Dermatology clinical trials present many challenges to researchers over clinical trials in other therapeutic areas. These include finding perfect clinical trial sites, patient recruitment, patient retention, etc. These challenges are more prominent in rare skin diseases. Dermatology is the branch of medicine which deals with the skin. Dermatologists diagnose diseases related to skin, hair, nails, and cosmetic problems. Its specialty includes both surgical, as well as medical aspects. Various dermatologists (surgeons, clinical investigators) look for new interventions (treatments) by doing clinical trials on different skin conditions to make more efficient treatments.
Do you know that skin diseases are the fourth leading cause of health afflictions and affect nearly 900 million people worldwide?
Skin diseases such as psoriasis, atopic dermatitis, acne, rosacea, melanoma, and actinic keratosis have therapies but, still, many skin diseases require research. And, there are numerous rare diseases in dermatology worldwide that do not have therapies or treatment. So, there is more need for research to find treatments for these unmet needs.
Let’s briefly discuss a few dermatological conditions, their symptoms, and treatment options.
Skin diseases: Symptoms and treatment
Acne is a skin condition that occurs when the hair follicles become plugged with oil and dead skin cells. It causes whiteheads, blackheads, or pimples.
- Acne is the most common skin condition in the United States and affects up to 50 million Americans annually.
- Acne affects people of all age groups but is seen most commonly in teenagers.
- The four main factors that cause acne include
- Excess oil (sebum) production
- Hair follicles clogged by oil and dead skin cells
The class of drugs the dermatologist use for treatment include rRetinoids and retinoid-like drugs, antibiotics, azelaic acid and salicylic acid and dapsone.
Atopic dermatitis (eczema)
Atopic dermatitis (eczema) is a condition that makes the skin red and itchy. It is common in children but can occur at any age.
- One in 10 people will develop atopic dermatitis during their lifetime.
- Atopic dermatitis is a chronic disease and tends to flare periodically, and It usually accompanies asthma or hay fever.
- The symptoms for this disease include dry skin, itching, especially at night, red to brownish-gray patches
- treatments usually last from months or years to control it. And even if treatment is successful, signs and symptoms may return (flare).
The treatment plan for this atopic dermatitis includes creams that control itching and help repair the skin, drugs to fight infection, oral drugs that control inflammation. Recently the US FDA approved a new injectable biologic monoclonal antibody called dupilumab (Dupixent).
Melanoma is the most serious type of skin cancer which develops in the melanocytes that produce melanin. Melanoma is the most common skin cancer in the United States.
- More than 9,500 people in the U.S. are diagnosed with skin cancer every day.
- The exact reason for the cause of all melanomas is not clear, but exposure to ultraviolet (UV) radiation from sunlight or tanning lamps and beds is thought to increases the risk of developing melanoma.
- The treatment plan for melanoma includes surgery to remove affected lymph nodes, immunotherapy, radiation therapy, and chemotherapy.
Psoriasis is a skin disease that causes red, itchy scaly patches, most commonly on the knees, elbows, trunk, and scalp. Psoriasis is a common, long-term (chronic) disease with no cure. It tends to go through cycles, flaring for a few weeks or months, and treatments are available to manage symptoms.
Approximately 7.5 million people in the United States have psoriasis. Approximately 25-30 percent of people with psoriasis experience joint inflammation that produces symptoms of arthritis. This condition is called psoriatic arthritis.
Psoriasis signs and symptoms can vary from person to person and includes
- Red patches of skin covered with thick, silvery scales
- Small scaling spots (commonly seen in children)
- Dry, cracked skin that may bleed or itch
- Itching, burning, or soreness
- Thickened, pitted, or ridged nails
- Swollen and stiff joints
There are several types of Psoriasis
- Plaque psoriasis
- Nail psoriasis
- Guttate psoriasis
- Inverse psoriasis
- Pustular psoriasis
- Erythrodermic psoriasis
- Psoriatic arthritis
The most common form of psoriasis, affecting about 80 to 90 percent of psoriasis patients, is plaque psoriasis. Patches of raised, silvery-white scale cover the reddish skin, categorize this disease.
The treatment for Psoriasis include the use of
- Vitamin D analogs,
- Calcineurin inhibitors,
- Salicylic acid,
- Coal tar,
- Goeckerman therapy, and
Other treatment methods include Light Therapy, oral or injected medications, such as steroids, retinoids, methotrexate, cyclosporine, and biologics.
Rosacea is a common skin condition that causes blushing or flushing and visible blood vessels on the face.
- Rosacea is a common skin disease that affects 16 million Americans.
- The signs and symptoms may flare up for weeks to months and then go away for a while.
- Rosacea can be mistaken for acne, other skin problems, or natural ruddiness.
- Rosacea can affect anyone, but it’s most commonly occurs in middle-aged white women.
