In the complex machine of modern healthcare, until recently the patient has been a small cog. In this special in-depth feature, we ask stakeholders from across healthcare to share their insights on how engaging patients is transforming the industry

There is a wind blowing through the pharmaceutical industry, a force with the power to fundamentally transform every function, every stage of the pipeline and every marketed product. This force is driven neither by cost-containment nor regulatory pressure, it is neither a clever gimmick dreamt up by a management consultant nor a knee-jerk reaction to a market dynamic. What's more, to an outsider, it looks like the industry has taken a long time to wake up to its potential.

This force is the patient, of course, the end-user of our medicines, the person who has, in Europe at least, been far from the forefront of pharma thinking. Until now. Born from demographic shifts, incubated by increased transparency and nurtured by the internet, companies are only now starting to realise the incredible transformative power of their insights and, increasingly, their advocacy.

In March, the industry gathered in Barcelona for the annual eyeforpharma conference, spending three days discussing the issue of patient-centricity – and barely scratched the surface. So, what is patient-centricity and how can engaging patients benefit pharma? In what ways can companies use patient insight to deliver better products and services? How can they engage patients without falling foul of regulatory agencies? Most importantly, what approaches work and what don't?

"Patient-centricity has moved from a buzzword to a very sharp reality for the healthcare industry," says Martine Leroy, head of insight at creative marketing consultancy Blue Latitude Health. "There is a realisation that unless they embrace patients, companies will face ever higher hurdles to get their products to market. However, the healthcare sector is not driving the change and pharma companies are not leading the way; they are reacting to events, creating new roles and are trying to innovate. They are spotting opportunities and pursuing them."

Some companies are reticent to fully commit to the transformation, says Paul Wicks, vice-president of innovation at data-sharing platform, PatientsLikeMe. "Change is difficult and a lot of people have become very successful in pharma by turning the crank of a machine they know and understand. Why would they stop turning the crank? We're saying they should stop the machine, take out a few gears and reconfigure the inner workings before putting it all back together again. Why should they do that and lose precious time in moving programmes down the line?"

Another barrier is when companies already think they are patient-centric. "When I work with a pharma client, I look through their annual report to count how many pages I have to read before the word 'patient' is mentioned; my record is 26 pages," says Wicks. "For many, patient-centricity has been nothing but tokenism. I have visited companies where the receptionist saw my name badge and asked if I was one of those patients who gives them a speech once a year and tells them what a great job they're doing. That is some people's experience of  'the patient', an inspiring talk or a poster in the hallway of 'Susan, 36, living with cancer'. We have to go past this level of tokenism to find out what the real opportunities are."

He points to the tech industry. "In tech, there is a tradition of open source and transparency; a lot of senior people have Twitter or Facebook accounts or they blog about their work, even their failures. Can you imagine anyone in pharma doing that? In tech, they agonise over whether they have delighted their customers but the pharma industry is optimised towards prescribers, payers, HTA bodies and regulators. Even though it is patients that use their products every day, industry has been slow to wake up to the fact that patients are their real customers."

Guy Yeoman, vice-president of patient-centricity at AstraZeneca, agrees. "As an industry we are very much focused on delivering for patients when we really need to start delivering with patients. It is not a difficult concept but in reality it's quite difficult to do from a behavioural perspective. Historically, we have gone to HCPs to get the patient perspective, we ask what doctors think patients need and think, and that's not good enough now.

"There are some great examples of best practice [but] as an industry there is a lot we're doing that doesn't really work because it doesn't address underlying patient need. We don't engage patients as we should in our research agenda, we don't ask them about their experiences in a study so we sometimes struggle to recruit and retain them. There are big challenges in the R&D space and there are big challenges in the patient services space. Historically, we have been very focused on our own brand; we generate brand-specific patient websites at a global level and replicated them at a country level, we generate adherence programmes that focus on our brand, sending reminders to take our specific medicine. These all fail to address fundamental patient need and as a result patients don't engage with them," he says.

What do patients want? 

As companies venture into this new space, the 64-thousand-dollar question is, what do patients want? For Andrew Schorr, two-time cancer survivor and president/founder of cancer patient community Patient Power, the answer is clear.

"Patients want transparency. Help us to understand what's working and what isn't. Share results. Give us hope for a cure or a better medicine. Treat patients taking part in a clinical trial as investors; they are investing their bodies in your trial so keep them informed about that investment. They also want to be thanked afterwards. These are basic things," he says.

"Patients want a 360-degree view of their condition so that they can get the best care for themselves. Patients want what you get in journalism – news, perspective, context, with all information up to date and credible. If you're a sports fan you can access a huge range of information on your team, game stats, player news, injury reports. If someone is living and breathing their illness, they want that same deep understanding and they want the confidence they have the right team," says Schorr.

