The patient advocates will see you now: Cancer groups trial the AIDS model for interacting with industry

Anna Wagstaff

It’s easy to say you are patient centred – until you are asked to spell out what that means in terms of your processes and practice. Last summer 11 patient groups invited nine pharmaceutical companies to join in a frank discussion about how to make ‘patient centred’ a reality. Anna Wagstaff reports on how it went.

“Most of us in this room will have participated in pharma advisory boards. They’re nice. You get to see faces you haven’t seen for a while – a bit like family reunions. But the topics covered are rarely the ones we feel address the main needs and wants of patients. And there is no follow up, so we never know what the outcomes are. That has to do with the fact that we do not set the agenda, and we are not the ones who send out the invitations.”

Ananda Plate, chief executive of Myeloma Patients Europe, was addressing around 100 European myeloma advocates, gathered at their 2018 AGM, to make the case for changing the way they interact with industry in favour of a model developed by the HIV/AIDS community.

The traditional pharma advisory boards she was referring to are meetings convened by companies, typically at the instigation of marketing departments, to help them with issues that crop up in developing a product, generating data, and creating educational and promotional materials.

The new approach she was advocating – community advisory boards, organised and driven by patient advocates – operate in the reverse direction. Their function remains to enable advocates to offer expert advice to companies. But their aim is to assist companies in their efforts to most effectively meet the needs of the patient community. It is the advocates who choose the topics on the agenda, and it is they who invite companies to send representatives with the expertise and authority to discuss how both sides can work better together to help achieve shared goals.

In June, Plate got her first taste of what that means in practice, as a key organiser and participant in the first ever haematology community advisory board (Hem-CAB), which took place in Stockholm, immediately after the European Hematology Association congress.

Formally convened by Myeloma Patients Europe, the meeting brought senior representatives from nine pharmaceutical companies active in haematology together with leading people from 11 advocacy networks, covering leukaemias, lymphomas, myelomas, and myelodysplastic syndrome as well as some non-malignant blood disorders. The companies contributed equally to cover the costs of the meeting.

The agenda was developed by the advocates and addressed three topics of especial concern to patient

  • Increasing patient engagement at all stages of industry research and development
  • Strengthening  the quality of evidence generated by patient organisations, and using it to improve industry decision-making
  • Legal compliance issues that hinder effective collaboration between patient organisations and industry.

Invited to the table were company representatives who had the authority to conduct meaningful discussions on these topics. They were offered the opportunity in advance to make suggestions for the agenda.

The discussions aimed at agreeing actions to try to move forward on the topics being addressed. To ensure all sides felt they could speak frankly, the details of the discussion remain confidential. The key point, however, is that they are minuted and act as a ‘to-do’ list for both sides, who will have to report back to the next CAB meeting on what they did to follow up on commitments made at the meeting, and what impact those actions had. A redacted set of minutes agreed by all sides are made publicly available to allow patient communities and the public to get a general idea of what was discussed and decided. These are précised here (see boxes), and can be read in full on the CML Advocates Network website.

The success of the initial Hem-CAB can only be judged in the light of what changes are implemented in practice as a consequence. But the fact that this CAB happened at all testifies to the growing maturity of patient advocacy. It also provided a valuable learning experience for people on both sides of the table, and offers a template that other cancer patient communities can adapt to their own needs.

Who was at the table?

Present at the Haematology Community Advisory Board on the patient side were the seven representatives elected to the patient advocacy group of Euro­BloodNet, the European Reference Network for rare haematological diseases, together with delegates from the 11 advocacy umbrella groups listed below:

ALAN, ‒ acute leukemia
CML Advocates Network, ‒ chronic myeloid leukaemia
CLL Advocates Network, ‒ chronic lymphocytic leukaemia
EFAPH, ‒ European Federation of Associations of Patients with Haemochromatosis
EHC, ‒ European Hemophilia Consortium
ITP Support Association, ‒ immune thrombocytopenia
International MDS Alliance/MDS UK, ‒ myelodysplastic syndrome
Lymphoma Coalition Europe, ‒ lymphoma
MPN Advocates Network, ‒ myeloproliferative neoplasms
Myeloma Patients Europe, ‒ myeloma
PNH European Alliance, ‒ paroxysmal nocturnal haemoglobinuria
Thalassaemia International Federation, ‒ thalassaemia

Present from the industry side, with two representatives each, were: Alexion, Celgene, Janssen, Jazz Pharmaceuticals, Novartis, Pfizer, Servier and Takeda. In addition, the meeting was supported by AMGEN who could not send a representative on that date.

