TRANSCRIPT
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Gergely Meszaros:
Good evening and good afternoon to everyone. Welcome to this webinar introducing the Pulmonary Hypertension Expert Patient Academy (PHEPA). I’m delighted to present this new initiative. When ERN-LUNG, the European Reference Network for Rare Respiratory Diseases, was approached with this idea, I was immediately enthusiastic.
This is a brand-new initiative, and I believe it can be fruitful not only for the pulmonary hypertension community, but also as a pilot for other rare disease communities. As you know, pulmonary hypertension patients are very well organized. Without putting extra burden on your shoulders, I would like to underline that this initiative could become a model for other rare diseases.
With that, let me hand over to Eva Otter from PHA Europe for a few welcome words. Eva, the floor is yours.
Eva Otter:
Thank you very much, Gergely. As you mentioned, I’m also delighted that this project is a collaboration between PHA Europe (PHAE) and the Alliance for Pulmonary Hypertension (AfPH)—our first joint initiative. Patient involvement in development processes is particularly important because we have to give rare diseases a voice, and so we need empowered patients.
With this project, we are creating a foundation that enables many patients to find and use their voice. From my side, what is important is to speak in clear words that everyone can understand, so we can educate and empower people. Thank you very much.
Mélanie Gallant-Dewavrin:
Hello, everyone. I’m very happy, as Eva said, to welcome you to our webinar. I’m very enthusiastic about introducing you today to this new initiative. As Eva mentioned, it is the first joint initiative we are doing together, and it is also endorsed by ERN-LUNG, which we are very lucky to have. I also want to say that Prof. Marc Humbert plays a key role by accompanying us in this project.
When we all created our associations in the 1990s, I would have loved for such an academy to exist—this wasn’t the case, and we had to learn everything as it happened. Now we would like to help other patients and other associations with the experience we have gained. All of us are patients or patient carers, and we hope this project will help you in managing your associations and also managing your disease.
Prof. Marc Humbert:
Thank you, Mélanie. I’m going to share my screen. My name is Marc Humbert. I’m Chair of the French Pulmonary Hypertension Network (PulmoTension), and I will show you a few slides together with Pisana Ferrari, who leads the Alliance for Pulmonary Hypertension (AfPH). We will walk you through the rationale of the project. It is based on very simple ideas and concepts.
When we first met with patients in the 1990s, as Mélanie said, we wanted to understand the needs of patients. This was discussed extensively at the time of the World Symposium on Pulmonary Hypertension, especially in Barcelona, where the patient voice opened the symposium—and it is here to stay. We are not working on pulmonary hypertension for any other reason than to help patients, their relatives, and their supporters.
What do patients need? First, expertise—from expert provider teams and pulmonary hypertension reference centres. In France, pulmonary hypertension centres were established more than 20 years ago, with strong support from the French Ministry of Health. At the European level, ERN-LUNG was created in 2017, with a core network on pulmonary hypertension, and today I represent ERN-LUNG together with Gergely.
Second, access to proper diagnostic modalities and tools everywhere. Through guidelines, we try to inform the community about the right approach to diagnose pulmonary hypertension and classify it in order to manage it. Third, access to medications and interventions. There are many barriers and hurdles, but worldwide, access to medicines and interventions remains a priority for patients, and we need to support that.
Fourth, multiprofessional support. We need not only doctors; we also need researchers and healthcare providers—nurses, physiotherapists, palliative care teams, support groups, and, of course, patient associations. Patient associations should not be passive partners. Over the past 30 years we have worked together on how to build a strong patient voice and empower patient associations.
A patient-centred approach and patient-reported outcome measures (PROMs) are very important, and we also need the expertise of patients to inform the field. On the next slide, I will briefly show you the current status of the French PulmoTension network. You don’t need to know anything about France—just note that our goal is to ensure access to expertise close to home.
On the map, the national reference centre is indicated in the middle, with centres of high expertise across the country and additional expert centres nationwide, including overseas territories such as Réunion Island in the Indian Ocean and Fort-de-France in the Caribbean. The great idea is that knowledge travels, not the patients, and patients should have access to expertise close to home.
