Dr. Maggie De Block:

I thank the Chairman, Senators and Deputies very much for inviting me to this session regarding a national action plan for patient access to innovative medicines. In the tight time slot allocated, I would like to explain how Belgium under my tenure as Minister of Social Affairs and Public Health can serve as an example of good practice.

First, I will explain the pharmaceutical pact although I will not enter into detail. I will highlight the most important aspects to demonstrate our case. Next, I will explain in a nutshell the BeNeLuxA initiative. We felt efforts to increase international co-operation is to the benefit of patients with rare diseases. Ireland was the fifth country to enter into the agreement, for which I am very grateful. I will emphasise budgetary aspects and procedural characteristics and how to influence the time of access to a reimbursed product for the patient.

In light of the elections of 2013, I thought it was important to consider a tailored pharmaceutical policy and there were several good reasons to do so. Together with representatives of the board and the generic and brand name drug companies, we decided on a multiannual agreement that emphasised four main topics for the benefit of the patient. The first was to guarantee better access and lower the out-of-pocket cost for the patient. Next, the pharmaceutical industry in Belgium is an undeniable asset of our national economy. Often, public opinion is very critical regarding the pharmaceutical industry and thus regulating the transparency and ethical aspects was the third pillar of the pact. Finally, we had to agree on a budgetary pathway that would allow growth and access to innovation but at the same time keep the budget under control. This meant that structural savings were necessary for some aspects and an agreement at an early stage would increase mutual understanding. One of the spillovers of this policy was the reduction of the time to access, although the pharma pact did not include structural reforms of the procedures that lead to the reimbursement itself.

Several factors influence the time delay in which a therapy will be accessible under reimbursement for the patient. One aspect depends on the strategy of the company itself, for instance, preferences for bigger markets. It is hard for national governments to influence these strategical choices. The second aspect that contributes to the time lapse in which a reimbursed drug is available is public investment and creating a favourable ecosystem. The last aspect is the administrative procedure to apply for reimbursement and how long this procedure takes, at which milestone a company can file for reimbursement, how much room there is for negotiation, what the conditions are regarding data and the evidence to obtain reimbursement for a certain medicine.

An important aspect that steered the time to access to reimbursed drugs was the budgetary framework. As we can see from the budgetary evolution, the pact scheduled structural budget cuts but at the same time left a margin for controlled growth.

On an annual basis, 0.5% growth was foreseen. There was negative growth in the previous years. In reality, the average growth was 1.4%. One of the reasons for this was an underestimation of the amount in the pipeline that would enter the market. For some medication, like immunological medication, we thought that the train was still far away but it was in front of our noses. For some pathologies, only poor national epidemiologic data was available and it was difficult to estimate the price. Approximately €1.4 billion of new measures and reimbursements in pharmacy were allowed.

Nevertheless, all the savings and expenditure cuts foreseen in the pact were implemented. Among others were the patent cliff, the clawbacks and encouragement to prescribe the lowest priced drugs. In 2018, the pharmaceutical industry paid a total product tax within the public healthcare insurance budget of more than €729 million. The budgetary framework had the perspective of four years because the elections were in 2018, so the pact was in place from 2015 to the end of 2018. As of 2019, the budgetary measures were taken without a multiannual perspective and, as can be seen, this resulted in an immediate rise in the budget.

In my opinion, to accelerate access and reduce waiting time, a sound budgetary perspective is equally as important as the administrative procedure itself. This brings us to timelines of procedures and reimbursement of other pharmaceutical products. Once the European Commission has endorsed the European Medicines Agency, EMA, market authorisation, pharmaceutical companies can apply for reimbursement in the Commission for reimbursement for medicines. That is stated in the administration of the ministry. A classic procedure takes a minimum of 180 days and due to possibilities to stop the clock for the pharmaceutical firms - for instance, to prepare answers to questions for the committee, the time-lapse might increase. If no solution is found in this Commission, a managed entry agreement can then be negotiated by law, covered by the confidential clause in a contract that defines the rights and the obligations of both of the parties. The operationalisation of the decision to reimburse a medicine sometimes took up to two months after notification. We used technical intervention to shorten this timeline.