Born from necessity

The CAB model was developed by HIV/AIDS advocates in the US, as part of their legendary battle to put the interests of the patient community at the centre of efforts to tackle the epidemic. European CABs (ECABs) have been run by the European AIDS Treatment Group (EATG) since the early 1990s.

That community-led approach remained confined to AIDS advocates until 2016, when, with help and encouragement from EATG activists, it was introduced into the cancer community by the CML Advocates Network (chronic myeloid leukaemia), which now convenes CML CABs twice a year.

CML advocates had been immersing themselves in the science and organisation of treatments and trials since STI-571 – later named Glivec – started offering them a lifeline nearly 20 years ago. They developed the concept of evidence-based advocacy and, for 16 years, have been meeting with clinicians and drug developers for catch-up sessions, while training new layers of advocates across Europe and beyond.

Other cancer advocacy groups have been watching and learning, but none can yet match what the CML advocacy community has built, as Plate readily admits.

“For quite some time we had followed the CABs that were out there in the HIV community and CML community, and we were quite keen to replicate this in myeloma, but were not sure whether we were ready yet from our community side, so we decided to watch and see what was happening,” she says.

As it turned out, the myeloma network did not have to go it alone. “Within the haematology and the cancer community there was a shift towards everyone trying to collaborate and avoid duplication, and join forces and so on. So we came up with this idea of having a cross- disease CAB.”

Within the haematology community, the catalyst was the establishment of EuroBloodNet, the European Reference Network for rare blood diseases, which under EU rules must have a patient advocacy group, which provides representatives to the Euro-BloodNet board. These seven advocates “formed the brain of the Hem-CAB,” says Plate.

A parallel move toward greater collaboration had been happening over the same period within the wider cancer advocacy movement. A group of 21 European/international cancer patient advocacy organisations joined forces as WECAN (Workgroup of European Cancer patient Advocacy Networks) to “collaborate, align and develop joint projects of the European cancer patient community towards all stakeholders, and to provide a resource for the participants and external organisations.”

One of their early projects involved engaging collectively with pharmaceutical companies to reduce the burden created by differing and often unfair requirements their legal departments impose regarding the terms on which their companies can support the work of patient groups (see WECAN and MPEurope).

These developments formed the backdrop to the Hem-CAB. “We felt we were a community strong enough to have a united voice and go for it. It was not possible before, because everyone was working in their own world and duplicating and doing everything that everyone else was doing and spending money in a crazy way for exactly the same things,” saysPlate.

“We wanted to run a CAB, we knew there were topics that were cross disease, that were not drug-specific, so no commercially sensitive stuff being discussed; no reason for companies not to speak openly. So we decided to give it a try.”

What they agreed: Patient engagement in R&D

Patient organisations said there are only a small number of documented cases where the input provided by the patient organisations has influenced decision-making in industry R&D, and they felt that the insights of patient experts and patient organisations could be included more systematically and effectively by industry researchers, e.g. in terms of defining research priorities and trial designs, as well as contributions to data safety monitoring boards and to clinical trial participants. One proposal discussed was the implementation of a ‘scorecard’ system that would allow patient organisations to monitor the patient involvement practices of companies, and would also allow companies to keep track of their own performance in this area. All agreed that a more structured approach to the input of the patients’ opinions was needed. Also, more involvement in the identification of needs was required so that these needs can be focused on subpopulations and groups. The patient groups said they want to be included in protocol review for phase II and III trials, but not necessarily phase Is. Data safety committees and investigator meetings should also include patient representatives. As a platform for collaboration, CABs by invitation of the patient organisations were seen to be one of the most effective platforms to bring in patient experts’ skills and knowledge, rather than any other affiliation with the company or otherwise.