Now, ERN-LUNG—the European Reference Network on rare respiratory diseases—has the mission to ensure and promote excellence in care and research for the benefit of patients affected by rare respiratory diseases. Pulmonary hypertension is a prominent area within ERN-LUNG. We do not want ERN-LUNG to be a ‘black hole’ attracting everything, but rather a knowledge hub that reaches the territories and the many countries.
You can see on the map that we are spreading to the east and to the south. Our strong mission is that knowledge travels, not the patients—although when needed, patients may travel based on real necessity. We have had European guidelines since 2004. Initially, patients were partners but not active players in guideline development.
In the 2022 ESC/ERS guidelines, I was honoured to be the writing committee lead, and we involved two patient representatives who are with us today: Pisana Ferrari and Gergely Meszaros. For the first time ever, patient representatives were included, setting an important precedent for the future.
Patient and clinical expertise are complementary, and the expertise provided by patients is extremely important for rare disease innovation and development.
We also published, together with the patient representatives, a lay summary of the guidelines—not only in English, but in 11 European languages—to disseminate knowledge in accessible language. Now, what we want is to place patient partners and patient associations at the centre.
With this Pulmonary Hypertension Expert Patient Academy (PHEPA), we aim to strengthen the role of patients in the development and dissemination of knowledge. I will now hand over to Pisana Ferrari to give you more information about how this partnership was built—with AfPH, with PHA Europe, and with national patient associations across Europe.
And I look forward to the next stage of the discussion today.
Pisana Ferrari:
Thank you very much, Marc, for setting the context for how we arrived here today to present PHEPA. You’ve clearly shown how we have moved from ‘medicine for patients’ to ‘medicine with patients’—the patient partnership revolution. This is now recognised in the pulmonary hypertension guidelines, where I had the honour to be part of the task force together with Gergely.
It has become evident that patients’ lived experience provides clinical insights that clinical data cannot capture, and that priorities may differ. There is increasing evidence that outcomes are better when patients actively shape care design. Over the past decades there has also been growing patient involvement across many organisations and institutions.
For example: EU institutions; scientific societies; European Reference Networks (ERNs), with European Patient Advisory Groups (ePAGs); regulatory authorities such as the EMA (and the FDA in the US); Health Technology Assessment (HTA) bodies; and the healthcare industry. The challenge is that effective participation in these technical environments requires specialised knowledge and skills.
These are the current organisations that offer training courses for patient advocates: EUPATI (European Patients’ Academy on Therapeutic Innovation)—we have two EUPATI fellows here today, Hall and Louise; EPAP (European Patient Ambassador Programme); and the EURORDIS Open Academy (including the Summer School for Patient Advocates). But what is missing is pulmonary hypertension-specific training.
This is the gap we want to fill: pulmonary hypertension advocates who feel confident to contribute professionally in technical environments.
Just a few words about PHEPA: it is a collaborative effort with a comprehensive curriculum; it fills a clear education gap; it is written by patients for patients in simple, understandable language; and it will be reviewed by international experts, including the scientific committees of PHA Europe and the Alliance for Pulmonary Hypertension.
It is practical and actionable, and it will be free and accessible. Gergely will speak later about the different opportunities for patient engagement. With that, I would like to hand over to Hall Skaara, who will give an overview of the modules and what they contain.
Hall Skaara:
Thank you so much, Pisana. So I will be sharing my screen. So what we did when we were thinking about developing this course was we first we sat down to figure out what we wanted to include in the course. So we wanted to have more than just studying of pulmonary hypertension as the disease and medication and so on. We wanted to have additional information that would be useful for a pulmonary hypertension patient advocate.
So we came up with 13 different modules, and then we split the task between us on writing these different modules based on, among others, the skills we had. And we have finished quite a few of these, at least finished to the point where we have a very functional good draft, and then we will continue developing the ones missing here, plus we will kind of glue everything together.
So I will just very shortly go through each module.