Pharma alone cannot deliver everything that patients want, he says. "Pharma can't do everything but it can do more. Patients are not necessarily anti-pharma but until recently the industry has not been sitting at the same table in any meaningful way. Traditionally, the grants that companies give to patient organisations are very small when you consider the money involved in their enterprises. They are not enough to do the big things that are
truly needed."

Yet, Schorr is encouraged by the moves by pharma to become more patient-centric. "It is clear that industry is engaging in a much bigger way now. Clearly some lightbulbs have come on inside companies as they realise, gosh, we'd better talk to patients to better understand their needs. But is it just talk? At the eyeforpharma meeting recently, I got the feeling that many of the senior people attending were lone wolves, voices in the darkness that were not yet part of the culture of their companies. Their challenges are around resources and creating a clear vision of the real human beings who benefit from medicines rather than numbers on a spreadsheet or an abstract concept."

As with all fundamental changes that sweep an industry, patient-centricity has left many scratching their heads about how to proceed, says PatientsLikeMe's Wicks, but he has some advice. "There's a great academic framework called Arnstein's Ladder that was developed in the 1960s. At the bottom is non-participation, even manipulation, then it rises to tokenism, where participants have a seat at the table but their input can be ignored by others. Higher up the ladder is true partnership, where participants not only have a seat at the table but they vote on issues and even help to choose who else sits on the committee and what resources are allocated. Their opinions matter. At the very top is citizen ownership, where participants make decisions and have the power, which a pretty radical model that industry might not be ready for. Pharma is probably more comfortable aiming for true partnership."

Yet it will take time to build meaningful patient-centric relationships, says Patient Power's Schorr. "The trust just isn't there at the moment and winning it is a big challenge. If you follow the US political chaos taking place right now, you'll hear Democratic candidates talking about pharmaceutical companies a lot. They say they need to be reined in, which in some cases is very unfair as there are many other quarters where costs need to be controlled. But pharma is portrayed as the bad guy, and examples like Turing give the impression that all companies are the same, which is certainly not true."

His advice to companies is to let go. "Companies that are serious about this agenda should aim to support the efforts of others and give up control, which means their agencies and PR firms and internal departments support, suggest and have a say, and those of us on the patient side have to listen. Companies with the leading science will win out in the end but only if they stop deciding that they think they know what patients want. So many attempts to develop relationships have used gimmicks, asking patients to sign up for and disclose their information, and these gimmicks are just sad. They are not a sustained effort to educate, support and empower communities, and patients recognise that."

For Schorr and other patient advocates, the long-term solution lies in therapy area collaborations. "It is almost impossible for an individual company to deliver the 360-degree view that patients want; they need to partner. Some companies are starting to understand that a therapy area strategy is needed where they collaborate with the other guys as well as healthcare organisations, governments, charitable foundations."

One company that is keen to engage in this way is AstraZeneca, says Yeoman. "We've only just begun the patient-centricity journey but we need to work together across the healthcare sector. There are several public private initiatives helping to define the process of patient engagement in R&D, and that will drive a collaborative approach. Personally, I am really keen for AZ to engage more particularly in patient services, with device companies, for example, where we can pool our combined expertise and resources so that when we develop a brilliant service we can scale it up much more efficiently than an individual company on its own. This is a challenge for industry, a tall ask, really ambitious."

Transparency across healthcare is essential, says David Loew, COO and head of global commercial operations at Sanofi Pasteur. "In the US, and in emerging markets, discussions [with regulators] are very transparent. For example, the Advisory Committee on Immunization Practices (ACIP), which recommends who gets vaccinated in the US, is completely transparent – you can watch discussion in person at the Centers for Disease Control in Atlanta or over the internet. Everyone can make their points – patients, industry, physicians, healthcare systems, payers – in an open, transparent discussion. Europe should learn something from the US system, making it so transparent that you take out the conspiracy that everybody just wants to make money, and actually have a constructive discussion. When you look at vaccination rates, for example in flu, they are massively higher in the US than in Europe."

The rise of the empowered patient has been much predicted over the last decade or more, and the impact of informed, educated and motivated patients is already being felt within healthcare, says Patient Power's Schorr. "There is a vanguard of patients that is hanging on every word and they are communicating what they know to other patients in many ways. For example, a lone activist patient walking into a doctor's office can accelerate practice change. The doctor may not be clued-in to recent developments and by advocating for a new therapy or participation in a clinical trial, the patient educates the doctor, which has a long tail with the less engaged patients sitting in the waiting room. These are patient opinion leaders; I might be one. We're out there and we want
your help."

It is these patients rather than patient advocacy groups that are leading the charge, he says. "I've been very frustrated at the slow pace of change with patient advocacy groups, who have been slow off the mark in some cases, for example, in digital. Gradually they are realising that they need to focus on what they do very well – advocating for patients – and should work with organisations who do digital very well. There also has to be more collaboration among patient groups as there are some very big groups out there that operate more like companies, with a CEO and brand directors, that don't feel they need to collaborate."

