Question: Let's start by talking about the amendments that insist on protecting incentives for innovation and economic viability of orphan drugs. Do you think the current European framework sufficiently guarantees that balance or that it needs to be strengthened?
Answer: Responding very directly, I believe it is necessary to reinforce it, although the changes that are being introduced recently in this reform of the pharmaceutical package place a great emphasis on both innovation and incentives that keep public interest objectives very present, that we improve the development of treatments for these unmet medical needs and also the market. It is, above all, about incentivizing research and facilitating the faster arrival of innovative medicines to the Member States. In the case of medicines for rare diseases, it is more difficult for them to reach the market and conduct clinical trials. That is why benefits are being considered that can be granted to pharmaceutical companies that conduct research so that, in the end, these diseases and these unmet needs are attractive to a sector that is so necessary, also for the patients and families who suffer from them.
Q: What lines are being explored from the European Union to ensure a predictable regulatory environment to give this confidence to laboratories to invest in the development of these drugs?
R: Broader protection is foreseen, above all. There is a maximum limit of up to eleven years, which in the case of orphan drugs can reach twelve, with combined regulatory protection where innovators addressing rare diseases, which do not have a current and available pharmacological treatment, can benefit from that market exclusivity. Here the problem we have, many times, is the competition we have, for example, with the United States or with Asia, while Europe has focused more on protecting the patient and the added value that European research can bring before economic benefit. And then we must also take into account that despite, well, the fierce competition that they want to give us, especially from the United States, with that announcement of tariffs also to the pharmaceutical sector and so many other issues - such as prioritizing American manufacturing -, in the end this market represents at least 25% of its production in the sector, which means that Europe remains necessary and attractive. And therefore, we must continue to insist in the Union, on that added value that puts the patient at the center and not just profit.
Q: One of the main complaints from patients across Europe is the differences in access, what should be done from the EU to ensure more equitable access to foster equity on the continent?
R: Certain measures are also being incorporated into European regulations to make this access much more equitable in the territories. For example, when we talk about a shortage of a medicine - and hopefully it will also apply to orphan drugs - if a Member State requests that medicine because it is not available and the pharmaceutical company does not supply it, it is true that the deadline is very long: three years. If this happens, it could lose its exclusivity in the European market, so it would have to be thought about very carefully.
But here we must point out, in the interest of truth, that often the barrier is not in the European framework: often the problem is in the internal market, within the Member States themselves. The main problems arise from the regulations when it comes to acquiring that medicine, negotiating with pharmaceutical companies, or the price when it comes to approving that medicine within the Member State. Hence the differences in price also for the same medicine from one Member State to another due to the economic capacity of national systems in its acquisition.
We are not part of a federal union of states, we have our own powers. And health is a national competence, so the internal regulatory framework must be greatly respected. We are faced with 27 different systems that, on the other hand, as in the vast majority of member states, we are talking about universal national health systems, we remain attractive for innovation and for research into these rare or uncovered diseases. Because, ultimately, it is not the same to operate in a market where the universal provision of health care is not mandatory, as in a market like the European one, of 455 million people, where States do have to provide a universal service. And this is what makes these general incentives attractive and tends to ensure that there is not so much inequality from one state to another. It is also fundamental to homogenize and coordinate all this regulation and these procedures that we are articulating so that, effectively, the postal code is not what dictates when facing a health problem.
Q: What impact do you think initiatives like the Biotechnology Law or the Critical Medicines Law will have on this innovation ecosystem and thus achieve the development of new drugs for rare diseases in Europe?
R: The Biotechnology Law now begins, it is one of the star regulations in this term, because it is precisely the one that talks about innovation, it is the one that must make us differentiate ourselves in this global market. I continue to insist on emphasizing that the difference in our legislation is the patient, who brings value to our systems, who brings value to citizens. And this law, above all, aims to foster research into new drugs through the genome, new oncological treatments, which will make us advance through other tools such as artificial intelligence.
