SEMPER Unmet Medical Needs Janssen

Unmet medical needs

Moving from the subjective to the imperative

Talking about unmet medical needs tends to make most stakeholders on the subject grind their teeth, as it is so complex to precisely determine the contours of a notion that is as subjective as it is impatient. However, an acceptance focused on patient needs seems to crystallize the beginnings of a consensus, at least in the thorny issue of the needs/satisfaction ratio.

Recently, the EFPIA (European Federation of Pharmaceutical Industries) published the following statistics: in 2020, 13,437 women in Europe died of cervical cancer.

However, innovation is helping to combat this scourge, with the papillomavirus vaccine reducing the risk of contracting this cancer by 90%. These are millions of families who will not lose a sister, a daughter or a mother.

Around 15 million Europeans live with hepatitis C. But thanks to innovative medicine, this situation could become a thing of the past for 95% of patients. By replacing a life of care with a life of memories.

As the EFPIA rightly points out,

“addressing unmet medical needs has been the starting point for all these advances. It drives the work of the 120,000 industry employees who hold R&D positions throughout Europe and it guides the investment of 42 billion euros in European R&D by industry”.

What are we talking about?

As is often the case in the field where public health meets public opinion, an ethical and societal question arises that resembles “what is the common good?” What is a need, and what is satisfaction? On the edge of the waiting room philosophy, you have three hours...

In the field of unmet medical needs, it is first at the level of the very definition of the concept that interpretations diverge. Because the "best" differs depending on the point of view of the person defining it: the patient, the industry, the healthcare structure, research, and the doctor.

Is an unmet need a remedy that exists but is not effective enough, a therapeutic void, a protocol that is too restrictive, an infrastructural or legal lack? Must the disease that this need combats be serious, or even fatal? Who then defines the notion of seriousness? Do chronic diseases fall within the scope of unmet medical needs? It is easy to fall back into the ethical or moral debate.

The complexity of the definition is undoubtedly at the origin of the obstacles that currently affect the development of solutions.

Broadening the scope of interpretation of the European definition

For the first time, in 2023, the European Commission proposed a definition of unmet medical needs as part of the general reform of the pharmaceutical sector. This definition is very restrictive, on the criteria we have mentioned (bi-mortality, no existing therapy, etc.) and risks not filling many blank areas in terms of pathologies and care.

On this point, IML fully shares EFPIA's position: "This definition is too restrictive. It excludes the scope of innovation, and does not sufficiently take into account the needs of patients".

Based on the evolution of science and the needs of patients, EFPIA considers that an unmet need is "a condition that is not properly prevented, treated or diagnosed by authorized interventions".

This meaning is part of a neutral understanding of the context and may be led to take up certain principles of distributive justice.

Starting from demand and no longer from supply

The position of the pharmaceutical industry is, and must always be, to provide solutions to meet the needs of patients. This is why it was born and continues to work.

However, it is at the level of the patient's feelings, who are the only ones to experience their illness, that the notion of need takes on the most meaning. Perhaps the time has come to think about new collaborative, inclusive models, to return to basics and start from demand, accepted as clinical proof of the real world.

This is also where the beauty of innovation lies: when it leaves the lab.

The use of complete and real patient health data (RWD), segmented and regularly updated, will also help identify where the real unmet needs are. They are added to the need to combine technological, digital, citizen, collaborative clinical advances, at the heart of governments' public health issues.

IML supports the EFPIA’s positions on the need for a less restrictive definition of unmet medical needs and defends the idea of ​​a bottom-up consideration of needs, both agile and measurable.

If the overall quality of life of patients is the objective, the search for therapeutic quality of life is a major means.

Aurélie Boob, Public Affairs Associate, IML

Biolabs innovations:

meeting patients’ hopes

Creating a drug that cures is the cherry on the cake and the dream of every laboratory. When the treatment makes the disease more bearable, is less restrictive to administer, significantly increases survival and reduces side effects, we are already talking about innovation. This is fully within the framework of a broader definition of unmet medical needs. Maria Fernanda Prado, General Manager for the Benelux at Johnson & Johnson Pharmaceutical companies, explains how a therapeutic innovation can lead to a cascade of positive effects.