The importance of patient power is being recognised across healthcare, says PatientsLikeMe's Wicks. "The engaged patient movement is showing up in systems like the 'Patients Included' accreditation for conferences and journals. This mark demonstrates that patients have been truly consulted – they are on steering committees and they are speakers, and patients have been invited to attend free of charge or watch from home on a live stream. There is definitely a small number of highly articulate and motivated people pushing their way into the consciousness of industry. What is lacking is the pull factor from industry."

patient-centricity cog 

Truly transformative  

With merger mania and the trend towards in-licensing showing few signs of waning, pharmaceutical companies are becoming ever larger and more complex entities. So, how do you change the course of such juggernauts?

Changing culture is the key, says Lode Dewulf, vice-president and chief patient affairs officer at UCB. "How you organise a company is a strategic choice, which means there's never a perfect answer. What you have to realise is that culture eats strategy for breakfast; the organisation is just a tool, it is the culture that makes it happen, so if you don't fix the culture, you can forget it."

Looking across the industry, he sees two clear approaches being adopted by pharma. "Some companies go for a centralised way, bringing all patient engagement into one team that can set up, create contacts, capture everything and make sure it's all shared. It will be done very consistently but it takes away some of the power of product teams. Then there's the decentralised way; the main reason here is to avoid product teams saying, 'I don't need to care about patients because they do that and if I need something I'll ask them'."

UCB has tried to get the best of both worlds through an organisational principle Dewulf calls 'space with consistency'. "We want consistency in how we capture insights and share them but we also want to give space to every team to do what works best for them. It's not easy and we're on a learning journey."

For AZ's Yeoman, there is no one-size-fits-all model. "Every company is trying to work out how to do this properly, and there are various titles, centres of excellence and roles. What we've chosen to do is to create a small group tasked with enabling the organisation to become more patient-centric, ensuring we deliver patient-centric medicines and services quickly and efficiently. We're committed to shifting our organisations culturally on this journey. The final part is to start embedding it into our business processes, to ask what the patient-centric component is in a medical plan, or a development plan, or a marketing plan. What does it look like? It is a significant transformational programme and we're looking at three to five years to really start embedding it in our DNA."

GSK has opted for the decentralised approach, says chief medical officer, Murray Stewart. "We've deliberately decided not to centralise it to make everyone feel a part of it, to make every part of the business think about the patient. For me and GSK, making sure the patient is at the forefront of everything we do is business as usual. As CMO my role is to remind the business that we're developing drugs to make a difference to people's lives. I try to get people in discovery to consider patients when thinking about targets and people in discovery or in medical affairs looking at the business in late stage to think about the patient. It is quite a challenge in big organisations to get everyone motivated and to think about process."

Novartis started with a clear vision and definition of patient-centricity, says CEO of pharmaceuticals, David Epstein. "I took my executive team up a Swiss mountain where the air was thin and the sun was shining and they started to recount personal stories about their experience interacting with patients. It was totally unprompted but we spent the next day writing out what became the Novartis Patient Declaration, delineating our aspiration of what patients could expect from us."

The companies shared it with 60 patient organisations to get feedback and has since been using it to change culture and priorities, and to kick off projects, he says. "The declaration has had a lot of implications for the organisation in terms of being more inclusive, more innovative, more collaborative, speaking out more and taking risks in order to help patients. We rolled out a new set of values and behaviours last year and a lot of training, plus management is walking the talk. We're asking the questions, then teams are working on it, then we're celebrating when there's a success – that's how you change culture."

At LEO Pharma, CEO Gitte Aabo built patient-centricity into the 2020 strategy. "Our strategy, Helping Sarah, sends a signal to everybody in LEO that patients are individual people who live individual lives and have individual needs. It is crucial that we understand that if you have a chronic disease like psoriasis you don't think of yourself as a patient but as a person living with a skin disease, so we need to develop treatments that work in normal, everyday life. It may sound simple but the focus on the individual has a tremendous impact on how we act as a company; the better you understand the patient, the more likely that the business follows."

The support programmes LEO offers patients do not require them to be taking one of the company's medicines. "Our success criteria is to improve the quality of life of everybody with psoriasis; if we're really true to our mission of helping people achieve healthy skin, we must acknowledge that it takes more than treatment. If you listen to the CEO of Amazon talking about how they obsess about customers, it is the same philosophy I want to see in LEO; that we truly care about providing solutions to people living with skin diseases," says Aabo.

