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.

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