PCM4EU @ MPNE 2023
collaborating on precision medicine implementation
Face to face workshop
28th April 2023, 9am-12pm
NH Brussels BLOOM
Rue Royale - Koningsstraat, 250, 1210 Brussels, Belgium
Program face-to-face meeting Brussels
Thursday, 27th April 2023
18.30 Dinner for workshop participants at De Ultieme Hallucinatie
Rue Royale, 316
+32 (0) 2 889 0316
Friday, 28th April 2023 - room INTO THE BOX, Hotel Bloom
Why DRUP-Like Clinical Trials, DLCTs, are relevant for the European Cancer Patient Community
Bettina Ryll, Hans Gelderblom and Kjetil Taskén
10.00 Coffee break
2 Case studies with discussion
Facilitation Andrew Evans
We will systematically work through 2 concrete challenges encountered during the implementation of DRUP-Like Clinical Trials, using a set of established tools:
presentation problem case - 9'
Ishigawa analysis -9'
Dot vote for top 2 fishbones - 5'
Fishbone 1 Productive Thinking Exercise - 9'
Fishbone 2 Productive Thinking Exercise - 9'
Present / reflect - 9'
Case study 1 - establishing international DLCT cohorts/ cross-border access to DLCTs
Case study 2 - access to Molecular Tumour Boards
Sahar van Waalwijk van Doorn-Khosrovani
Conclusion and next steps
12.00 lunch in cafeteria
13.00 MPNE2023 starts
Motivation and background for this workshop
PCM4EU is a precision medicine implementation project funded under the EU4Health program. At the centre of PCM4EU are a group of national clinical trials built after the original Dutch DRUP trial, therefore referred to as DRUP- Like Clinical Trials, or DLCTs for short.
Why are DLCTs relevant for the European cancer patient community?
DLCTs offer access to molecular testing- this is the particular focus of PCM4EU- and matched therapies for those cancer patients whom all other therapies have failed. Inclusion criteria are broad and there is no risk of randomisation to an ineffective comparator. The trial has an early decision point preventing that patients are exposed to side effects from ineffective therapies. Some countries have succeeded in building structures that ensure that every patient in the country can access the trial, resulting in high equity.
The figure below shows the different tumour entities represented in IMPRESS, the Norwegian DLCT, showing the relevance of DLCTs for the most desperate patients from the entire cancer patient community.
What makers DLCTs different from other clinical trials?
The core of every DLCT is an independent national academic clinical trial. However, some (NL, NO) DLCTs already include a pragmatic outcome-based risk sharing agreement and collaboration with HTA bodies, this has for example led to the reimbursement of Nivolumab for MSiH patients in the Netherlands. REF
DLCTs further link up with basic, implementation and health economic research. From an organisational perspective, DLCTs are interesting as the community is self-coordinating but has independent units, with similarities to the working of MPNE and WECAN. DLCTs should therefore be thought of as 'precision medicine implementation devices' rather than stand-alone clinical trials.
With their focus on benefit to patients, cancer patient organisations involuntarily accumulate knowledge and networks along the full implementation spectrum, linking decision makers in their respective cancer area. We are used- albeit not always successful- to work in heterogeneous communities, leveraging diverse expertise in a setting where resources are limited. DLCTs are relevant to the patient community as additional treatment options for our most desperate patients. The cancer patient community has implementation and organisational expertise of value to the DLCT community.
Please not another patient engagement tick box activity....
Patient advocates often complain about ineffective interaction with researchers and clinicians, mere tick box engagement for the sake of funding. Researchers and clinicians often complain about ineffective interaction with the patient community, mere tick box engagement in the name of the patient voice. Considering the number of cancer patients who are still dying, surely, this is unacceptable.
This is why this is a problem-based workshop: clinicians will present concrete problems they currently face when implementing a DLCT in their respective country- and then we discuss how to best address them. And we will only take forward what both sides find beneficial- win/win or no deal.
Why should I as cancer patient advocate participate?
Prepare to invest unpaid time to learn as much as you like and to try something new. This is an experiment, so we cover full-board accommodation and a travel allowance but there is no remuneration foreseen. It is a rare opportunity to work through a real-life problem with a group of like-minded people- everyone here wants to see more options for desperate patients and everyone invests considerably more than asked for in their usual job. It's an opportunity to truly use all we have learned over the years. And the future will depend on finding solutions that work for everyone.
But I don't have a Science background....
This project definitely favours those with a background in Science but as it has a heavy implementation part- which is what we in patient advocacy do most of the time- it will require a diverse set of expertise along the pathway. So the real point is the willingness to both learn and teach- everyone will be in and out their area of expertise and experience at times. So even without no Science background whatsoever, as long as you are willing to learn and put in the necessary effort- there just is no shortcut to knowledge- we can solve the rest. Precision medicine challenges the entire system, from the way we conduct experiments, how we design trials , approve drugs, definitely HTA, how we diagnose, make decisions, use therapies and how we think about outcomes. So this is a fantastic opportunity to learn as much as you like about a diverse range of topics and actually put that knowledge to use.