It takes two to tango

CABs can only work if both sides see value in participating. And while companies are always happy to declare themselves to be patient-centred, not all welcome patient advocates having input into how they conduct their businesses, while others have simply been less exposed to this sort of engagement.

Plate sums up her perception of the range of experience on the industry side.

“You had three categories. The well-prepared – you could tell that these people had been at a CAB before, and came with clear positions and questions; others, who tried to get away with generic, non-specific statements, and discovered they couldn’t; and some who had no idea what they were getting into, and obviously felt quite uncomfortable not being in control of the agenda.”

The three companies – Takeda, Novartis and Janssen – that agreed to speak to Cancer World about what they got out of the meeting were among those with previous experience of the CAB model.

Multiple companies

This, however, was one of the first community-driven meetings they had attended that involved several companies all sitting in the same meeting. The opportunity this offered to hear what others are doing across the spectrum of haematological diseases was an important plus point, says Sanja Njegic, Head of Patient Affairs for these patient communities at Takeda. “It enabled us to engage with patients with different haematological malignancies, as well as with the companies who have a similar strong interest in the haematological malignancy setting. There was a huge opportunity to discuss big topics of mutual interest.”

“I think that is a unique aspect of this meeting,” agrees Louise Huneault, who holds a similar brief at Novartis. “Every company did their little eight-minute presentation, so it was interesting for us to see what other companies were doing and get some ideas of best practice that we may also wish to consider, moving forward. It was very enlightening from that perspective.”

A systematic dialogue

Daniel de Schryver, their counterpart at Janssen, was there primarily to present his positive experience of working with HIV/AIDS ECABs for the best part of two decades, and to encourage all the companies to embrace this approach.

The great thing about the CAB model, says De Schryver, is that it is not managed by the industry.

“The approach is the other way around, and therefore more systematic, by definition, because the agenda is put in a regular way by patient advocates, not depending on your own calendar, as a company. It is more regular, and in my opinion it is a good approach. The success stories are clear.”

“It’s the sustained conversations that are critical,” says Huneault. Her experience of the twice yearly CML CABs is that the companies and the patient groups commit themselves to a list of actions. “When we meet again we report back on the progress that we have made; the barriers that we’ve run into; other things that have cropped up that we need to discuss.

“This is why a critical success factor to this model is sustained and regular contact that has actions attached to it. Otherwise it’s just some nice talk.”

The CML model has delivered this, says Huneault, and she is confident that that Hem-CAB will do so too, especially as some concrete actions were suggested at the first meeting.

Common issues

Companies have a clear interest in getting feedback and advice in relation to their own specific products, but those discussions cannot be done within a multicompany meeting for reasons of commercial confidentiality. The strong attendance at this first Hem-CAB meeting indicated that companies could also see value in discussing common issues regarding how to better align the industry actions with what patient communities want.

“The dialogue was extremely constructive because the agenda items were very relevant and there was a very collaborative atmosphere,” says Takeda’s Njegic. The big issues, including patient engagement in research and development, access to care, and digital solutions, are cross-cutting topics that will be increasingly important in the future, she argues. “Only by moving together with other healthcare stakeholders in a systematic way can we start to move the needle. So CABs provide an important platform for figuring out how we can work together to tackle these issues and also come out with some viable solutions that are acceptable to all.”

For Janssen’s de Schryver, it’s a no brainer. The healthcare environment is focused increasingly on outcomes and on value, he says, so if you want to succeed, you need to be working with patients to understand what they value and how they rate outcomes.

“We are doing this because we believe it is the right thing to do, not from a sort of ethical, moral perspective, but because it will bring better solutions and provide better outcomes. We are convinced.”