And the first module we’ll have is a module about the background of pulmonary hypertension and our understanding of the pulmonary hypertension. So we’ll look into the first cases and how some forms of pulmonary hypertension wer recognized as a disease. Then comes the discoveries of prostacyclin and nitric oxide and so on. The right heart catheterization was used as the gold standard to diagnose the disease. We will also talk about the different landmark clinical trials that shaped the treatment, and then how all of this, of course, has moved the science forward, and now we have a much better survival rates than we had in beginning.
The next module focuses on the patient association landscape: how it started, and how national associations have developed into global networks. We discuss how patient associations are increasingly helping to shape healthcare policy, as well as the challenges and opportunities this creates. We also cover practical aspects such as legal frameworks, statutes, boards, compliance issues, fundraising, strategy, and communication with PH stakeholders and patients (including via websites and social media). A patient association can complement clinical care: going to the doctor is one thing, but once you leave the doctor’s office, an association can support you with practical information, emotional support, and education—like the academy we are developing now.
In module 4, we look at the stakeholder landscape: EU institutions that a pulmonary hypertension advocate should know about; regulatory authorities such as the EMA in Europe and the FDA in the US; scientific societies and networks (ESC, ERS, etc.); public health NGOs (EURORDIS, the European Patient Forum, PVRI, etc.); and, of course, the healthcare industry.
In module 5, we start to look into the disease itself: the causes of pulmonary hypertension and the mechanisms behind the disease, and the different pulmonary hypertension groups (classification 1 through 5) and WHO functional class. We are very grateful for the support of Prof. Marc Humbert and other experts who can verify that what we write is scientifically sound. We also look into the different patient populations across pulmonary hypertension groups.
Once we have talked about the groups, we will talk about pulmonary hypertension treatment. And as you will know, we have several pathways. We had three, and now we had one more, and there are new therapies coming up and emerging treatments that we will discuss. And for one of the groups, then also there is a surgical option that will be talked about. And of course, we’ll talk about how the treatment of pulmonary hypertension depends on what kind of type of pulmonary hypertension you have. So for some patients, a given pulmonary hypertension therapy would be totally wrong, and for another type it will be just the right type. So this is an important topic.
Then, in Module 7, we look at patient-reported outcome measures (PROMs). This focuses on how the patient feels, which is very important, and how PROMs can be used—for example for shared decision-making with the doctor when deciding on treatment. PROMs are also used in clinical trials, often as secondary endpoints. They can be very helpful in discussions with your doctor and can contribute to improving quality of care.
Then, in Module 8, we look into key reference documents and scientific literacy, including the guidelines Marc mentioned (first published in 2004, with the latest in 2022). In module 8, we cover key reference documents and scientific literacy. We discuss guidelines (first published in 2004, with the latest in 2022) and how to read and interpret scientific literature. Increasingly, when pharmaceutical companies conduct research and trials, they also provide lay summaries, making it easier for patients to follow new evidence.
We also discuss PH registries: there is a large registry in France, and registries in many other countries. Some registries are combined—the PVRI has one called GoDeep, which brings together data from different registries worldwide. With larger datasets, we can do more research and better understand outcomes and treatment effects.
In module 9, we discuss clinical trials, including how the different phases work (Phase 1 through Phase 4). We will also talk about ethics and informed consent that are very important when conducting clinical trials. And we will talk about how once the patients are more empowered, how they can also play a role in research, not only as a participant in a trial, but as a participant in, for instance, they’re a committee of the trial to help shape and develop a trial to give a patient voice to talk about which endpoints should be included and so on. So more and more patients are involved in these kind of things.
In module 10, we will talk about access to medication and treatment. So we have the regulatory approvals, EMA, FDA, and once it’s approved on this level, then we have the health technology assessment that needs to be performed in every country in order to get the drug to the market. And all this will be covered in module 10.
Module 11, we’ll talk about a new emerging concept, telemedicine and artificial intelligence. It’s going to play more and more important role in the future, and we will talk about how telemedicine can improve the care, talk about remote monitoring that, of course, during COVID-19 became more pronounced than before. And artificial intelligence will be used more and more, not only in helping writing, as we all know, and probably all use, but also help diagnose and help with risk assessment and so on. But of course, the challenge will be privacy and data protection issues.
In module 12, we cover legal and ethical issues: how to know and protect patients’ rights; privacy and confidentiality; and managing conflicts of interest.
