Patient perspectives: Oli Rayner

Oli Rayner of the Cystic Fibrosis Trust shares his experience as a CF patient and discusses the myths that surround CF and the unmet medical needs of those with the disease.

Oli Rayner has cystic fibrosis (CF) and has recently joined the Cystic Fibrosis Trust in a six-month post as Special Adviser on Research and Patient Involvement.

Oli has been tasked with ensuring people with cystic fibrosis can find out what clinical trials are open to them, in addition to putting mechanisms in place to help increase the influence their views have in the commissioning and design of clinical studies.

He speaks with us here, in our latest 'patient perspectives' interview, on the needs of CF patients and the misconceptions that surround CF.

Interview summary

RA: Thank you for agreeing to take part in the interview. Could you please tell me about your background as a patient with cystic fibrosis and what has driven you to join the Cystic Fibrosis Trust, and lead the effort on patient engagement in clinical research?

OR: I was diagnosed with CF when I was three years old. The outlook was quite different in those days, and thanks to research things have changed significantly for children who are born with CF today.

There are over 1,800 mutations of CF, and even people with the same mutation can be affected in very different ways.

I've been quite lucky in that I've been able to do most of the things that I want to so far in life, things like sports, and school, and university, and travel, and work. As is common for people with CF, I've developed CF-related diabetes.

Now my CF is more demanding, and getting all the treatments done each day is essentially a full time job.

I've always been curious and interested in research; actually CF forces you to become a bit of a scientist and to think about life as a series of experiments. I've always felt the need to empower myself with knowledge so I could make informed decisions and find what works for me.

It's a very interesting time for cystic fibrosis, as new treatment approaches have the potential over the next five to 10 years perhaps to change what it means to have the disease.

In 2012, a new drug called Ivacaftor or Kalydeco came onto the market. For people with a certain mutation this drug is transformative, but that mutation only applies to 4% of the UK population. The clinical trial data was fairly spectacular, but it took a very long time for the NHS to agree to pay for the drug.

The process made me think about drug development and reimbursement. It made me realise that people making the decisions about how to test new medicines and whether to pay for them probably don't know very much about what it's like to live each day with CF.

Patients are the only ones with a 360 degree view who can sometimes join all the dots together.

People like me have a responsibility to speak up and communicate, and better quality decisions will be made, better outcomes achieved if more patients are involved, and I thought I could contribute to that.

I'm very happy to be taking on this new role for the trust and excited about what we can achieve. I've been inspired by what patient groups have achieved in Duchenne recently, almost forcing regulators and pharma to work together in a new, much more proactive collaborative way.

"...people making the decisions about how to test new medicines and whether to pay for them probably don't know very much about what it's like to live each day with CF..."

RA: Can you tell us about the projects currently being funded by the CF Trust?

OR: The Trust has stepped up its activities recently, and the new research director, Doctor Janet Allen, announced a five year research strategy earlier this year. This aims to achieve a balance between backing transformational science to correct basic defects, to restore CFTR function, and help alleviate and manage symptoms of CF.

The Trust is also committed to increasing the capacity and quality of CF clinical trials in the UK, recruiting and investing into CF research and enhancing the involvement of patients in shaping research.

There are a number of excellent projects which predate the new strategy that are ongoing.

The most recent projects are focused on infection prevention and control, CFTR's role in the development of CF-related diabetes, new treatment approaches for the most dangerous kinds of bugs with CF, a new treatment approach to correct the underlying defects in nonsense mutations of CF, gene therapy and gene editing that may address the underlying defects in a way that is not mutation specific.

The most important aspects of the new strategy are a commitment to work in a more open and collaborative way with other groups, academics, biotech, pharma, NHS, NIHR and other funding agencies, and of course people directly affected by CF. We know that when we work together we can actually make some really exciting things happen.

RA: What misunderstandings do you find that there are around cystic fibrosis?

OR: Well first of all you can't catch it – it's a genetic condition. People with CF can look very healthy, but be significantly disabled and very poorly. It's a strange disease to get your head around, it's very complex.

It affects different people in different ways, there are even cases of identical twins who are affected in very different ways.

It's a progressive disease, that gets worse with age, but the trajectory can vary enormously from patient to patient.

CF directly affects many organs and systems in the body including the lungs, liver, pancreas, intestines, immune system, reproductive system, and it also negatively impacts the way that organs interact with each other. Problems in the pancreas can create problems in the gut, and bugs in the gut can affect bugs in the lungs etc. It's difficult for people to understand.

Most people have permanent infections in their lungs, they need to do lots of treatment each day to keep the infections at bay. My own daily treatment regime includes around 40 pills, seven nebulisers, four inhalers, a saline nasal rinse, two injections, ambulatory oxygen, and two 30 minute sessions of physiotherapy or airway clearance. It takes about three hours if I'm feeling well, and it takes longer if I'm not feeling well, because it's tiring. It's difficult to fit everything into the day, especially if you're an adult living independently.

Psychologically, it can be challenging. One big challenge for the community is the fact that we're advised not to meet each other in person due to the risk of cross infection, which is something people don't understand. That can make people feel very isolated.