A question of trust

Trust issues may also have been a factor companies weighed up in deciding the value of attending the meeting – did they trust the advocates to organise a constructive conversation, and would the meeting help build advocates’ trust in them? Novartis’s Huneault makes the point that pharmaceutical companies are used to having challenging conversations with payers and health technology assessors, but not with the patient community. “This is new. Challenging conversations with empowered patients who are very knowledgeable – patient opinion leaders – this is a different stakeholder.” Companies need to send representatives who are “able to manage what may be a challenging conversation and respond appropriately,” she adds.

It is also in the interests of the patient advocates to build industry confidence that the discussions will be constructive and in good faith. In this regard, the involvement of the CML Advocates Network paid off. Jan Geissler, a co-founder of the CML Advocates Network, organises the CML CABs and played a leading role in developing the Hem-CAB agenda and bringing companies on board. “There were no concerns from our side, because of knowing and trusting those who were organising the CAB,” says Takeda’s Njegic. She appreciated the efforts Geissler made to consult company delegates over the objectives and expected outcomes of the meeting.

If anything, the board meeting was a bit too polite, says Huneault. “In the one-to-one CABs we’re past the honey-moon stage. We’re now rolling up our sleeves and really getting down to the topics of true interest, and the patients are very persistent in not letting the sticky issues off the agenda. The conversation is very fruitful, very honest, very transparent.”

She hopes that Hem-CAB will go the same way, “so that we can get into the nitty gritty.”

What they agreed: Patient-generated evidence

Patient organisations wanted to know from the companies how they take advocacy-generated evidence into account, and what evidence industry needs from patient organisations to effectively influence their own decision making. They asked how patient organisations and industry can collaborate towards a more systematic methodology on the generation of patient evidence. The participants acknowledged the fact that patient communities generate different evidence from what companies or researchers may collect and generate, and their research has different motivations. This unique information needs to be incorporated into industry processes.

Also, a closer feedback loop is needed between industry, research and patient groups. Currently, patients contribute to research in many different ways, but find it difficult to access the results or outcomes. The CAB model was recognised as a feasible avenue towards better integration of all stakeholders’ work.

Several company representatives urged patient organisations to develop and improve their publishing practices. Indeed, patient organisations have not been systematic enough in publishing and disseminating their results and research findings, even if the quality of such research matches international scientific standards. Also, patient organisations believe that they can do as good a job as agencies hired by pharmaceutical companies to do the kind of research that collects patient needs and generates patient evidence.

An important proposal and agreement at the meeting concerned the possible establishment of a ‘research institute’ or ‘office’ by patient organisations, which could be responsible for the coordination and pooling of patient-generated evidence, its dissemination and authoring, and which could also control the ownership of data so generated. There was agreement that patient organisations should increase their capacities in this area by building better infrastructure/collaborative models in a way that allows for good data collection and proper analysis and publication in peer-reviewed journals for all stakeholders to use as a reference.

Did it work out?

“I thought they did pretty well,” says Tamas Bereczky, a veteran of the AIDS ECABs who had offered his advice and experience to the Hem-CAB and was present as minute taker.

“I enjoyed the honesty and the fact that company reps could sit at the same table and talk. I think that was a great achievement that hardly ever happens. Hem-CAB was able to successfully create this neutral space, which is what we always preach, where people can meet and talk about things that matter – about science and policy. I like the fact that the agenda was controlled by the community and they could keep to the point, and maintain a high-level conversation around issues that matter. I was very happy to see that.”

De Schryver, from Janssen, was also pleased with how the meeting went. “The patient advocates had really prepared their case. They were very united, they were organised, they were singing from the same songbook, and with a clear vision.

“And I felt there was willingness [on behalf of the companies] to say, ‘OK we are listening to you and these things can make sense.’ My takeaway was that most of them were convinced. Smaller companies said, ‘We don’t know yet, because we don’t know how we can do that.’ That’s a fine detail. I felt most companies were agreeing and saying ‘let’s find out how’.”

Sanja Njegic, from Takeda, believes that the collaborative atmosphere will have encouraged other company representatives, with less experience of working with patient advocates, to get more involved in similar initiatives.

