Transplants – the FDA Asks Patients

I was reading an article the other day about the 25th BIO International Convention – for biotechnology R&D organisations worldwide – held in Boston, early this month. I read this stuff to find out the latest in research about new products that may be useful for us BigD-ers, or (more likely) those who are facing dialysis sometime in the future.

FDAWhat caught my eye was a summary of a “Town Hall” session with the FDA (the US Food and Drug Administration), who manage the development and release of drugs, vaccines, biological products and medical devices for US humans. The session was a forum for senior FDA officials to meet with industry representatives.

Dr Janet Woodcock,One of the speakers was Dr Janet Woodcock, the head of the FDA’s Center for Drug Research & Evaluation. At one point in the session, Dr Woodcock was asked what she was currently most excited about.

Three things excited her. The first two were about a new type of drug development, and advances in manufacturing.

Patient-Focused Drug Development

But the third was a big surprise: She is very excited about patient-focused drug development, which “will really be a force over the next decade,” she said. “We can’t even estimate how strong this will be. The patients are on fire. They are getting into the tent all the way.”

I’d never heard of patient-focused drug development, so I Googled it. The first thing that came up was the FDA site Patient-Focused Drug Development: Disease Area Meetings Held over the last four years. It summarised 24 patient-focused meetings to get the patient viewpoint on specific diseases and their treatments.

Patient-Focused Drug DevelopmentThe meetings enable the FDA to gather and record patient experiences and insights about each disease and to use these insights as key design criteria for new drug development. This wonderful initiative is a direct outcome of the ePatient movement that has grown to prominence over the last decade.

While the primary beneficiary of being an e-Patient has always been the patient, organisations around the world have come to understand the benefit of talking to and listening to patients has created a powerful new health resource that can dramatically improve patient care.

VoiceI cast my eyes down the list to find something kidney related, and halfway down, there it was: Patients who have received an organ transplant.

I clicked on Meeting Report and downloaded 12-plus pages of excellent patient discussion (many of them kidney recipients) of their experiences and perspectives on managing their health post-transplant.

Patient meetings

The meetings covered two topics:

Topic 1 – Post-Transplant Health Effects.

Findings: For some participants, the health effects they experienced were described as being manageable and tolerable. Others said they placed a significant burden on their overall health and well-being.

rejectionPatients were asked to describe the most worrying impacts of their organ transplantation on daily life. In response, they… highlighted organ rejection, cancer, and infection. And many who had not directly experienced these effects described them as being their biggest worries for the future. As one patient said, “while others may breeze out the door in the morning, I am already preoccupied with preventing rejection, infection and cancer.”

Overall Impacts on Daily Life

Participants shared numerous perspectives on how their lives had changed post-transplant. The most commonly mentioned impacts on daily life included:

  • Concern for the future
  • Emotional impacts
  • The burden of managing day-to-day life
  • Social impacts.

(See the report for more detail)

Topic 2 – Transplant and Treatment Impacts

Here, patients spoke about the effectiveness of their treatment programs, their experience with immunosuppressants, the side effects they had experienced, and the impact of their treatment on daily life.

Detailed and frank patient experiences with prescription drugs, treatment downsides, clinic visits and monitoring, adherence to medications, non-drug therapies, and other types of treatments are set out in the report, and well worth a read.

Outcomes

As set out in the Conclusion, the workshop produced two valuable outcomes:

1.      Patient opinions on ideal treatments for organ transplantation

Based on this opportunity to come together and discuss these topics, patients have also developed a range of what they see as ideal objectives for transplant treatments, to help inform the processes of the FDA, the organisation that has the most say in their development:

Patient-Focused Drug Development2

New approaches are needed to improve the long-term success of transplanted organs, to prevent and treat antibody-mediated rejection, to individualize treatment, and to reduce the adverse reactions associated with immunosuppressant regimens.

2.      The value of Patient Involvement

Even more encouraging is the recognition of the value that patients have to offer:

Patient perspectives play an important role when considering how to best facilitate drug development post-organ transplantation. These perspectives also inform how patients view the benefits and risks of various drug products in the complex area of post-transplant treatment.

ePatientPatients…  provided a vivid examination of the challenges and burdens facing patients who have received an organ transplant… The meeting… provided FDA with the opportunity to hear from patients and caregivers first-hand the impact of organ transplantation and post-transplant treatment regimens on patients’ lives.

The FDA recognizes that patients have a unique ability to contribute to our understanding of their condition and treatment management, which is important to our role, and that of others, in the drug development process.

From what better perspective is there to offer ideas and input to the development of long-term success of transplants, the prevention and treatment rejection, personalised treatment, and the reduction of adverse reactions to immunosuppressant drugs?

And this from just one of the 24 patient-focused meetings.

No wonder Dr Woodcock is excited. So am I.

Will my boyfriend die young?

Kristy sent me an email recently:

Good evening Greg. 

My boyfriend suffers from dense deposit disease and although he is fine at the moment, I fear the time he may need dialysis or a transplant. I suffer from anxiety so it’s hard to shake these worries. He’s very active, likes a drink, likes to socialise and I just worry about how much of an impact kidney failure will have on his life. Any experience you could share would be a big help to me. At times, I catastrophise the thoughts to simply the fear that he will die young (he’s 26 at the moment).