While a focus on patients has always been a fundamental motivator for Janssen, there is always more to be done, says company group chairman EMEA, Jane Griffiths. "We are incredibly patient-focused, it's in the first line of our Credo. Patients come first and they're at the centre of everything we do. Paul Janssen's mantra was that 'the patients are waiting'. However, if we reflect a little, we see that while we have the patient in mind we might not always understand exactly what they want. Do oncology patients always want to live longer or do they want a better quality of therapy? Patient outcomes aren't always the same as payer outcomes and we have to look at both when developing a drug."

There's a difference between thinking about patients and really having them in your heart, she says. "Ask yourself, do I talk to patients every step of the way? Historically, industry didn't interact with patients because it didn't want to be seen to be promoting to them but the rules of engagement are clearer now and there is better governance at patient advocacy groups, which opens up a more constructive dialogue.

"The more you can involve patients in their healthcare the better; that means involving them in protocol design about what outcomes are important to them, partnering to better understand disease and patient pathways, what's it like, for example, to be diagnosed with a condition, how many tests you have to have, how many times you have to visit the hospital or whether you are treated at home. All of these questions need to be asked, not only to give more patient-centric therapies but to reduce costs of healthcare delivery and seek different ways of delivering health," says Griffiths.

In formulating its approach, Boehringer Ingelheim developed a set of six principles – open, responsive, proud, empathy, commitment, and trust and respect – and has come up with a way of measuring itself against them, says head of corporate affairs Alan Sumner.

"We want to ask ourselves, 'If we were an open company, what would we be doing? We could score ourselves highly if a cross-section of people from within our organisation and people from outside were able to talk at length about recent and frequent examples where we have been open and receptive to the views of patients and those who care for them. The most valuable bit that comes out of patient-centricity is not to give ourselves an award saying we're great, but to come up with things that we can work on and need to improve," he says.

"I don't want to prejudge the score, but I would expect that out of six principles we're doing two fairly well, and there are four that we really need to up our game in if we do want to continue to talk about ourselves as being a patient-centric company."

While companies are charting their own course when itcomes to patient-centricity, are their efforts paying off? For UCB's Dewulf, the answer is clear. "We aim to double our impact on patient value by 2020 and, ultimately, we want to be patient-preferred, which is a very ambitious vision but a good north star to aim for. Are we successful? I would definitely say 'yes', but the deeper question is 'what are the metrics of success?'" he says.

"From our employee surveys it is very clear that our people are more motivated and better understand how to add value, and there's abundant research that says better employee motivation creates better results. We also have very good feedback from people who work with us as patients, for example, better feedback on our clinical studies. So, while I can't give a single hard parameter, we are very encouraged by the first signs. Interestingly, we have just launched a drug and, for the first time, we've not just looked at financial metrics for launch success but also added three patient metrics. It will be very exciting to see if there's a strong correlation. Maybe in the future all we need are the patient metrics."

True success can only be measured when patient insight is in conflict with company strategy, says PatientsLikeMe's Wicks. "A hard question for a company to ask itself is whether there is anything it is currently doing that it wouldn't be prepared to stop or change if patients really put their foot down. If you could tell me about a company that has made such a decision in the last 12 months – that they went back to the drawing board because of patient opinion – then that would be a significant change in the culture of the industry. I believe that in a few years' time, all companies will have case studies of when this has happened."

While many companies are seeing the benefits of engaging patients more intensively, there is a question mark around whether change is happening fast enough, says Wicks. "The danger is that patients could bypass industry entirely; for instance, patients are already hacking their continuous glucose monitors and insulin pumps and, in motor neurone disease we've seen large numbers of patient running their own virtual clinical trials.

"We've also seen groups of patients with very serious conditions deciding that clinical trials aren't working for them and so they enrol in clinical trials and systematically unblind themselves using social media to work out which group they are in. Some patients in trials are even sending off their pills or blood tests to a lab to figure out what's going on. If we're not careful we'll see groups of disenchanted patients going their own way. If you think it's hard to recruit to clinical trials now, it could get a lot harder if the patient thinks they're not being listened to or if a trial is simply there to bring a me-too to market to boost shareholder value. It is much smarter to be a leader in this space, rather than run the risk of a large group of key patients deciding that you're against them and not with them."

For Sanofi Pasteur's Loew, patient-centricity is a natural evolution. "It's the essence of what we do – we work to protect people from getting sick or to get fit again when they are sick. When you work in the pharma industry, you don't work for shareholders, you work for patients – they could be your friends, your families. I was faced with a situation where I was responsible for a cancer drug that both my mother and my mother-in-law were given. That's a life-changing moment, and it makes you really aware how important it is what you do in your daily work," he says.

Patient Power's Andrew Schorr is encouraged. "There are very sincere people in pharma trying to do things in a new way. Hopefully if the orders come down from the top that we're doing things in a different way and with patients in mind, then it can happen in a more expedient way. But the amounts companies are currently dedicating to advocacy relations is a pittance; they need to pass the hat around because all departments touch patients in one way or another."

Read part two of our special feature on patient-centricity.