So what does this have to do with Melanoma and MPNE?
Nothing and everything really. MPNE got involved with the DLCTs via something of a coincidence. Finding options for those who have nothing left has always been our priority and we still loose to many of our community, in particular to Rare Melanomas. MPNE has always been about Science and how to teach scientific concepts, including WECAN Science.
We also get increasingly contacted by cancer patients out of options from other cancer communities for whom we can unfortunately do much less. The DLCTs are therefore a welcome opportunity to make a difference for all patients with advanced cancer, in particular those with solid tumours.
And MPNE 2023 is the next upcoming event, so rather than waiting for the next WECAN Science, we thought we simply invite all our colleagues from other cancer communities who are equally passionate about this topic!
The workshop will be facilitated by Andrew Evans.
Andrew Evans was diagnosed with choroidal melanoma in May of 2013. He holds an MS in Bioinformatics from The Johns Hopkins University and at the time, he was working as a bioinformatician, developing analysis pipelines for genetic diagnostics in cancer research (ironically enough).
Today, Andrew is the Chief Product Officer at one of Europe’s hottest seed-stage tech startups, Supernova.io. A serial entrepreneur, Andrew has spent his career in product - from leading the customer experience at CareerBuilder.com as a founding team member through IPO, to training global enterprises like Unilever, Philips and Friesland Campina to think like startups when developing new products - both digital and physical.
Andrew has served as a consultant, mentor and trainer in Lean Startup and Agile processes for large corporates as well as highly-regarded digital business programs through Lean Startup Machine (now Javelin), Hyper Island, Innoleaps and The Talent Institute.
Since his own diagnosis, Andrew has striven to help other patients
make sense of the complexities of their diagnoses, through active
participation on social media and by writing a blog that attempts to
explain cancer concepts in accessible terms.
He moved to The Netherlands in 2014, and subsequently became
actively involved with the Melanoma Patients Network Europe.
In his other life, Andrew is the lead singer for Birmingham Electric.
Before the workshop
To make optimal use of the time in Brussels, we plan 2 preparatory online sessions before the face-to-face event (work in progress, time and details tbc):
1- Introduction to DRUP and DLCTs
- Understanding the design behind DLCTs and the history of the DLCT community
Hans Gelderblom, coordinator PCM4EU, Kjetil Tasken, coordinator PRIME-ROSE (grant agreement in preparation, both tbc)
- Capture questions relevant to the patient advocacy community, so far
1- what do we do for patients for whom there is no actionable mutation? Link-up with further research?
2- strategy to increase therapeutic effectiveness?
3- how can we refer patients to these trials?
References- work in progress
2- Online workshop: stakeholder mapping (online, miro, introduction will be provided)
Precision medicine today is in its infancy. While the concept seems intuitive 'the right medicine to the right patient at the right time', the reality is far from it as we cannot find actionable information for every patient and therapies rarely lead to impressive, long-lasting results. We will therefore create a stakeholder map of those able to tackle these uncertainties as important stakeholders for the implementation and future direction of DLCTs.
The starting point will be the Series of Unfortunate Events in Precision Medicine that started as a post on LinkedIn and that has already created wide multi-stakeholder engagement. For those who attended Bootcamp21, the approach builds on the experience from our last attempt to map a patient-centric research ecosystem. The resulting map will serve as background for the case studies of the 28th April face-to-face event.
WHO SHOULD APPLY?
We are looking for motivated cancer patient advocates from any cancer type with interest in precision medicine, the determination to make a real difference for patients out of options and the willingness to explore new ways to collaborate with the research community. This is for people who want to put their knowledge and abililities to the test- and not everything will work out, so we expect people to be comfortable with experimentation and learning. This is about getting it right, not about being right. And there will be lots of tools, resources, help and support available- we only expect you to make use of it!
HOW DO WE SELECT?
Participation is upon application only. While relevant patient advocacy experience and a scientific background are a plus, we are first and foremost looking for motivation and the will to make a difference.
WHAT DOES IT COST?
Accepted participants will receive free registration and full-board accommodation for 1 night (Thu, 27th April) of the meeting. Participants are invited to attend as much of MPNE2023 as they like, fully acknowledging that this is obviously a Melanoma conference, just let us know and we find a solution. And as a patient organisation, we are obviously always grateful if you find alternative funding sources, too.
Travel needs to be arranged for by participants themselves but will be reimbursed up to the maximum amount of €380 for economy/ 2nd class travel after full attendance of the conference. Please look for the best deals and keep the original receipts of your bookings.
Cannot afford to pay ticket upfront? Please contact us.
No attendance, no receipt = no reimbursement.