Social media and web based platforms are helping.

RA: What needs do you think cystic fibrosis patients have that are not currently being met, and what can the pharmaceutical industry do to change this?

OR: There's no cure for cystic fibrosis, and we all want to find a cure. Ivacaftor, Vertex's drug which is also known as Kalydeco, addresses the underlying genetic defect and significantly restores CFTR function, but it's not a cure.

We need to develop therapies to restore CFTR function for all mutations. We also need better treatment options to deal with the really dangerous bugs that are a threat to people with CF. They can significantly impair quality of life, and can be life shortening for people with cystic fibrosis.

The burden of treatment is fairly large for people with CF, so things which can make treatment go a bit quicker or easier could have a big impact on quality of life for people with CF and their families. It's helpful if treatments can be delivered in the most convenient way, so pills instead of nebulisers or inhalers instead of nebulisers. Lots of people are trying to develop apps or other things to help people take their medicines. I take medicines from 19 different companies every day, 19 different apps aren't going to help me. Adherence is fashionable at the moment, but there's a real danger that the relevant individuals in pharma could spend a lot of money producing a shiny new website or app that satisfies their boss but actually makes the situation worse for patients. There is a need for integrated solutions.

The CF Trust is setting up an initiative in this area, and we are hoping to engage pharma as partners in a central platform that will address some of these issues.

"Patients are the only ones with a 360 degree view..."

RA: How can patients with CF find out what clinical trials are open to them?

OR: They can ask their CF team at their specialist centre, or they can go onto the CF Trust website. They can also look at websites like the NIHR clinical trials gateway or NHS Choices.

We are finding that some of these third party clinical trial registers, including clinicaltrials.gov, are not always up to date. This can cause problems so it's important to tell people quickly when a centre opens.

One of my tasks is to make it easier for people to find up to date reliable information on clinical trials open to them.

One of the CF Trust's tasks is to make it easier for sponsors to set up trials in this country which is important on a number of levels.

RA: What considerations do you think need to be put into place when designing a trial for patients with CF?

OR: I would advise people to come and speak to the CF Trust, because there may be things we know about CF which can help you get where you want to faster.

There's a lot of scientific and clinical knowledge residing in the CF Trust, which is a big asset. The Trust is keen to put that to work to help people to develop better treatments.

The UK patient registry, which the Trust runs, holds information on the 10,000 plus people with CF in the UK. It's the only registry in the UK and is a valuable resource for researchers.

People with CF already have a heavy burden of treatment, so talking to patients or patient representatives about trials is quite important at an early stage.

Also, bear in mind that CF affects multiple organs, so for example if you're developing something to help the lungs think about potential implications for people who have CF-related diabetes.

Think about how to make the trial attractive from a recruitment perspective. Open label extensions are a very good idea because then participants know they will have a chance to try the new treatment if they are randomised to the placebo arm.

I would encourage pharma to think about whether it's possible to incorporate real-world data, and remote monitoring devices to reduce the necessity for clinical visits, but also just to capture how the drug is really affecting people as they go about their life. There is sometimes a gap between the clinical trial setting and the real life setting, and I think we need to think about how to close that.

Finally, on a practical issue, some patients need to travel relatively long distances to get to centres, so think about what you can do to make this easier. Generally to talk to patients at all points in the process, because they can be equally useful resource. We're all trying to achieve the same thing, we all want treatments that work, so let's work together.

"One big challenge for the community is the fact that we're advised not to meet each other in person due to the risk of cross infection..."

RA: Finally, if there was just one thing that you could say to the pharma industry what would it be?

OR: I would say invest in a clinical data package that provides patients, clinicians and payers with the data they really need to make sensible decisions rather than just delivering regulators with what they need to grant a licence.

Some of the new therapies if they're given to people early enough could have the potential to be very much like cures, because they could be given at a point where lungs have not started to become damaged in an irreversible way. There's room for some innovation in clinical trials for younger children, the gold standard endpoint is FEV1 but it's very difficult to do that in younger children, also it's not very reliable.

I urge pharma to think about how they can make trials easier for younger children so that they're less invasive in terms of the tests they do and can be powered properly so that they can be done with smaller populations. That is probably the route to an effective cure. It looks like the best we can hope for is to freeze the disease in its tracks, and clearly if you do that soon enough then it's effectively a cure.

RA: Brilliant, well thank you Oli for your time.

About the interviewee:

Oli was diagnosed with cystic fibrosis in 1978 at the age of three. After graduating from UCL with a law degree in 1998, he spent 12 years working in investment banking and private equity in New York and London. He is currently working with the Cystic Fibrosis Trust as a Special Adviser on Research and Patient Involvement. Oli is also a director of Zespa Media, an independent TV production company and a consultant to Asset Match, an online peer-to-peer platform for trading shares in UK private companies. In addition, he acts as a consumer reviewer for Cochrane's Cystic Fibrosis & Genetic Disorders Group and writes a regular column on CF research and the drug development pipeline for CFUnite.org, backed by The Wellcome Trust.

What misunderstandings do you find that there are around cystic fibrosis?