Module 13 is an addendum with a glossary of key terms, a list of acronyms, and recommended further resources (including webinars from the Alliance for pulmonary hypertension and PHA Europe). So these are the 13 modules we are planning to provide. Thank you.
Louise Bouman:
Good evening, everyone. Today I have the honour to briefly show you how a module is structured, using Module 7 as an example. Remember: this is not about the detailed content, but about how the learning experience is designed. This is a draft version of Module 7 and it still needs to be reviewed by our scientific committee, so it is not final yet. We want to give you a preview: each module is built to be clear, accessible, and practical.
Let me briefly tell you how this looks in practice. Each module always starts with an estimated reading time and clear learning objectives that immediately give participants clarity: how much time do I need, and what will I actually learn? Then the content is divided into clear sections with structured headings, making it easy to follow and easy to revisit later.
Every module concludes with references and additional information. For those who want to go deeper on the scientific side, the sources are there—but they don’t overload the main text. So the structure is consistent, predictable, and very practical. Next slide, please. I want to show you how the module itself looks, so you can see how it feels in practice.
You can see at the beginning the estimated reading time and the learning objectives for PROMs and quality indicators. Every heading is very clear and the content flows. It’s not too dense and not too academic—easy to read, with bullet points, highlighted key messages, and some figures. This is just a sneak peek.
You see also patient-related things. What is practical for a patient to know? Different things, how to use it, where to use it, what kind of things you have, what do you measure? What do you want to measure? What can you measure? When do you need it? It looks like a lot, but if you read it, it’s not too difficult or too intense. And it’s really written from the patient’s perspective.
So everybody can read it and everybody can understand what it’s about. And here’s an example of how this is a simple figure to just show us different kinds of PROMs that are available and are used in pulmonary hypertension and with the pros and the cons and which ones are used where.
So that’s just an easy overview of handy things, I would say.
At the end of the module, you see the references—we use scientific materials and publications. You can also see additional resources, not only from the Alliance for Pulmonary Hypertension or PHA Europe, but also from sources such as EUPATI and relevant videos on YouTube. Some people learn more from watching videos than from reading, so we include different types of resources. We hope to do this for every module.
So every module is built around three principles: clarity (easy to understand), consistency (easy to navigate), and credibility (based on science).
So it’s also based on science. And module seven is simply one example of this approach. And together the modules create a structured pathway toward becoming an expert patient. And that’s what we want. We want more patients to be able to do more than just being a patient and well, to make the PH world a little bit better from the patient’s perspective. Thank you for your time.
Gergely Meszaros:
You’ve already heard a lot about this academy—from the module overview and from Louise’s walk-through of how we approach learning design. You might now ask: who are we addressing with this expert academy? We try to be as inclusive as possible. Primarily, we are focusing on people living with pulmonary hypertension, but of course caregivers, relatives, and loved ones may also benefit. Friends, schoolmates, and co-workers of a patient might also be interested, and we do not want to limit participation. In general, anyone committed to the cause of pulmonary hypertension is welcome—whether they want to better understand the disease and life with pulmonary hypertension, or whether they want to help a specific patient, their family, or the wider pulmonary hypertension community.
And last but not least, and we truly hope that many of the patient experts who are attending our academy would like to help a specific patient or his or her family, or in general, the pulmonary hypertension community.
As mentioned, we built this academy under the umbrella of ERN-LUNG (European Reference Network for Rare Respiratory Diseases). This initiative does not come from a vacuum: ERN-LUNG already has a successful Academy for physicians, and plans are in progress for nurses and other allied health professionals. As you can see in the graphic, the patient expert academy sits at the heart of the ERN-LUNG education landscape.
We have been considering different formats for modules and learning materials. Currently, ERN-LUNG has an online ‘Academy’ webpage where these materials can be hosted and browsed, and we are also considering other platforms to support dissemination.
We aim to be as inclusive as possible, while making sure that the materials are shared in appropriate contexts and remain easy to navigate and up to date.