Kind regards, Kristy

Me:

Hi Kristy, thanks for your email. As I understand it, dense deposit disease is bad news for kidneys, in that it primarily affects kidney function. So you are right, kidney failure is a likely outcome in the future.

How far away that is, only time will tell. Everyone is different, but even sick kidneys can hang on to some function for years. I was diagnosed when I was 20 but, by gradually changing my diet and keeping reasonably fit, I didn’t start dialysis until I was 43. So I had 23 years of fairly normal life before dialysis. I say fairly because during the last couple of years I felt progressively sicker. But during that time, I got married, had three kids and lived a full, satisfying life. Then, of course, I went on dialysis.

At the time, I thought my life was over, but within a few weeks I began to feel healthy again and found that I could still live a full and healthy life: I just had to build dialysis into my weekly routine.

This pattern is not at all unusual, many people have the same experience. And it is likely that you and your boyfriend will too.

Your boyfriend is your boyfriend because of who and what he is and how you are together. Those things won’t change once he starts dialysis, even if he has to modify his life to fit it in. The challenge can bring you both closer and make your bond stronger (it did for me and Julie). Life will go on and you will be happy and sad, frustrated and delighted in the same way you are now.

So don’t catastrophise (I love that word!). Take the long view and all will be well.

Regards, Greg

From Kristy:

Greg, thank you so, so much for this email. It has really helped me to feel a sense of ease regarding the future. I really appreciate you taking the time to respond.

The first time

scared-womanWe all remember our first dialysis session. I certainly do. At the Central Dialysis Unit, just down the stairs from Ward 4, the kidney ward. It was a super emotional time. Julie walked with me into the unit, my heart thumping, my mind racing, looking everywhere, taking in everything. I kept my face blank, hiding both fear of the unknown and anger that it was really about to happen. Everything was new and unfamiliar. Nurses moving briskly about. Patients on dialysis machines in all directions. And there, across the room, an empty chair next to a humming and flashing machine, just waiting for me.

Luckily, the Unit Manager was an experienced and sensitive soul. She knew it was my first session and made sure I knew she knew. “I know you’re not too happy about being here, but we’ll do our best to make today a gentle start to your time on dialysis. *Heather will be putting you on today. She is one of our best cannulators, (more…)

Flash: Dialysis staff go home and leave patient locked in

This would funny if it wasn’t so serious. Was this just a bad day or is there something fundamentally wrong with the culture of the organisation that runs the unit?

I hope that, like me, most dialysis patients just can’t imagine being locked in their unit after everyone else has followed Elvis from the building.  Hello?  Hello?  Is anybody there?  Anybody?? (more…)

Speaking of dialysis…

1-IMG_1532As I hoped, here is the video of my speech to the Diaverum Annual Conference at Cascais, Portugal, last month.  It was called: The View from the Chair, a Patient’s Perspective.

It covers a bit of ground, but the highlights (apart from the joke at the start!) are: (more…)

Off to the Diaverum Global Dialysis Conference

I’ve been missing in action for the last few weeks, for a couple of reasons.

Josie & Liam Firstly, our No.2 Son just married his lovely fiancé here in sunny Melbourne (on the beach at Elwood).  It was a great wedding, but they live in London, so Julie and I have been pretty busy preparing. Not much time for blogging.

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Getting fit and staying fit on dialysis – bit by bit

1-fitbitIt is pretty well accepted all over the planet that being fit helps you to enjoy life more and is a major driving force for a longer life.  Not just BigD-ers; everyone.  And the two biggest challenges to our fitness are getting fit and staying fit.  We all face the same problems getting fit.  But staying fit can be a little trickier for people on dialysis.

This is because those of us who have made the effort to get fit between dialysis runs can get our legs cut out from under us by a spectrum of medical dramas that pounce on us from nowhere.  Dramas that people with full kidney function would either barely notice, or not need. (more…)

20 Years of dialysis: My plan for the next 20

ElderThis month it’s my 20th anniversary on dialysis.  Back in 2005, I am very sure I didn’t expect to be still dialysing (though I fully expected to still be here).

When people ask me the secret of successful dialysis I say, “Just keep turning up”.

And one failed and one iffy transplant later, here I am.  A little worse for wear, but happy and healthy, and expecting to last for at least another 20. (more…)

eHealth – What’s in it for us?

kixTwo reasons I worked through the eHealth MOOC I wrote about last time were to find out just how big the eHealth movement is and where it was or can be successful in making life easier providing better health outcomes for us BigD-ers.

Firstly, it’s big, very big.  Most countries are setting up or designing an eHealth framework of some kind or other.  They include the usual suspects, like Scandinavia, the UK, most of Europe, Hong Kong, Singapore, Australia and New Zealand, and parts of Canada and the US.  Thankfully much of the rest of the world is also on the job, like Russia, Brazil, India, Pakistan, the Philippines, Mexico, Turkey, Nigeria, Israel, Iran, Saudi Arabia. (more…)

The rising eHealth tide is lifting all boats

Most of us have heard of eHealth – short for Electronic Health, and in its simplest form meaning the use of information and communication technologies for health delivery and management.

While that sounds pretty dry and not that interesting, eHealth is a tsunami, riding on databases of health information and innovative, patient-centred, wireless and wearable technologies that are changing health care systems and health services delivery around the world. (more…)