Mary Butler (Waterford, Fianna Fail) | Oireachtas source

I thank the Deputy for tabling this Bill which seeks to address an issue of great importance to us all. I would like put on the record the apologies of the Minister, Deputy Donnelly, who, as the Deputy mentioned, is attending the Cabinet sub-committee, but I understand the Minister engaged privately with the Deputy on the Bill in advance of this debate. I recognise his sincere interest in patients who are in need of orphan medicines. It is a cause he has championed for quite some time. We hear about it regularly at our parliamentary party meetings. I acknowledge the work of former Deputy, John Brassil, on this issue. He, too, was very passionate about it.

The Minister supports the intent behind the Bill. He also wants to secure faster access to medicines for the most vulnerable of our patients. I can safely say this is something we are all aligned on. As public representatives, we are all moved by the tragic circumstances faced by many people suffering from rare diseases. The pipeline of new innovative medicines coming to market remains strong and it is vital that Irish patients continue to benefit from these medical advances. I understand the Minister is open to any ideas or proposals that might improve our existing pricing and reimbursement assessment processes, with a special focus on orphan products. For that reason, he is not opposing the Bill. I do, however, want to use this opportunity to set out the current process and to outline recent reforms that have been undertaken.

The Health (Pricing and Supply of Medical Goods) Act 2013 is the statutory framework under which all new medicinal products must be assessed by the HSE. The HSE is the statutory decision-maker for the reimbursement of such products and the Act ensures that these decisions are made on an objective and scientific basis. In making decisions, the HSE carefully considers a number of criteria that are contained in the Act, such as clinical evidence, efficacy and the available resources. It is only fair to acknowledge that these are difficult decisions and those involved must balance the availability of finite Exchequer resources with ever-competing demands.

The 2013 Act does not include a specific rule set for the assessment of orphan drugs. However, the HSE and National Centre for Pharmacoeconomics, NCPE, have assured the Minister that in making their decisions they have particular regard for the unique circumstances surrounding orphan drugs. These criteria include not only cost-effectiveness and budget impact, but aspects such as clinical need and added therapeutic benefit against existing standards of treatment. The HSE can, and does, take a nuanced approach with respect to the particular challenges posed by rare diseases, such as small patient populations and unmet clinical need. This capacity for nuance is reflected in a number of reforms carried out to date under the existing Act. The rare disease technology review committee is fully operational. It carries out reviews of reimbursement applications for new orphan medicines in cases where the drugs group requires further guidance. This committee gives voice to rare disease patients and to clinicians with expertise in the specific illnesses in question. The committee played a key role in the HSE’s approval in July of Patisiran, a new medicine for the treatment of the rare, inherited life-limiting condition called hATTR Amyloidosis. Using the guidance from the committee, the HSE was able to take account of the challenges faced by patients in terms of unmet need and the potential impact of the drug, alongside considerations of cost effectiveness.

The HSE drugs group has been expanded to also include representation from the National Patient Forum and further clinical expertise in the area of rare diseases. The NCPE’s patient organisation submission process encourages the lived experience of patients, carers and their families, as well as their views on the difference a new medicine may make. This information is then used as part of the health technology assessment of the medicine. Since implementation of this process in 2018, there has been a doubling in the number of patient organisation submissions received and it is expected that this number will continue to increase. The challenge of accessing innovative medicines at affordable prices is one shared by our international partners. It is estimated that 100 new indications will receive European Medicines Agency, EMA, market authorisation in Europe each year over the next five years. The Minister, therefore, fully endorses the State's participation in international collaboration and co-operation in efforts to secure affordable and timely access to new medicines for rare diseases. Our involvement in the Beneluxa international initiative with other EU countries demonstrated its potential to have a positive impact in relation to orphan medicines.