In this regard, there is also a directive and a specific fund to invest in health and artificial intelligence and in the European Data Space, which I believe is a revolution and which will have a great impact on this new model of research and progress that it will bring to science through biotechnology. It will be a substantial change that I believe will be very perceptible in a very short time.
The Critical Medicines Law responds to the consequences of COVID. We realized that the strength of our systems was not such. Or that it was not as great as we understood. In Europe we have lost being very pioneers in this sector and in this strategy, so measures must be promoted so that citizens do not run out of basic or critical medicines, such as an antibiotic or a commonly used medicine that cannot be available on the market.
It would be about having a series of European reserves in which we never reach a shortage. We do not forget about the masks, for example, even though these are devices and they made us go through such a bad time.
It is about that list of critical medicines that is updated regularly, is available in Europe. And if one Member State has it and another does not, it can be requested so that this equity shortens the differences between one and another.
Q: How can data interoperability and real-world evidence help improve the assessment and access to treatments for rare diseases?
R: I believe it will be a before and after. If there is something that is going to revolutionize the research of rare diseases, precisely the key is in the data. The reason is that we have very few patients. Furthermore, we all know that it takes a long time to diagnose a rare disease, being very optimistic four years. And, being more realistic, we would be closer to ten
Therefore, it is a true revolution to be able to share experiences, to be able to have reference centers where to examine which patients are having the same pathology and which treatments are being effective.
Having said that, one must also be very respectful - and this is being worked on very intensely - that in the end the data belongs to the citizens, to the patient. And, therefore, consent must be key: we can use this data for research, we can use the data for interoperability, as in Spain with our health card. If we move or relocate or go on vacation to another member state, we must have our history to be able to receive treatment. And it must also be available to science, always with respect and awareness that the data belongs to the patient.
Regarding artificial intelligence, it will also represent a clear revolution: it will be able to manage and store all that necessary data and the symptoms provided by both patients and the professionals who are treating these patients with rare diseases. and offer solutions in a much more agile way. But emphasizing, in an absolutely unanimous and necessary way, that there must always be a health professional behind it to analyze, diagnose, treat, and offer a solution to the patient. A machine can never do it.
Q: The European pharmaceutical reform is proposing changes to orphan drug incentives. How do you think the framework of Regulation 141/2000 should evolve to continue attracting innovation to the continent?
R: Coordination is fundamental and financing is fundamental. Because, as I like to say, without financing there is no implementation. We can develop a wonderful plan with a series of guidelines to give to the Member States - because I insist that health is a national competence -. Now we are discussing that we would find it very interesting if we could have a regulation for rare diseases that can ultimately reach the people who really need it.
For that, pharmaceutical companies must be made interested in this research, by obtaining incentives. Even if it is, for example, for other drugs that are more profitable for them, as long as they have the commitment to continue researching these types of rare diseases or conducting joint cross-border clinical trials. Or that if there is research outside that wants to be implemented in Europe, there is that obligation that within 90 days you will request authorization to start marketing it within the European Union. Because what we cannot do is invest in research or clinical trials and have a third party take advantage of them.
Therefore, yes to incentives, yes to financing research, yes to financing the plan, but let the benefit be a European benefit. And that the pharmaceutical companies we are paying for their work do not end up relocating afterwards and then go to sell the product, which with so much effort we have managed to bring to market, and go to take advantage of other markets that are not ours. When I speak of this, I am referring to our patients, who must be at the center and be the beneficiaries of those incentives that the industry ultimately receives.
Q: Let's finish by talking about the future. What measures do you think should be strengthened to consolidate the position of rare disease drugs in the coming years?
R: Coordination, absolute coordination. We cannot have such an ambitious and broad pharmaceutical package where Biotech, Medical Critical Act are included, where medical devices are included, where we are working on cancer plans, cardiovascular, now on the rare diseases plan… And there is no coordination. Without that necessary coordination, which is what we want to capture in this plan we are now immersed in, we would be more doomed to failure.
Therefore, we must be able to create an instrument that harmonizes all this legislation, these instruments that we are putting in place, so that they are effective and so that there is permeability in the public health systems of the European Union that make the effort truly worthwhile.