6 Mar 2018

The Oireachtas Committee on Health, a cross-party group of parliamentarians tasked with generating and scrutinising health policies and legislation, has published its report into the availability of orphan medicinal products in Ireland. The work is the culmination of several months’ analysis of the current landscape and engagement with the HSE, NCPE, patient groups and industry – prompted by the apparent growing frequency with which orphan drugs were not being recommended for reimbursement by the HSE. Orphan drugs, by their very nature, often treat severe and life-threatening illnesses for patients with otherwise unmet need, resulting in a highly difficult situation for patients, industry and politicians alike.

While the report is still hot off the press, we take a look at some of its major talking points and learnings, as well as its potential implications in the both rare and broader pricing and reimbursement space.

The Case for Change
First and foremost, the most obvious takeaway from this report is that the Committee is unhappy with the low level of access to orphan drugs in Ireland, particularly when these same medicines are increasingly available elsewhere in Europe.

The difference with other EU States, it appears to suggest, is that whilst others have refined and adapted their own approaches to overcoming the classic challenges of assessing a rare or end-of-life medicine against the traditional HTA, Ireland has yet to do so.

Some very practical steps are recommended in the report to identify the barriers and remove them, including calling for an independent review of the entire pricing and reimbursement process, a consideration as to whether a QALY-based HTA is appropriate at all for ultra-orphan medicines (going so far as to suggest a QALY assessment neglects to ensure the best outcome for patients), a re-negotiation between IPHA and the State to agree appropriate and timed steps for negotiation post-HTA, and a legal review of the Health (Pricing and Supply of Medical Goods) Act 2013 to ensure the law is not impeding reform.

A Philosophical Change?
The report takes three very symbolically significant stances that are in many ways, the core foundations for driving real change in medicines reimbursement.

  1. It acknowledges that access is unacceptably low and that Ireland will inevitably fall behind our EU counterparts in terms of outcomes;
  2. It acknowledges that patients have a right to the best possible diagnosis and care, “irrespective of the rarity of their condition”; and
  3. It acknowledges that policy-makers have an obligation to identify barriers to access and deliver workable solutions.

At first glance, this seems a fairly reasonable stance to take, however for some patients with rare diseases, this will come as a significant victory. The view that Ireland is behind the curve on access to orphan drugs has been disputed and denied by officials, who, in the absence of authoritative and quantifiable evidence, can be difficult to argue. Equally, the ‘population health’ concept is often broached when discussing improving access to medicines – the view that providing access to one patient with an ultra-rare condition will directly reduce physio hours or the availability of prosthetic limbs elsewhere. To take the view that the State must seek to provide access to patients – regardless of the rarity of their condition but on the grounds that it is simply the right thing to do – is certainly refreshing, as is the recommendation for a standalone orphan drug budget.

The Demand for Accountability
It is clear the Committee is unimpressed with the lack of improvement to access for orphan drugs arising out of the 2014-2018 National Plan for Rare Diseases, given the level of accountability tied to each of their recommendations. Many recommendations are timed, while one frankly demands the identification of who exactly within the HSE and Department of Health is responsible for improving access, as well as their summoning before the Committee on a recurring basis to provide tangible updates on their progress.

It equally demands that reforms to date are effective and not simply done out of lip service to political pressure. The timing of the announcement that a Chair had been appointed to the Rare Diseases Technology Review Group (a committee for qualitative input to a HTA, not unlike PACE in Scotland) may have come across as less-than-coincidental to Members of the Committee, for example. The Report, interestingly, recommends the information gleaned from patients and clinicians be guaranteed meaningful weighting against quantitative data – presumably in response to patient outcry that their voice is not being heard during the current process.

Don’t Hold your Breath for Europe
Cross-county collaboration may well be the future of assessment and reimbursement of high-tech medicines, with the recent draft proposals on an EU-wide HTA and Ireland’s attempts to join BeNeLuxA presenting a clear indication that the Government views this as the solution.

Those of you reading the text to the Commission’s proposal or tracking joint collaborations across Europe know, however, that these do not come about quickly (it could be over six years before a set HTA is adopted). The major risk in Ireland therefore would be that policy-makers would leave the current process as is, in the expectation that it would be replaced in due course anyway.

In what will no doubt be viewed a very positive stance for industry, patients and advocacy group who see the current system as being in need or urgent reform, the Committee considers the EU collaboration as “a longer-term potential solution and should therefore not preclude the HSE or Department of Health from undertaking immediate activity to enhance the availability of orphan drugs in Ireland”.

What Next?
A Committee Report can go either one of two ways – it may sit on the top shelf of the Oireachtas Library gathering dust, or it can act as a framework and catalyst to drive real political change.

My gut is this report will be the latter. Firstly, the orphan problem is also a political problem. Politicians are acutely aware of the scale and sensitivity of treatments being unable to secure reimbursement, where access programmes either do not exist or are coming to a close. Minister Harris, for his part, does appear to legitimately want to reform this space and is looking for sensible recommendations. Finally, (you thought you would get through a whole political briefing without jargon) in the era of ‘new politics’, TDs and Senators have been thrust forward into the position of driving policy and legislative change – particularly in the area of health where Sláintecare is the roadmap for health reform.

For more information, please contact Colin O’Donnell at codonnell@hanovercomms.com.