Vitiligo Patients Share Their Experiences, Frustrations With Treatment Options With FDA

Christine Kilgore

April 01, 2021

Patients with vitiligo have faced significant impacts psychosocially and in many cases, profound losses of identity – and they've had only minimal success with treatment, according to participants who spoke at and provided input at a public meeting on patient-focused drug development for the disease.

The virtual meeting, held in March, was part of the Food and Drug Administration's Patient-Focused Drug Development (PFDD) initiative, which began in 2012 and aims to provide a systematic way for patients' experiences, needs and priorities to be "captured and meaningfully incorporated" into drug development and evaluation.

Seemal Desai, MD, of the department of dermatology at the University of Texas, Dallas, who attended the meeting as an observer, said in a later interview that while "all skin diseases have a psychosocial component … vitiligo is a really unique one, because it really relates to the patient's own identity.

"What I heard loud and clear from the FDA [leaders who ran and attended the meeting] is recognition that patients are suffering. They needed to hear about the emotional devastation of the disease and how it is a medical condition," Desai said.

The meeting was the "first-ever vitiligo meeting at the FDA" and was a "historic moment for the vitiligo community," he added.

The pigmentation disorder affects 1% of the world's population. Nearly 50% have an onset before age 20, and onset before age 12 is common, Brenda Carr, MD, medical officer with the FDA's Division of Dermatology and Dentistry in the Center for Drug Evaluation and Research, said in an introductory overview.

The only FDA-approved treatment for vitiligo is monobenzone cream, but this is indicated for final depigmentation in extensive vitiligo and is no longer marketed. Treatment options include corticosteroids, calcineurin inhibitors, vitamin D analogues, phototherapy, surgical treatments (tissue grafts and cellular grafts), and camouflage (make-up, tattoos, self-tanning products), Carr said.

Patients participated in one of two panels – one about the health effects and daily impacts of vitiligo and the other about treatments – or submitted input electronically. All patients were invited to answer poll questions and open-ended queries, including questions about how they would assess new treatments.

Several panel members who are Black shared series of photos that showed the evolution of defined white patches into widespread, generalized depigmentation. One man with skin of color who lives in the Netherlands said he has had vitiligo since the age of 12, but that when he became older, over a 4-year period, he was "transformed from a man of Indonesian roots to a totally white man."

Experiencing only minimal benefit from treatment and the short-term effectiveness of treatments were the top two answers to a poll question asking participants about the most burdensome impacts of the medical products and interventions they have used. Difficulty in accessing treatment, concern about serious risks of treatment, and uncertainty about long-term effects of treatment were other frequently chosen answers.

Patients described the onerous nature of phototherapy (treatments repeated several times a week over long periods) and other treatments, and several described feeling that some physicians did not take the condition seriously or fully know of treatment options.

In her closing remarks, Kendall Marcus, MD, director of the Division of Dermatology and Dentistry at the FDA, acknowledged the input. "Some of you have had difficulty having your disease taken seriously by physicians who view it as a cosmetic condition and are reluctant to treat because they believe your expectations will not be met, that it will be an exercise in frustration," she said.

Regarding the impacts of treatments that have been utilized, "some of the treatments make it impossible to do other activities such as work or care for yourself in other ways," Marcus said. "Certainly that's not the kind of treatment … that anybody wants to have."

Desai, who utilizes an array of oral and topical treatments and phototherapies in his practice, said he was surprised and disheartened to hear the level of concern about side effects of treatment. Most of those who expressed concerns alluded to phototherapy. "I think light treatments are very safe and effective," he said in the interview. "I might equate [such concerns] to the older PUVA [psoralen plus UVA ultraviolet light] therapy, but not so much the newer therapies."

The FDA participants probed patients for their perspective on a meaningful level of repigmentation and an acceptable level of risk for any new hypothetical treatment. Specifically, they asked whether patients would use a new topical cream approved for vitiligo if the cream needed to be applied once a day, would have up to 50% efficacy in some people, and would have common side effects of redness and irritation at the application site, mild acne, and burning, as well as several rarer but more serious side effects.

Only 36% answered yes; 24% said no, and 40% answered maybe. Some patients said during the meeting that they had accepted their condition and were not pursuing any treatment. Others said they were very interested in treatment but only if the level of repigmentation were significantly higher than 50%. Some described their fear that positive treatment effects would be short term only.

Meri Izrail Kohen, who lives in France and has lost half of her skin's pigmentation, said that treatment efficacy is "not only about how much recovery of pigment it allows, but how long the recovery will last." Some treatments will work for some patients, she said, "but even in these cases when we stop the treatment, it will come back somehow."

Lee Thomas, a TV anchor in Detroit, and a reporter and author of the book "Turning White," described how he tried "every treatment he could afford" but stopped trying 10 years ago. A treatment in Germany "gave me 80% of my pigment back, but it has gone again," he said. "I would love to have my face back again. I was born a Black child, and I'd like to die a Black man."

Patients also spoke of their skin burning easily outdoors; skin sensitivity, itchiness, and burning with the spread of disease; treatment expenses and not being able to afford treatment; and worsening of their vitiligo with the stress of the pandemic. Parents expressed having fear that their children would develop vitiligo and experience bullying, isolation, or other emotional or psychosocial impacts that they had experienced; one described having an almost-paralyzing anxiety when he saw patchy white spots on his 20-month-old daughter (it was not diagnosed as vitiligo).

Calls for further advancement with home phototherapy – which Desai said is a growing market but not yet adequately covered by insurance plans – were also made, as were pleas for research on the root causes of the disease.

Patients clearly indicated "that they need more efficacious treatments, and more comprehensive treatments," said Desai, who chairs the advisory committee of the Global Vitiligo Foundation. "It's disappointing to me that patients come in with a not fully optimistic viewpoint, with a lot of anxiety and angst that treatments are not going to work. … But the Agency needs to hear that. This means that there haven't been good treatments and we need more."

The FDA will accept public comments until May 10, 2021, at which time comments will be compiled into a summary report. FDA officials assured patients that the report would be visible and circulated not only within the FDA but among drug companies, researchers, and other product developers.

This article originally appeared on, part of the Medscape Professional Network.


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