It’s often said that two heads are better than one to tackle a problem. This can be said for Watson, Crick and Franklin, the Avengers, and even when it comes to developing life-saving medicines.
The more complex the problem, the more heads are needed to solve it, and that’s certainly true for the complex world of haematology – which spans multiple different diseases of the blood, affecting millions of patients worldwide. Despite considerable progress over the years, many of these patients still have limited treatment options available to them. The hope is that partnerships in science and healthcare could offer a route to furthering progress in haematology, as patients, physicians and pharmaceutical companies work together toward a common goal: delivering groundbreaking advances with the potential to transform lives.
Katya Moreno, Senior International Medical Director, Haematology, Roche, agrees: “Co-creating with partners, and both having the right mindset, is key for fulfilling our main objective – to improve patient lives.
For pharmaceutical companies, including Roche, this means looking outward as well as inward, to share data and learn from others, in order to help get innovative medicines to those who will benefit from them most, as quickly as possible.
Research and development is a fruitful area where collaborations can flourish. There is a wealth of scientific expertise across academia, with many inquisitive and passionate scientists who have hypotheses they want to test and patients they want to help. As a result of this, independent investigators and pharmaceutical companies often collaborate on trials and research projects – bringing together expertise and curious minds from across both fields.
“Collaborating with independent investigators is a no-brainer,” says Aurélien. “These independent trials allow both sides to answer relevant questions, contributing to advances in scientific knowledge and in clinical practice.”
Collaboration between the academic world and healthcare industries such as Roche, who have been working in the haematology space for decades, can help identify trends or insights from the vast amounts of clinical data across blood diseases. In turn, this could lead to future innovations. For example, this data could reveal insights that help predict whether a certain type of patient might respond to a certain type of treatment, which could help physicians and patients make more informed and personalised treatment choices.
It’s not just about delivering clinical trials and analyses of data. Partnerships can help to improve how trials are actually designed – fulfilling not only regulatory but also patient needs.
Innovation in drug design is moving at a rapid pace. This means that the way clinical trials are designed also needs to constantly evolve, to keep pace with the latest standards of care and technology. There are many examples of innovative trial design in drug development, all with the ultimate goal of generating outcomes that matter to patients and are relevant to the physicians managing their treatment.
There is also an interest in challenging the usual methods to evaluate a treatment. Blood cancer treatments have improved substantially over the years, with more patients in clinical trials living longer. This has led researchers, pharmaceutical companies and regulatory authorities to explore different measures of success that can effectively measure how a patient has responded to treatment in potentially shorter timeframes.
“One particularly interesting way of measuring success in some blood cancer clinical trials, that Roche has collaborated on with academic institutions, regulatory authorities and other companies, is minimal residual disease,” Aurélien says. “Using highly sensitive technologies to search for traces of blood cancers may help guide treatment decisions or expedite development of new treatments.”
The absence of remaining cancer cells may suggest a lower risk of a cancer coming back, so measuring minimal residual disease provides physicians with an opportunity to assess the extent to which a treatment may be working, and much more quickly than other conventional endpoints.
Healthcare professionals are the ones working on the frontline, providing care to patients. In doing so they get to understand the struggles patients face – from remembering to take their medication, to balancing their treatment with everyday life. They also bear witness to how well a given treatment is working for a given patient.
In haemophilia, Roche is working with hospitals, universities and academic centres to explore digital solutions, such as remote patient monitoring to help understand patients’ real time bleed status.
“The results of these studies would translate into impacting outcomes for patients,” Katya explains, “because they have the potential to be used to help personalise treatment decisions”.
And while it may be true that healthcare professionals bear witness to the challenges patients face, clearly it is the patients themselves who know what it’s like to live with a blood condition – day in, day out. So in order to make treatments that meet patient needs, who better to ask than patients themselves?
“We have patient advisory councils, where we bring patients into discussions from the beginning to understand what is important to them when it comes to treatment approaches,” says Katya. “If they are living with a chronic condition for example, what quality of life really means for them?”
Katya and her team then take these insights and feed them back into research and development, not only tailoring clinical trial designs but also making trials less burdensome for patients, to help make it possible for patients to take part by minimising the impact it has on their daily lives.
Partnerships come in all shapes and sizes, but one thing that is common across the board is the ingredients for a successful one. Like in a friendship, each side must have respect for the other, listen genuinely to the other’s ideas, and be willing to compromise and find a middle ground.
“Collaboration is central to Roche’s approach in haematology because we fundamentally believe more can be achieved by coming together,” says Aurélien.