Taking market authorisation as a starting point, the minimum procedure time is 180 days plus 120 days so that results in 300 days. However, when we look at the statistics on the average delay to availability, it is much longer at 440 days. If we wanted to further reduce this time-lapse, structural reforms of the procedures would be necessary. The fast track is an example of such a reform. We created a fast track for new drugs with new indications. The old procedure still remains. However, if some drugs prove their quality and robustness of outcome, in terms of quality of life or other indications, then the procedure is shortened for the new indications. This procedure of EMA is then one plus one month. Learned conditions in data to support the demand are directly linked to the new indications.

This was mostly done for immunotherapy which was very new in the treatment of cancer, for example. Since the introduction of the fast track in 2017, 20 indications for immunotherapy have been reimbursed and the impact of these procedures in terms of an adjusted quality of life and years gained is enormous. This was a big achievement and great progress has been made in treating patients for diseases for which nothing was on the market previously.

This system needs to answer so-called "compassionate use" for unmet medical needs and applications can be submitted even before market authorisations are licensed. The procedure can lead to early access for the patient two years earlier than in the regular procedure. Most of the innovative drugs are reimbursed under a managed entry agreement. Specifically with regard to orphan drugs, in the period between 2016 and 2019, 29 new products became available in Belgium. In Ireland, it was only 12. Between 2014 and 2020, we signed 33 new orphan drug products for 36 indications for reimbursement. Previously, it was only a few a year.

With regard to expenditure for managed entry agreements, if a company does not comply to contractual objectives, then refund levies are charged. Managed entry agreements, MEAs, now have a share of 11% in the total refunds for medicines budget exceed levies. That is compared to 1% in 2014. We can see that the share has been growing.

We will now come to the BeNeLuXA initiative. It was first Belgium, the Netherlands and Luxembourg. Austria and Ireland then joined but BeNeLuXair sounds more like an airline so we decided to keep the name because it was so well-known, but we were very glad that Ireland joined us. The BeNeLuXA initiative involved international co-operation. We started on horizon scanning, health technology assessment and shared policy expertise and practices. Towards the end, we also added in price negotiations which were not simple because all countries had different administrations and different systems of reimbursement. You might ask why we did it? I mentioned earlier that bigger markets are more interesting to the pharmaceutical companies, and possibly lead to lower prices for the patients. This was one of the main motives for considering co-operation between smaller countries because it resulted in added value for the patients. It is clear it is not only about reimbursement. The horizon scanning taught us much and helped us understand what could be expected to enter the market in coming years. Through mutual recognition of each other's health technology assessments, reimbursement procedures run more efficiently and effectively. Information sharing is always good to improve the market position as are joint negotiations for pricing. For rare diseases, the prices are very high. We also wanted more transparency on pricing between the collaborating countries. The first practice case was Spinrasa. Last year, with the BeNeLuXA countries, Ireland also reached an agreement for the reimbursement of Zolgensma.

To conclude I will outline, in my opinion, the most important lessons learned from the pharma pact and BeNeLuxA. The multi-annual framework for the budget allowed for the budget allowed for better guidance of the pharmaceutical policies. Having several procedures to come to reimbursed medicines increases access. Within these procedures there is also room to deploy several instruments. For example, it is possible to have fees and taxes and to establish different entry moments to apply for reimbursement, reducing delays in the decision process. A sustainable reimbursement ecosystem creates spillover effects that promote accelerated accessibility for the patient. In the mid term, BeNeLuxA should ideally evolve to enhanced co-operation.

I thank Senators and Deputies for their attention. Of course, I am willing to answer questions and members may also contact me via the address given to the committee. I will also share everything I said today with the committee.