You may ask whether there will be ways to measure whether participants benefit from the course. We are considering assessments, not only to evaluate participants’ understanding, but also to evaluate how well our materials and methods work—so it is a two-way evaluation. We also want to understand whether we are reaching the right audience and whether the targeted population is appropriate.
There are many possible assessment methods. The easiest is an online multiple-choice question (MCQ) test. However, there are more sophisticated approaches. While we trust participants, we also want to ensure that all modules are well understood.
Other options could include remote proctoring (secure online testing solutions offered by specialised companies) or in-person testing (which can be ideal from a security standpoint but may be expensive).
In addition, there are alternative assessment methods that naturally discourage cheating. For example: case-study analysis (working through real pulmonary hypertension scenarios to demonstrate applied knowledge), problem-solving exercises, portfolio-based assessment (written work, presentations, practical demonstrations), or oral examinations via video call with multiple assessors.
For physicians, CME credits are based on a very rigid methodology. The European Board for Accreditation in Pneumology (EBAP) reviews modules to ensure they are appropriate and up to date, and EBAP also provides guidance on how multiple-choice questions should be developed.
The takeaway for our work is: what we deliver should be relevant, user-friendly, and focused on important content. We should decide whether we aim to test applied knowledge rather than purely theoretical facts, and we want to deliver knowledge that can be used in real life.
It is also important to find an appropriate level of difficulty. As Louise mentioned, questions should be concise, phrased in simple language, and avoid double negatives. We should also avoid unintentional cues that help guessing, so that we can genuinely assess how well participants understand the materials.
With that, you have a better overview of our programme, and I would like to open the meeting for discussion and questions.
Prof. Marc Humbert:
Thank you very much, Gergely. Maybe Pisana and myself and yourself, we can animate. I don’t see any question in the chat, so maybe I will have one. It’s always good to have one question ready. So a question maybe to Pisana and Gergely. Of course, this is a very important task and Louise mentioned that it is a lot of work and you do that on top of your current burden.
So I would like to know if it will be a living document with updates and if you have already planned the regularity of updates and whether some aspects will be replaced by more updated information.
Pisana Ferrari:
Well, pulmonary hypertension is a very rapidly evolving field, so it’s absolutely essential to keep up to date. You’ve seen just in the last few months how many new things have come up. So the answer is yes, it will be regularly updated to take into account any of the most recent developments.
I would like to say also because maybe it hasn’t been said that we’re all volunteers, we’re all doing this because we’re very committed to the pulmonary hypertension cause and we really believe this project to be valuable and important, and we plan to continue working on it. I don’t think we’ve spoken about a timeline, but we are kind of hoping to finish by the end of the year. It might be ambitious with the revisions, but we’ll see.
Prof. Marc Humbert:
Congratulations. Maybe another question from a doctor to patients: I see people speaking German at home, speaking Hungarian, speaking Dutch, speaking French—many languages. So I would like to know whether this material will be available in different languages.
Pisana Ferrari:
. Yes, absolutely. And actually, we’re lucky that artificial intelligence has improved enormously, so translations are actually quite good these days. So with minimal post-editing, I think we can start with an AI translation and post-edit it and have it verified. I think that’s this is. a problem that would’ve been very serious not so long ago, but we are now very lucky to have this opportunity.
Gergely Meszaros:
Yes, and on top of that: we want to be as inclusive as possible. In many countries there are advocacy activities where we need expert patients communicating in their local language—for example around access to medicines or Health Technology Assessment, which will become more coordinated at EU level due to new EU regulations. So we believe we should not exclude people who do not have proficiency in English, but rather try to reach as many patient advocates as possible. There is a lot of work ahead, and we need many more experts—new generations are coming. So it is really high time for us to be prepared and have answers for these questions.
Prof. Marc Humbert:
Excellent. I see a question in the chat. I’ll read it: will there be a form of accreditation—such as a certificate—so that participants who complete the course can show proof of successful completion? This connects to what Gergely just said about expert patients being invited to speak with regulatory bodies in many countries.
Hall Skaara:
I can say something about this. We have not finally decided how to handle it. Gergely talked about exams and assessments, but I would like to stress that you do not need to take an exam—people can use the materials for their own learning. However, our clear aim is to offer a certificate for those who want to study more formally. We would like to set a high bar, so that obtaining a certificate indicates a meaningful level of knowledge that will be useful for patient advocates.