As announced previously, in October the HSE approved the gene therapy drug, Zolgensma, for reimbursement for the treatment of spinal muscular atrophy. This followed the first joint agreement on the price of a drug between Belgium, Ireland and the Netherlands, carried out through the Beneluxa initiative. This Bill presents one such opportunity. However the Minister is also keen to maintain an open mind and be receptive to non-legislative avenues. He wants to look forensically at the current system and identify further options for reform. Increased transparency and communication would make for a more responsive system. The Minister is committed to seeing where this could be achieved across the NCPE, the HSE and rare diseases technology review committee.

Turning to the provisions of the Bill, I wish to outline the relevance of quality-adjusted life year and incremental cost-effectiveness ratio measurements. The quality-adjusted life year metric is the economic evaluation system used by the NCPE to determine the cost-effectiveness of a given medicine. It is a measure of the state of health of a person or group through which the benefits in terms of length of life are adjusted to reflect the quality of life. One quality-adjusted life year is equal to one year of life in perfect health. The incremental cost-effectiveness ratio expresses the value of health outcomes in terms of the cost per quality-adjusted life year. Our experts believe the quality-adjusted life year metric is a valuable tool in the decision-making process. The HSE and the NCPE consider it to be the best method we have to assess the cost-effectiveness of medicines in a systematic and scientific way.

If the HSE were unable to consider the cost per quality-adjusted life year thresholds for orphan medicines, it would have no guidance on their cost-effectiveness to support commercial price negotiations. The executive would, therefore, enter such negotiations in an uninformed and disadvantageous position. The Minister is assured by the NCPE and the HSE that the current system accommodates the approval and reimbursement of expensive orphan medicines. Fifteen of the 43 new medicines approved by the HSE in 2021, up to 12 November, were orphan medicines for the treatment of various conditions, including, but not limited to, rare cancers and spinal muscular atrophy. Together, they represent more than one third of approvals this year. In many cases, these drugs significantly exceeded the guideline cost-effectiveness threshold of €45,000 per quality-adjusted life year. That metric, therefore, does not pose any barrier to the reimbursement of orphan medicines. Indeed, the HSE has expressed concern that without having carried out such cost-effectiveness assessments, it would end up paying significantly more for orphan medicines than would otherwise be the case. The advice provided is that abandoning the methodology would potentially have added an additional €250 million cost over the next five years in respect of the orphan medicines already approved for reimbursement in 2021.

It is clear, too, from an analysis of the trends over recent years that the overriding factor in medicines making it through the process to reimbursement is the availability of Exchequer resources. The 15 orphan drug approvals to date in 2021 were enabled by an allocation in budget 2021 of €50 million for new drugs. By contrast, when no such funding was included in budget 2020, only eight orphan drugs were approved and, moreover, they were approved only when funding was made available through industry price realignments. For this reason, budget 2022 has allocated a further €30 million for the approval of new drugs. This will provide the HSE with the resources necessary to continue providing access for Irish patients to new innovations in the area of rare diseases. We cannot overlook that, in the context of finite resources, paying more for orphan medicines would have a very real impact on the health service's capacity to fund even more new medicines, including subsequent innovations in the area of rare diseases.

To reiterate, the existing assessment criteria in the 2013 Act do not pose an impediment to the approval of orphan drugs. However, our system is not perfect. No one familiar with the challenges faced by citizens with rare diseases would make that argument. The Minister has asked his officials to consider the available policy options in this area. In particular, he is hopeful that opportunities exist to improve the transparency of the assessment process and communication with its stakeholders, including patients and industry. Notwithstanding the concerns I have outlined today, I reiterate that this Bill is honourable in its intent and must serve as an occasion to explore the policy options available to us. For this reason, the Minister does not intend to oppose it, as we continue to strive for an assessment process for orphan medicines that will provide some of our most vulnerable patients with access to the innovative treatments they deserve. I congratulate Deputy Pádraig O'Sullivan on all his work in this area and his determination to bring the Bill to the floor of the Dáil.

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