Tackling uncertainties for rare diseases (L6)

Go fast or go far?

I am a medical doctor by trade. As all medical doctors will know, the first commandment of being a medical doctor is ‘first, do no harm’. But a very close second is the mantra that evidence is the holy grail, and the holy grail of evidence is the RCT. But what if an RCT is simply not possible, for example if the disease you are studying simply does not have enough patients to conduct one? That is where Real World Evidence (RWE) comes in. Real World Evidence refers to information collected outside of a clinical trial, for example from electronic medical records, and it can help address evidence gaps in the field of rare diseases.

There are about 7000 rare diseases, defined as diseases that affect fewer than 1 in 2000 people. People with rare diseases are often overlooked when it comes to SDG3. To quote Helen Clark, former prime minister of New Zealand: “No country can claim to have achieved universal healthcare if it has not adequately and equitably met the needs of those with rare diseases.” The small number of patients in these diseases, sometimes as few as 50 or 60 worldwide, makes it very hard to generate evidence the traditional way. This leads to heart-breaking examples where drugs or technologies with possible major impact are developed, but access cannot be guaranteed due to the uncertainties surrounding them. We need to address these evidence gaps. Continue reading

Man vs Machine (L5)

AI is risky but rewarding

Gastein this year has overflowed with optimism about the opportunities presented by the use of AI in healthcare. But many people have deep concerns.

This session, organised by Acumen Public Affairs, was a first for the EHFG: a ‘Cambridge Union’-style debate – there is no need to talk of that other place. This means that two speakers propose a motion, which is voted on by the floor. They then speak in favour for ten minutes and two other speakers respond and argue against the motion. The debate is then opened to questions and comments from the floor, to which both sides get a chance to respond. Finally, the proposing side get five minutes to give their closing remarks, followed by the opposing side, and there is a second vote.Brian O’Connor and Rachel Dunscombe proposed the motion: ‘AI in healthcare: the rewards outweigh the risks’. Tamsin Rose and Martin McKee spoke against, and David Rose and his very loud bell kept everyone to time. Continue reading

Sustainable strategies for addressing health inequalities (F12)

A future we could have

Imagine a sunny autumn weekend in a not so distant future. You’ve had a stressful week at work and you could really use a day off to wander a beautiful alpine valley or maybe have a relaxing stroll at the seaside. You leave for your preferred destination by a train in the morning, you have a tasty and healthy meal after spending a majority of the day hiking and taking photos of scenic panoramas. On your return journey, you share the photos with your friends over social media – as is the norm. You arrive back home quite late completely exhausted. But also relaxed and without any anxieties that bothered you throughout the week. Now imagine the train ride, the tasty meal, and the data transferred to upload photos were all free of charge. Well, at the point of service at least. What would our world look like if we accepted the concept of universal basic services? We are already familiar with the concept when it comes to education and healthcare. Would expanding the array of services to include, for example, transportation, nutrition, and communication make our societies more equitable?

Considering universal basic services as an alternative to universal basic income was one of the concluding remarks of Sir Michael Marmot at the forum on sustainable strategies for addressing health inequalities. It definitely is a bold political choice, but could it be sustainable? Could it be effective? It is an interesting food for thought. But much of the discussion during the forum focused on actions that we can do here and now. Continue reading

Enabling the digital transformation of cancer care in Europe (F11)

Seeing eye to eye – Enabling the digital transformation of cancer care in Europe

This morning at EHFG we are talking about enabling the digital transformation of cancer care in Europe. In this session we are exploring current opportunities and disruptive technology to inform policy solutions to transform cancer care across the European Union. The possibilities seem endless. We cannot blame technology anymore for not providing the hardware/software. Access to personal data, a health record that is fully accessible to the patient, an app that connects the patient directly with his doctor, it is all readily available. So why is it we are underusing all these opportunities? After all, just using what is out there can save 125.000 lives per year on digestives cancers alone.

To be frank, many European healthcare providers are rather illiterate when it comes to digitalization and the use of their data. I am not saying we don’t know how to work a CT scan and make a solid diagnosis from this data. But benchmarking this data real-time and, with this, creating added value for the patient and the healthcare provider is far beyond the technological knowledge and capabilities of most healthcare providers today. Continue reading

Vaccines, Leaving no one behind. It’s time to give it our best shot! (F10)

Today at the European Health Forum, we were challenged to consider what actions should be taken to implement a life-course approach to vaccination across Europe. Many countries have been struggling with vaccine mistrust and misinformation. Measles outbreaks have led to dozens of avoidable deaths around the continent this year. Other countries, including my native country of Ireland, have seen major declines in HPV uptake. John Ryan of the European Commission emphasised at the outset that vaccination can save countries ten times more than the cost of the vaccines themselves. An important reminder of the relevance of this issue, even if preaching to the converted!

A series of four case studies were presented to inspire actions that could be taken across different European countries to improve vaccination uptake. Representatives of the European Patients’ Forum, UK’s Vaccine Confidence Project, Ethno-Medical Centre in Germany, and EU Pharmaceutical Group were put through their paces in a series of ‘TED talk’ style presentations. Emilie Karafillakis highlighted the strategies, which have worked best in reversing the decline in HPV uptake in Denmark. This resonated most with me, since in Ireland we have improved HPV coverage from 50% to 65% in the last 18 months. We are still a long way off the>80% coverage we had achieved in 2014, but by engaging with key stakeholder groups, getting on social media, and highlighting real-life personal narratives in our communication strategies we are finally counteracting much of the misinformation and scaremongering around this vaccine. Certainly, we learned and benefited from the Danish experience in this regard. Continue reading