health select committee. Without a robust evidence base allowing patients and clinicians to weigh up any risks and benefits of medicinal cannabis products, they “will remain unlicensed for the many areas where patients wish to know if they could be effective.”
In a report published on 3 July, the committee urges the Department of Health and Social Care to investigate cases where companies are not providing their products, and to “set out a plan to incentivize industry to take a more active role in research itself.”
It also says that even though medicinal cannabis products were reclassified as Schedule 2 controlled drugs last year, allowing them to be prescribed by specialist doctors under specific conditions, “very few prescriptions have been issued for such products,” because most of them do not have a UK or EU marketing authorization, nor have they been OKd by the health technology assessment (HTA) body, NICE.
Noting that medicinal cannabis has been used to try to treat conditions such as chronic pain, spasticity in multiple sclerosis, chemotherapy induced nausea and vomiting, and intractable epilepsy, the committee says that what is needed now is “a sense of urgency to explore the potential of medicinal cannabis in these conditions so that there is a robust research base on which to base future clinical decisions.”
It is not clear exactly which particular companies the committee is referring to, as no names are given in the report. It is true that for now, major drug makers are largely expected to sit on the sidelines in this area, although as our sister publication In Vivo reported recently, in the US companies of various sizes are looking to take advantage of the potential of medicinal cannabis. (Also see "Understanding The Evolving Medical Cannabis Market " - In Vivo, 17 Jun, 2019.)
Professor David Nutt, a neuropsychopharmacologist and former drugs advisor to the UK government, was sceptical of the committee’s conclusions about the role of pharmaceutical companies in the medicinal cannabis.
“I don’t think they understand what they mean by the pharmaceutical industry,” Nutt told the Scrip. “The industry has had 40 years to do this, and it hasn’t done it.” He said the only drugs it had produced in 40 years were GW Pharmaceuticals PLC’ Sativex (nabiximols), and synthetic cannabinoids like nabilone. “So at what point do you say, let’s give up and try something different?”
The traditional pharmaceutical industry “isn’t going to do it,” Nutt said. Companies specializing in cannabis products generally are not big enough, and “they are very worried that whatever comes out doesn’t get approved by NICE.”
“There are too many gaps in the evidence to allow most forms of medicinal cannabis to be licensed for use and approved by NICE” – Sarah Wollaston
The health committee’s report is the result of an inquiry into the lack of research into medicinal cannabis products, the paucity of products available, and the low numbers of patients being treated.
The committee’s chair, Sarah Wollaston, said there were “too many gaps in the evidence to allow most forms of medicinal cannabis to be licensed for use and approved by NICE.”
And while last year’s rescheduling decision was to be welcomed, there was “a failure to communicate what this would mean in practice for the availability of medicinal cannabis, Wollaston said. "Expectations were unfairly raised that these products would become widely and readily available, and there needs to be far clearer communication that this is not the case.”
While the committee has welcomed calls by the UK National Institute for Health Research for research proposals in this area, it said it was “important for industry to be more involved in developing medical cannabis products” and supplying them for research. “There is potential medicinal benefit to cannabis-based products but the gaps in the research base mean that we do not know where this sits alongside other therapies,” the report says.
At present, the only medicinal cannabis product with a UK marketing authorization is Sativex, which has been approved for use in spasticity in multiple sclerosis since 2010. GW Pharmaceuticals has also developed the oral cannabidiol drug Epidiolex, but while this has a US approval for Dravet and Lennox-Gastaut syndromes, it is still under regulatory review via the the EU centralized procedure (as Epidyolex). (Also see "Romosozumab Among Latest Drugs Up For CHMP Opinion" - Pink Sheet, 25 Jun, 2019.)
Two synthetic cannabinoids are available in the UK: nabilone, which is licensed for use in treatment-resistant nausea and vomiting caused by chemotherapy, and dronabinol, a synthetic version of tetrahydrocannabinol that is listed under Schedule 2 but does not have a UK marketing authorization.
According to the committee, one of the key barriers to research into new drugs is that the pharmaceutical industry is not always prepared to supply products for clinical trials to clinicians or organizations proposing to carry out those trials.
“We heard throughout our inquiry that some pharmaceutical companies were not willing to provide their product for trial,” the report says, noting that one witness to the inquiry, Professor Helen Cross, related her “experience of being in discussion with one particular company where it has taken six months to persuade them to supply, and the company have been known to pull out of trials early.”
Cross, who is head of developmental neurosciences at the UCL GOS Institute of Child Health, said there were “one or two companies that are discussing with us about doing the trials, but they cost a lot of money, and are they going to get their licence at the end? If it is going to be prescribed anyway, they may or may not need to.”
“Companies may also not want to carry out research because randomised controlled trials can be expensive to carry out" – Commons health committee
“We appreciate that it is not all pharmaceutical companies who bring this attitude, but it is unacceptable that some are behaving this way,” the report says, adding that “industry’s lack of engagement is one of the reasons why there is a lack of robust international evidence.”
It says companies might not want to carry out the research themselves partly because of the difficultly of obtaining a patent on a cannabis-based product, especially if others might copy those products. “Companies may also not want to carry out research because randomised controlled trials can be expensive to carry out,” it declares.
Nutt agreed with this asssessment. “If the cost of doing trials is so expensive that the price of the drug has to be high to recoup investment costs, NICE won’t approve it,” he said. Nutt chairs the medical charity Drug Science, which is planning a national UK medicinal cannabis pilot, Project TWENTY21, which aims to enrol 20,000 patients using a real world registry to assess the safety, efficacy and cost effectiveness of a number of cannabis-based products (this will be covered in forthcoming Pink Sheet article).
The committee recommends that the DHSC should investigate those instances where pharmaceutical companies do not provide their medicinal cannabis product for research and should “take appropriate action where necessary.”
The department “should not be afraid to ‘name and shame’ companies who are not doing all they can to make their products available for research” and should also set out a plan to “encourage industry to take a more active role in research itself and should present this plan in response to this report.”
Another problem identified by the committee is the lack of a domestic supply of unlicensed medicinal cannabis products for specialists to prescribe. Baroness Blackwood said “there is no UK supply. That is something we are working with industry to try to address, because the import cost is significant. That is another issue that we would like to encourage industry to help us to address.”
High costs, the report says, makes it more difficult for local prescribing committees to approve prescribing applications. It recommends that the department should take steps to secure long-term international deals to ensure a consistent supply of products “so as to ensure that patients are not delayed in receiving their prescriptions and the cost of the medicinal cannabis products are kept as low as possible.”
Baroness Blackwood said the department was “working with industry on establishing supply.” The committee says it welcomes this work and recommended that the department should also work with governments abroad “to make a more collaborative and attractive deal for industry. We expect to hear from the Department what success it has had in this area by the beginning of 2020.”
The report also notes what while Sativex is licensed for MS spasticity, NICE has said that the product is not cost effective, which means many patients can only access it through a private prescription – or not at all.
NICE is, though, working on guidance on medicinal cannabis products and expects to publish a draft in October this year. The report says that the HTA body should “take account of patient voices in its creation of guidelines for medicinal cannabis by allowing patient groups the opportunity to comment on the draft guidelines and receive a response to those comments from NICE.”
It adds that the case for cost-effectiveness could be improved if more research was carried out to demonstrate efficacy. “This is another illustration of why robust evidence is so important.”
Scrip has asked the Association of the British Pharmaceutical Industry for a comment.
Scrip: industry news and insights
18 Sep 2020
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