“I enjoyed the honesty and the fact that company reps could sit at the same table and talk. I think that was a great achievement that hardly ever happens”

“I think it had a very positive snowball effect. Companies have a different organisational maturity when it comes to patient engagement – some of us have been working with patients for 10 years; others just came into the picture. For example, those who were not involved in the WECAN initiative on legal agreements indicated that they were very interested in being part of that.”

Within Takeda she has already started to work with legal colleagues on actions arising from the meeting. “We definitely started to work on the simplification of some of those legal contracts that we have with patient advocates.” She has also strengthened discussions with the company’s R&D team about the right way to involve patient advocates systematically, from a European perspective. The emphasis in CABs of involving people with key roles not just in patient affairs, but also the legal and medical side of the company, helps speed up internal processes, adds Njegic.

Huneault of Novartis also highlights progress with concrete actions. “We came up with some really good ideas together in terms of how we might tackle some of the issues that were brought forward in the Hem-CAB.” This includes the potential to use more patient-led evidence from such things as surveys and patient preference studies. “The haematology community are very advanced, and they are generating their own evidence to move the scientific conversation forward. We are very excited about this and were keen to have a discussion with the patient community about how we develop models where we can help build their capacity.”

A good start

Patient advocates were also positive about the meeting. Plate was proud of how the patient advocates performed. “There will always be patient advocates who are more quiet and shy, because they are not used to the setting. The moderator [an expert with experience from the European AIDS Treatment Group] tried to open them up, and shut up the ones who spoke too much. He did a good job.” Also important was the advocates’ pre-meeting the previous evening – another tip from the HIV community. “That was super helpful, because after that we were all very aligned.”

However, Plate adds a note of caution. “I was happy, but it will be easier to answer when we sit down at the next CAB and see what has changed. We had some very well prepared industry reps in the room. But we had others who seemed to think this was just a tick-box exercise. We don’t want someone just sitting there saying pretty words.”

She stresses, however, that both sides have responsibilities. “The whole burden shouldn’t just be on industry. On our side it is hard enough to get a group together that knows enough to talk in depth about everything and be able to put time into what we want to discuss.”

MPE is now planning to organise single-company myeloma CABs sometime in 2019, and has been piloting a patient advocacy training course.

Sophie Wintrich was at the Hem-CAB on behalf of patients with myelodysplastic syndrome. She has been chief executive of MDS UK Support Group for 10 years and is on the board of the (global) MDS Alliance. She is also adopting a ‘wait and see’ policy before pronouncing on what was actually achieved, but felt very positive about the way it went, and the possibilities it could open up.

Wintrich particularly appreciated being able to discuss issues that matter to her patient community with a group of senior company representatives who are tuned into the European regulatory environment and cultural attitudes. Her experience has been that, even when companies are headquartered in Europe, decisions on trial surveys or protocols are driven by US perspectives.

What they agreed: Compliance issues and legal challenges

The patient organisations asked each of the companies about: how to create an environment where a trusted and safe collaboration is possible without overburdening the patient groups with excessive regulation; what approach each company takes to strike an acceptable benefit/risk balance; and what the patient community can do, on its side, to reduce compliance hurdles for industry. Some companies have already started a process to streamline their internal processes and documents. Other companies have now been made aware of the challenges.

Addressing this issue has been the focus of the WECAN Project on Reasonable Legal Agreements (see WECAN and MPEurope), which was initiated by the patient community in oncology, working in collaboration with a workgroup of legal and compliance officers from multiple companies. Both sides at the Hem-CAB agreed that the WECAN project is in a unique position to identify the key interests of patient organisations and advocates that need to be protected when signing agreements with the pharmaceutical industry. Companies that are not yet involved in this project were urged to join.

The discussion also included some considerations around the fair market value of, and remuneration for, the work of patient experts and patient organisations. This is a particularly sensitive issue in some settings in Europe, which is further complicated by different industry rules and standards. This topic will be discussed further with the Hem-CAB stakeholders in future meetings, based on a proposal currently being worked out by WECAN.

