The failure to set up a separate evaluation process for orphan drugs and the lack of opportunity for patient engagement when considering their reimbursement has been criticised by politicians from all parties.
The criticism aimed at the HSE at yesterday’s Joint Oireachtas Committee on Health comes at a time when the State has either refused or has yet to decide on reimbursement of a number of orphan drugs.
The therapies — to treat extremely rare conditions — include genetic emphysema drug Respreeza; Kuvan for treatment of rare metabolic disease PKU; and Translarna, for Duchenne muscular dystrophy (progressive muscle degeneration).
Respreeza should be available to 21 patients who took part in a clinical trial with CSL Behring but a row between the drug company and the HSE over who should administer it means they are currently not getting it. This is contrary to the claim by the National Centre for Pharmacoeconomics (NCPE), in its opening statement to the committee, that Respreeza was available to patients under a compassionate access programme.
In relation to Translarna, where reimbursement was refused — a decision currently under appeal by the drug company PTC Therapeutics in the High Court — Sinn Féin senator Rose Conway Walsh asked “would it not be possible to grant access on a conditional basis” to the two little boys in the State who are looking for it.
Shaun Flanagan, chief pharmacist in the HSE’s Corporate Pharmaceutical Unit, said they were heading into “ropey” ground by discussing a case before the courts.
Fianna Fáil TD John Brassil questioned why, of 148 orphan drugs, only 53 were reimbursed in Ireland compared to 68 in the UK and 84 in France. The figures were disputed by John Hennessy, HSE primary care chief.
“We are not performing as we should be,” said Mr Brassil. “If the system is not broken, it’s certainly not working as it should be.”
Mr Brassil said those tasked with deciding if drugs were worth reimbursing needed to switch from a “quantitative” to a “qualitative” approach. Patients with rare diseases were a “tiny cohort” and drug companies were never going to be able to supply the data justifying their reimbursement.
Mr Hennessy said: “I think what you are suggesting is a change of policy to make access to orphan drugs easier.”
He said existing legislation doesn’t make separate provision for orphan drugs, “so it’s an area the committee may wish to consider from a policy or legislative point of view”.
In response to requests for more structured involvement of patient groups in the reimbursement process, Mr Hennessy said: “We’d be open to exploring that.”
Fine Gael senator Colm Burke called for a special budget and a separate reimbursement process for orphan drugs. Sinn Féin health spokeswoman Louise O’Reilly said the current system has a “significant blind spot” when it comes to approving orphan medicines for rare diseases.
NCPE director Michael Barry said: “I don’t believe the process is broken.”
“It’s a fair, open, transparent, but rigorous process,” he said.
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