Value of early access to new medicines questioned by NCPE

The head of the committee that recommends to the Health Service Executive whether new drugs should be approved or not has said he is opposed to early access for medicines, like those for rare diseases, if that means they're not assessed first.

Director of the National Centre for Pharmacoeconomics (NCPE) Professor Michael Barry told RTÉs This Week: "I would argue that because these drugs are very high cost, because they come with a very significant budget impact, they should be assessed for value for money."

Families of those affected by rare diseases have repeatedly called on the Government to speed-up access to new medicines, and thereby prevent degenerative conditions from worsening, however Prof Barry questioned both the efficacy of such a move and the associated cost.

"These drugs are very expensive," he said. "You'll be paying more, because you won't have a negotiated price reduction. You will have a very high budget impact. And, unfortunately, the reality is many of these drugs don't work very well."

Asked to clarify, Prof Barry referred to assessments of drugs for rare illnesses so far this year: "None of them improved overall survival. And we got an improvement in quality of life of 16%. When it comes to cancer drugs ... less than 50% will improve overall survival ."

"Early access to drugs, that don't work very well, is not a very good way of spending taxpayer's money in my view."

Prof Barry said the cost of such a policy could have a significant impact on the health budget, given introducing such drugs would have a cumulative effect.

"I'd estimate that you would add €1 billion to the drugs budget in approximately five years. You would add €2 bn to the drugs budget in eight years. So big financial implications for introducing early access."

He said introducing early access for cancer drugs would be "even more challenging."

"You'll increase the drugs budget by €1bn by year three,€2bn by year four, and €3.5bn by year five. In other words, you have nearly doubled [the costs]."

Prof Barry said his understanding of what early access meant was "... when a pricing and reimbursement application is made, the drug will be made available immediately.

"Early access for all drugs I would not support. But early access for drugs that work, in others words have a proven over-all survival benefit, you might be able to justify that.

"In any event, I don't see it happening because I don't see that the resources are there to support it."

Prof Barry said that while speeding-up access to drugs was possible, it was important to recognise that "... the majority of the delay that we see is due to the pharmaceutical industry, and that is not what you will hear commonly."

He cited data regarding drugs for rare diseases, which had taken 871 days to reach a determination, but pointed out that 706 of those days related to the pharma-companies and only 151 regarding the assessment of the drug.

A comprehensive review of how Ireland approves access to new medicines, and the knock-on impact on the Exchequer, should be completed by the end of the year, according to Minister for Health Jennifer Carroll MacNeill.

Prof Barry said the heavy lifting needs to come from the pharmaceutical companies: "It's up to the pharmaceutical industry to present their drugs sooner; to be more agile in the assessment process; and they'll be more than matched by us."

"I don't have any problems with a review. And in fact it may be positive because, at last, we'll see where the true figures are and we'll see where the delays are as well."

Asked about Mounjaro, the new drug focused on helping people who are overweight or have diabetes, Prof Barry said: "My understanding is that a significant price reduction has been offered. I suspect that we will be saying the drug is cost effective, or very close to it."

He said this will pose a significant challenge to the Government because the drug would likely deliver "a whole lot of positive outcomes for many, many people" but the cost would be substantial.

"Depending on the price reduction, you could still be talking about €2bn-€3bn, over five years, expenditure."