Our Scientific Projects Officer, Abby’s experience of the first REMEDi4ALL Hackathon!

Jul 3, 2024

This week’s blog is written by our former Scientific Projects Officer, Abby Stock-Duerdoth who went to Barcelona for the REMEDi4ALL Hackathon event.

The Hackathon is a 5-day educational retreat where early career researchers come together to explore drug repurposing.

The R4A team guided them through the process, from exploring their assigned disease through to developing a research plan.

They worked in teams to ‘hack’ rare diseases and try to identify potential repurposing solutions while they learned about repurposing.

At the end of May, I attended REMEDi4ALL’s first Hackathon event.

Nestled in the hills of Montserrat just outside Barcelona, our hotel had stunning views.

But we weren’t there for the picturesque scenery or warm Spanish spring, we were there to guide 14 early career researchers (ECRs) through the drug repurposing process.

This was no easy task.

A huge range of expertise is needed to see a drug repurposing project through from idea generation all the way to agreeing a price with national health bodies.

Our ECRs were working through the stages as hypotheticals, but it was still a huge amount of work for the three working days they had.

The ECRs were divided into three groups and each was assigned a different neurodevelopmental disorder: Rett Syndrome, CDD, and Dravet Syndrome. Their task was to identify a repurposing candidate and create a target product profile and repurposing development plan for how they would get it to patients.

I was really impressed by how they worked together in their teams, learning from each other’s expertise and utilising each other’s strengths. It represents beautifully how in a real-world project, collaboration and team work really are key to success.

No collaboration is more important than the one between researchers and patients. Patients (and their representatives) need to be at the heart of decision making and our ECRs really took this on board.

We organised a video call for each group with a patient family representing the disease they were researching. These conversations had a massive impact on the teams and where they took their projects.

Each team learned something different, and discovered the unique needs of the patient population they were working with. They saw that, even with the best intentions, it is impossible to truly meet patient need without working with them directly.

There was diversity in how much risk patient families were willing to take with a new treatment. For example, the family representing Dravet syndrome felt their child’s condition was well managed with current treatments, even though they’re far from perfect.

They were wary of new treatments disrupting their routine and would need a repurposed medicine to have a very well understood safety profile and minimal side effects.

In contrast, the family representing Rett Syndrome had a much greater appetite for risk. They had previously been involved in clinical trials testing medication that had a risk of making their child blind.

They would risk that again for a treatment they might help their child communicate more easily. This highlights how, even amongst relatively similar neurodevelopmental disorders, there is a range of needs, expectations, and priorities amongst patients.

Our ECRs only spoke to one family each and to really understand the needs of the patient population, they would need a much larger group.

However, the insights that they gained did change the way they think about patient engagement and how they will work moving forward.

Several participants told me how much they valued talking to the people who would benefit from their proposed treatment.

They said that getting an understanding of the lives rare families stuck with them throughout the rest of the hackathon.

At every phase of development, they referred back to what they had learned and had the experiences of the family in their minds.

A key take-away for the group investigating Dravet Syndrome was that children grow up. Often we think about the children who live with rare disease, but forget that they too become adults.

As a child grows into a teenager and beyond, their needs from a medication may change too.

Their priorities and feelings about side effects might change too and we need robust ways to engage with patients throughout their lifetimes as well as their families.

Throughout the hackathon, patient engagement was weaved into every aspect of the repurposing process. On the arrival afternoon, participants played a game which simulated the repurposing process, where patient engagement was a key step.

On the second day I, along with colleagues from REMEDi4ALL, presented a plenary session outlining key aspects of patient engagement.

We introduced participants to the different methods used to work with patients including focus groups, surveys, and bring patients into development team meetings.

We highlighted how crucial patient engagement is for a clinical trial in terms of patient recruitment, retention, and outcome measures. This helped participants to plan what they wanted to discuss with patients in their calls the next day.

At the end of the Hackathon, the three groups presented their findings and their plans. I was delighted to see how seriously they had all taken patient engagement and how it clearly shaped their plans.

Their drug selection was informed by the symptoms and side effects that mattered most to patient families and their trial designs were adapted to the needs of the participants.

Some groups even began thinking about how they would use the patient voice to support their health technology assessments and business cases.

I hope the hackathon has taught our ECRs a lot about understanding medications from the perspective of the people who will use them.

Educating researchers in this way early in their career ensures that patient engagement becomes a cornerstone of their approach to drug development. I hope that the next generation of clinical, scientists, and health economists appreciate, respect, and aim to understand patients and the role they have in research.

This will create an open-minded drug development community with the flexibility and compassion to truly serve the rare disease community.