One proposal concerned the setting up of a training course or capacity building resource from patients to companies to educate case workers and decision makers in different industry departments about the real-life applicability of legal and compliance processes in patient advocacy. Other actionable points from this part of the meeting included inviting legal and compliance persons to the next Hem-CAB meeting, putting together a catalogue of hurdles and challenges, developing contracting templates (WECAN is working on relevant guidelines), and organising a ‘roadshow’ by patient groups for companies to discuss current challenges.

“They tell us they’ve checked with patient advocacy groups, but they’ve checked with US groups, and the US groups are not as involved or advanced in terms of their ‘critical friend’ attitude towards industry. We advocates in the EU interact with industry in a slightly different way.”

The CAB offered a great opportunity to tackle tricky issues, she adds, such as the compliance contracts companies expect patient advocates to sign. “So that was an opportunity to put it on the table openly and realise we are all struggling with the same thing, and if we as patient groups give you the opportunity to say it, you may then feed that back internally to your colleagues and hopefully improve matters.”

“Above all”, says Wintrich, “it was a great learning environment, for both companies and advocates. Advocates could show companies that they expect the same best practices from everyone, while companies could voice their frustrations with the patient advocacy world.” It was also a welcome opportunity to learn from other advocates.

“So that was an opportunity to put it on the table openly and realise we are all struggling with the same thing”

Her one reservation is the extent to which discussions within the Hem-CAB filter into the wider consciousness and practice of the companies. “I’ve spoken to other people in some of the companies since, and they are not aware of the Hem-CAB.”

Wintrich is very interested in exploring the possibility of an MDS-only CAB, but needs time to build capacity among advocates. “We are still making baby steps in that regard,” she says. As she points out, the CML community has an advantage of being younger on average and less debilitated by disease.

The MDS Alliance will also need to consider the divide between the US and EU mentalities. “It may only work with European participants,” she says.
“I think the US ones would feel a little uncomfortable.”

For Michael Rynne, board member of the chronic lymphocytic leukaemia CLL Advocates Network, the big issue for patients  is access to prognostic tests prior to treatment as well as access to the newer drugs. This is a problem not only in his home country Ireland, but across much of Europe. Access issues were not on the agenda for the first Hem-CAB, and would probably need to be tackled company by company because each drug is different and there is a need for commercial confidentiality.

Nonetheless Rynne gained a huge amount from the Hem-CAB. “I learnt that even though there are differences in diseases, there are similarities in how we interact with pharma. What was interesting on a European level was that patients organisations can work together, looking at the same goals, whether pharma were there or not. When pharma were involved, we were able to ask questions, and I was asking on behalf of all the advocate organisations. And I thought that was a very good concept.”

Having access to more senior company representatives, who were able to answer questions and make commitments, made a big difference. “I’ve been involved with the CLL network since 2014. Fairly often we have phone conversations with local representatives. We often ask the companies about how it’s coming along, and they never give a straight answer, probably because they don’t know. We haven’t really entered that space where we can ask, ‘When is this drug going on compassionate access?’ That’s the space we want to be in. So we need to be able to talk to the senior people.”

Rynne believes the CLL Advocates Network will use what they learned from this experience to improve the quality of their interactions with industry. “What I came away with is that we now know that pharma want to work with us, so it is a question of how we can work together to make things run a lot easier.”

Highly recommended

Would these patient advocates recommend the CAB model to other cancer patient groups?

“Absolutely,” says MPE’s Plate. “It’s a way of making sure that the needs and wants of patients are addressed, and to put pressure on important topics in a more systematic way. This is a effective way of turning around the system and doing it from the patient point of view. It’s also a good way to train the community. I absolutely recommend it.”

“It’s the way to go,” agrees EATG’s Bereczky. Training and capacity building will be needed to develop layers of advocates who can adequately discuss trial protocols, biomarkers, quality of life measures and survey techniques. As he points out, patient advocates are driven by the powerful motive of wanting to stay alive. “Learning, self-education and self-empowerment are a brilliant way to cope.”

Is years of experience and expertise a requirement to get involved? Certainly not, says Bereczky. “This is a process. You cannot expect the patients to sit in these meetings, all of them, with the same standard of knowledge. It is just happening now, as we speak.”

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