LONDON, 22 FEb (APM) - England’s health technology assessment (HTA) body NICE has been branded 'inflexible' when it comes to approving medicines for rare diseases in a report calling for it to be reformed, the Daily Mail said on Wednesday.
The report from MAP BioPharma found that 13% of rare disease medicines make it through single technology appraisal (STA) assessments, compared to 68% of medicines for common diseases.
The paper said that of the 24 completed STA reviews of rare medicines between 2013 and 2017, only six were recommended. In the same period, 50% of rare disease medicines were given a 'restricted recommendation', compared to 21% of other medicines.
It quoted Dr Jayne Spink, chief executive at Genetic Alliance UK, who said: “This report helps to show the systemic failings in our decision-making systems for access to rare disease treatments.”
North of England rivalling UK ‘golden triangle’ for life sciences
The north of England is establishing itself as a rival to the ‘golden triangle’ of London, Oxford and Cambridge for UK life sciences, the FT said at the weekend.
The paper spoke to several biotechs working in the area, including Evgen Pharma, RedX and Tissue Regenix.
Stephen Franklin, CEO of Evgen, told the paper that his company has benefited from AstraZeneca’s decision to leave Alderley Park in Cheshire and relocate to Cambridge.
He described AZN’s departure as a “real catalyst for the entrepreneurial sector because it just left so many talented people” who chose not to move south.
Groundwork must be laid for NHS to benefit from AI, says UK Health Minister Blackwood
The UK's Health Minister, Baroness Nicola Blackwood, authored an article in The Times on Wednesday pushing for the NHS to adopt artificial intelligence (AI), but warning the "ground rules" must first be established to gain critical public trust.
For this reason, the UK government is updating its code of conduct for data-driven health this week following extensive discussion with the NHS, patient groups, academics, charities, the data science industry and the National Data Guardian, Blackwood noted.
The code lays down 'gold standard' principles for suppliers of AI and data technologies to the NHS, and specifies that any partnership between the NHS and an AI company must result in clear benefits to the health system.
"There can be no more one-sided arrangements where a private company uses NHS research to build highly profitable products without any of the value flowing back to the NHS," Blackwood said.
The aim by 2030 is to use algorithms to detect the early indicators of disease "years before patients would even think of going to their GP with symptoms", she said. This should make drug discovery more efficient and affordable and help researchers find the best drug candidates for clinical trials, she added.
"But to get to that future we first have to lay down some ground rules. Any technology that runs on patient data requires public trust," she wrote in The Times.
"People have to be confident that their data will be used safely and responsibly, for the public good and not just private gain, that their rights and privacy will never be compromised. We also have to ensure that only the very best of this technology is supplied to the NHS, able to deliver real, quantifiable benefits to patients and clinicians."
Rushed used of AI in medicine may lead to inaccurate results
Meanwhile in AI, the FT at the weekend reported on comments from a leading U.S. computer scientist that its use in biomedical research is being rushed, leading to inaccurate findings.
Genevera Allen of Baylor College of Medicines and Rice University said: “I would not trust a very large fraction of the discoveries that are currently being made using machine learning technique applied to large data sets.”
“A lot of these techniques are designed to always make a prediction,” she told the annual meeting of the American Association for the Advancement of Science. “They never come back with ‘I Don’t know’ or ‘I didn’t discover anything’ because they aren’t made to”.
Orkambi stalemate ongoing in UK
The Daily Mail on Wednesday described a schoolboy as “living with a death sentence” because the NHS in England does not fund Vertex’s cystic fibrosis drug Orkambi.
The paper said NHS is in deadlock with U.S. pharmaceutical company Vertex for the drug, which has a list price of £104,000 per year per patient.
Vertex refused an offer from NHS England of £500 million for a five-year supply, the biggest offer it has ever made, and talks have completely broken down.
The paper focused on George Monckton, a child living with cystic fibrosis who would be eligible to receive the drug if it was funded.
His mother said: “Not having access to Orkambi fills me with fear, anger and a great sadness.
“It's like a veil over us, knowing CF can strike George and damage him at any time.”
The Guardian also reported on the continued deadlock, but from a different angle. On Tuesday the paper said that two UK directors of Vertex had made more than £15 million from share options in 2017.
Simon Lem and Simon Bedson (who has since retired), also received pay totalling £1.1 million in 2017.
Christina Walker, the mother of a child with CF, told the paper: “When we see the excesses of their executive remuneration, it really sticks in the throat that they say they can’t compromise on price, because their excuse is always that it will jeopardise future [research and development].”
Hedge fund Starboard Value attempts to join BMS board ahead of Celgene deal
Activist hedge fund Starboard Value is trying to force its way onto the board of Bristol-Myers Squibb ahead of its planned takeover of Celgene for $90 billion, the FT said on Wednesday.
The paper said that Starboard has nominated five people for election to the board after purchasing a million shares in the company. The hedge fund did not give a reason for the move, but the FT said that analysts speculate it would prefer BMS to put itself up for sale instead of making a large acquisition.
EMA loses battle to cancel lease for London HQ
The European Medicines Agency has lost a legal battle to get out of a £500 million rent bill for its London headquarters because of Brexit, The Times said on Wednesday (APMHE 61953
A High Court judge dismissed the agency’s claim that its 25-year lease of offices in Canary Wharf would be legally “frustrated” as a result of Brexit.
In a separate article, the legal editor of The Times said that the “battle” is not over and that the High Court granted the agency an extended period beyond the normal 21 days in which to appeal.
UK's Health and Social Care Select Committee chair Wollaston leaves Tory party over Brexit
Three "Europhile" MPs resigned from the governing Conservative party on Wednesday and pledged their allegiance to a new independent parliamentary group aiming to seize the centre ground of British politics, the FT reported on the same day.
Among the three was Dr Sarah Wollaston, the UK’s Health and Social Care Select Committee chair (APMHE 61955
), as well as two other Tory MPs, Heidi Allen and Anna Soubry, the FT and other papers reported, including The Guardian, The Times, The Telegraph and The Daily Mail.
The MPs said their resignation was over the party's shift to the right and Brexit policy.
They join eight Labour MPs who quit to form the party known as the Independent Group earlier in the week.
Immunomedics’ drug can change breast cancer treatment
Immunomedics’ antibody sacituzumab has the potential to change the way women are treated for one of the most deadly forms of breast cancer, said the Daily Mail on Wednesday.
The paper covered a clinical trial involving 108 women with triple-negative breast cancer that had spread around the body. The drug led to 'significant' tumour shrinkage, researchers reported.
The women had all previously failed to respond to two or more previous treatments. All had a poor prognosis.
Senior investigator Dr Kevin Kalinsky, from Columbia University, New York, said: 'I think this drug has the potential to change practice, because the data looks so compelling, even with the relatively small number of patients in the trial.”
AbbVie accused of abusing patent system in U.S. to block Humira competition
AbbVie’s Humira now costs patients five times more in the U.S. than in the UK following the launch of biosimilar competition in Europe, the Daily Mail said on Wednesday.
The paper accused AbbVie of abusing the U.S. patent system to block competitors and continue “jacking its price up”, while competition has been able to launch in Europe due to patent expirations.
A prescription for two shots of Humira now costs an average of $5,684 for a carton of two injections in the U.S. In the UK the same thing now sells for $934 after the launch of biosimilars forced prices to drop.
The paper said that Abbvie has filed 247 different patents for, effectively, the same drug, keeping a monopoly on it for 39 years. It added that for each 'new' patent, the company leaves the key components of its formula unchanged but makes “tiny tweaks” to clinically insignificant elements of the injection.