Patient Voices
Sharing the impact of living with the pain of Pachyonychia Congenita
Elevating the voices of the experts who know best about what life with PC is all about
The word Pachyonychia Congenita literally means “thick nails”. Because of this name, for years, PC patients often go undiagnosed because doctors are looking for 20 thickened nails. And even when patients are diagnosed correctly, not everyone understands that a disease that was thought to simply be a nail disorder could really affect a patient’s life as much as it does.
At PC Project, we continue to strive to share their experiences so that all may understand the profound effect PC has on the individuals who suffer from it. In this section, we elevate the voices of those who know best – the patients who limp, crawl, and get carried – and who deal with PC pain every day.
I don’t know what a painless day is.
Why does patient input matter?
Dr. Julie Beitz answered that question during her opening comments at the EL-PFDD meeting on Pachyonychia Congenita held on 6 April 2018. The following is an excerpt from that talk.
“First, patients may place different values to drug risks and benefits as compared to their healthcare providers, to their family members, and to drug regulators. In addition, patients themselves may have different perspectives on drug benefits and risks. Some may be willing to accept greater risks to achieve a small benefit, whereas others may be risk averse, requiring more benefit before accepting certain risks…
In the premarket period, patient input can inform a company’s decisions about which drugs or which formulations are developed. Patient input can inform the selection of meaningful efficacy outcomes—we call them endpoints as well—to be used in clinical trials, and what magnitude of change is important…
Patients can tell us which risks we should be monitoring in trials and can provide us with an understanding of the level of benefit that would be required in order to accept a certain level of risk…
Working together, we can expect to see innovation in a number of areas: in venues for patient engagement such as this, and in the systematic collection of patient preference information; innovation in clinical trial designs that incorporate patient preferences; innovation in development strategies for drugs in rare diseases, so that every patient counts and every measurement counts.”