The EFPIA proposes a broader definition of unmet medical needs, focused on the patient, which would encourage different research avenues for all patients. Do you support this initiative?

Maria Fernanda Prado: Yes, I support this initiative.

The current definition is limited to specific life-threatening or severely debilitating diseases and then to drugs that meet certain criteria such as significant reduction in morbidity and mortality. I think the concern of EFPIA and our industry is how we view the concept of UMN, from what perspective. We believe it should be patient-centred.

While we have therapies for many difficult-to-treat diseases, the critical question is how can we be sure that we have addressed, in the best possible way, all the unmet needs of these patients. Some diseases, such as chronic diseases, are not life-threatening but nevertheless have a significant impact on patients.

In a strict framework, some needs may not be perceived as unmet because the main disease is already covered by a treatment.

However, there may still be a substantial need for the patient, to improve their treatment, their adherence, their quality of life or even their outcome. That’s why we believe in ensuring that the patient perspective is taken into account when defining unmet needs.

What are the latest advances in the industry in UMN
and do you have any specific examples to share?

Maria Fernanda Prado: There are many examples in our industry, but let me mention that the evolution of haloperidol is an important example of incremental innovation that addresses a significant unmet medical need. Haloperidol is a substance that has been used as an antipsychotic since its invention in 1960. At that time, there were no antipsychotics, which represented a huge unmet need. Even today, it is a very relevant medicine that has paved the way for other active ingredients, other treatments.

From the perspective of psychotic or schizophrenic patients, one of the main problems was compliance, because these patients had difficulty following the treatment. When this drug was invented, it had to be taken in the form of drops several times a day. Getting patients to fully adhere to their treatment was, understandably, a major challenge.

Today, decades of research later, the administration can be done in a radically different way. The new molecules act over a long diffusion period, which allows patients to only worry about their treatment a few times a year. From the perspective of patients, who need it their whole life, this is a fundamental improvement, even if it is a classic incremental innovation.

Other examples?

Maria Fernanda Prado: We also see the beauty of innovation in oncology, and the treatment of multiple myeloma is an example: cell therapy offers a new approach to treating cancer by using the patient’s immune system.

CAR-T (Chimeric Antigen Receptor T-cell) cell therapy is a specific form of cell therapy in which CAR-T cells are designed to destroy cancer cells that express a specific antigen. CAR-T cell therapy is a personalized treatment for a given patient, based on the collection of their own blood cells. This cutting-edge technology offers prospects for

It is important to ensure that the patient perspective is taken into account when defining unmet needs.

patients for whom other therapies have no results or have insufficient results. These solutions will become essential in the near future and will meet the needs of patients.

Because it goes further: once you have triggered a mechanism of action, you evolve a therapy with new molecules, and now you even combine different mechanisms on the same treatment, to get a superior result. Until we find the cure, there will always be unmet needs to solve and improvements to bring to patients. That is why I say that if we consider unmet medical needs only as therapeutic breakthroughs or in very specific and narrow areas, we risk losing sight of what an unmet need really is from the patient’s perspective. That is why it is so important to have a broader definition starting with the eyes of the patients. This is what EFPIA, pharmaceutical companies and pharmaceutical associations agree on and make their voices heard in the EU pharmaceutical package.

Do you think that pharmaceutical companies are ready to adopt this mindset, to become patient-centric?

Maria Fernanda Prado: As a society, we will have different healthcare needs in the future. We will have more complex diseases to treat because we will live longer. I think the industry is ready to go the extra mile to meet these needs. But the big question is whether all stakeholders are on the same page. I fear that this is not the case if we look more closely at this discussion on UMN. There are different perspectives from the European regulator, payers, patient associations and industries.

We are ready to discuss constructively with all stakeholders and move forward. In my company, as in many others, we believe that health is a whole. Our strength in health innovation allows us to build a world where complex diseases are prevented, treated and cured, where treatments are smarter and less invasive, and where solutions are personal.

avril 2024 - Semper Luxembourg