Gergely Meszaros:
Thank you. I would like to add that there are big differences between certification and accreditation. It may be difficult as a first step to aim for formal accreditation of this course, but in the long run we should not exclude it. At the beginning, a certificate is likely a realistic short-term goal. And because this is a pilot, it could also be followed by similar initiatives for other rare lung diseases.
Prof. Marc Humbert:
A comment from me as member and vice-coordinator of ERN-LUNG: we have a similar academy for doctors, especially young doctors, and I am always very happy to welcome doctors from all over Europe (and beyond) for training. For doctors there are short videos created by young colleagues from the Netherlands, France, etc., and then participants can come for a one-week training visit.
We recently hosted a young doctor from Ukraine, and today I met a doctor from Ireland who wants to come. Maybe one day we can also invite patient representatives for a similar experience—this is something we can push at ERN-LUNG level. ERN-LUNG is patient-centric, and we want to serve the community.
I’ve just read a nice comment from Seth: congratulations on all the hard work you have done—very exciting things to come. Maybe Eva and Mélanie can also say something. Mélanie, from a French perspective: the French don’t always feel comfortable in English. Will you make efforts to disseminate this for the French community?
Mélanie Gallant-Dewavrin:
Well, I think that now with new technologies and all the possibilities to broadcast things, to translate directly in another language, I think it can be more useful than it used to be. So I think that will be new solutions that will help us a lot.
Prof. Marc Humbert:
Thank you. And Eva, from a PHA Europe and Austrian perspective?
Eva Otter:
Yes, from an Austrian perspective, I agree with what Mélanie said: artificial intelligence an help a lot, and I use it as well. It can support translation—for example, for Module 7 you can translate it into your own language, which makes it much easier. Using artificial intelligence is a simple thing people can do. And I think that some patient representatives will want to complete the modules and take an exam to obtain a diploma or certificate. For them, doing it in English may also be possible.
Pisana Ferrari:
I’d like to make a comment based on a real-life situation. I have a brilliant member of our board in Italy who has a physics degree. She is a scientist, a patient, and incredibly knowledgeable. She could have an honorary degree in medicine. I suggested her for a conference in Rome, and they told me she needed to prove she was an expert patient—despite being on our board for years and having a scientific background. So I think it will become increasingly important to show that you have something tangible: a certificate for the moment, and maybe accreditation for the programme later. This could be very valuable.
Hall Skaara:
Could I add a comment? We sometimes see that we are treated just like ‘regular patients’. For instance, at the upcoming ERS Congress in Barcelona, patient representatives may not be allowed to enter the exhibition area or attend certain sessions because we are treated as patients. But there is a difference between a patient and a patient advocate/representative. A certificate like this could help in that respect.
Pisana Ferrari:
Very good point. Totally agree.
Prof. Marc Humbert:
Very good point indeed. Last year I was very pleased to be with Louise at ERS discussing both the burden of pulmonary hypertension and scientific facts, and Louise and others made very strong contributions. You are right: we need to put you in the right role. Regulators also have good ideas on how to manage influence and conflicts of interest. I see the clock running. Maybe Pisana can give a brief conclusion. For me, it’s a great honour to be with you. Over the last 30 years I have walked many paths with patient associations, and today feels like a milestone. Congratulations on what you do.
Pisana Ferrari:
Thank you very much. We are honoured to have you and to have the support of ERN-LUNG—it is absolutely fundamental. Thank you for your support over all these years; you have been a very vocal advocate for patient involvement, and we are very grateful. I would also like to thank Robert Plátika from admedicum, who has been behind the setup of this webinar today, and of course all the participants.
Prof. Marc Humbert:
Thank you. Have a good day.
Pisana Ferrari:
We forgot to say that tomorrow is Rare Disease Day, and we’re doing this webinar in honour of Rare Disease Day.
Prof. Marc Humbert:
Rare Disease Day is every day.
Pisana Ferrari:
True, true. Indeed. Bye-bye everybody. Bye-bye.