- To date, there’s no cure for rosacea, but treatment can control and reduce the signs and symptoms.
The common signs and symptoms of Rosacea include facial blushing or flushing, visible veins, swollen bumps, burning sensation, eye problems, and enlarged nose.
Epidermolysis bullosa is a group of rare diseases that cause fragile, blistering skin. The blisters appear in response to a minor injury, even from heat, rubbing, scratching, or adhesive tape. In severe cases, blisters may occur inside the body, such as the lining of the mouth or the stomach.
The signs and symptoms of Epidermolysis Bullosa include fragile skin that blisters easily, especially on the hands and feet, nails that are thick or don’t form, blisters inside the mouth and throat, thickened skin on the palms and soles of the feet, etc.
Pemphigus is a chronic disease that causes blisters and sores on the skin or mucous membranes, such as in the mouth or on the genitals. Pemphigus is most often seen in middle-aged or older people.
The two common types of pemphigus are
- Pemphigus Vulgaris
- Pemphigus Foliaceus
The treatment for Pemphigus includes corticosteroids, steroid-sparing immunosuppressant drugs such as azathioprine (Imuran, Azasan), mycophenolate (Cellcept), and cyclophosphamide.
Dermatomyositis is an uncommon inflammatory disease marked by muscle weakness and a distinctive skin rash. The condition affects both adults and children. Dermatomyositis usually occurs in the late 40s to early 60s, and in children, it most often occurs between 5 and 15 years of age. Dermatomyositis affects more females than males.
The most common signs and symptoms of dermatomyositis are
- A violet-colored or dusky red rash develops, most commonly on the face and eyelids and knuckles, elbows, knees, chest, and back.
- Progressive muscle weakness involves the muscles closest to the trunk, such as those in hips, thighs, shoulders, upper arms, and neck.
The treatment for dermatomyositis includes corticosteroids drugs such as prednisone, corticosteroid-sparing agents such as azathioprine (Azasan, Imuran), and methotrexate (Trexall). mycophenolate mofetil (Cellcept), rituximab (Rituxan), and antimalarial medications.
Hives, also known as urticaria, are a kind of skin rash with red, raised, itchy bumps. The condition is considered chronic hives if the welts appear for more than six weeks and recur frequently over months or years.
The treatment for Hives includes antihistamine drugs such as loratadine, fexofenadine, cetirizine, and desloratadine.
Itch (also known as pruritus) is a sensation that causes the desire or reflex to scratch, and dry skin often causes the itch. Depending on the cause of itchiness, the skin may appear normal, red, rough, or bumpy.
The symptoms of pruritis include redness, scratch marks, bumps, spots or blisters, dry, cracked skin, and leathery or scaly patches.
The general treatment for pruritis includes corticosteroid creams and ointments, antidepressants (selective serotonin reuptake inhibitors, such as fluoxetine and sertraline, and tricyclic antidepressants, such as doxepin. and phototherapy.
Clinical trials statistics in dermatology
After knowing various diseases, their symptoms, and treatments, let’s look at the clinical trial statistics of these diseases. The below chart presents the list of diseases, total trials on clinicaltrials.gov, ongoing clinical trials, and countries ranking based on the number of studies.
|S No||Disease||Total trials||On-going trials||Top 1||Top 2||Top 3|
|1||Acne||686||71||United States (386)||Canada (60)||France (24)|
|2||Atopic dermatitis||1,067||231||United States (457)||Europe (390)||Canada (130)|
|3||Melanoma||2,766||834||United States (1,801)||Europe (783)||Australia (204)|
|4||Psoriasis||1,808||296||United States (735)||Europe (706)||Canada (306)|
|5||Rosacea||175||17||United States (125)||Canada (15)||Germany (10)|
|6||Epidermolysis Bullosa||104||35||United States (51)||France (20)||United Kingdom (10)|
|7||Pemphigus||53||12||United States (24)||France (14)||Germany (7)|
|8||Dermatomyositis||101||29||United States (54)||France (12)||Sweden (8)|
|9||Urticaria (Hives)||342||64||Europe (162)||United States (131)||Canada (45)|
|10||Pruritus||452||59||United States (171)||Germany (58)||France (39)|
Clinical tirals registered on clinicaltrials.gov in dermatology
Clinical trials in dermatology present some unique challenges. In order to overcome these challenges, researchers need to consider multiple aspects.
- patient population availability,
- resources at the site, and data collection procedures,
- site personnel-related qualities such as interest and commitment,
- communicative skills, and
- experience in clinical trials need to be taken into account.
Researchers shall consider four crucial factors during site selection. These include predictability, enrollments, retention, and data quality.
In the coming section of this article, we will discuss the patient recruitment challenges and site selection strategies.
Patient recruitment challenges in dermatology clinical trial sites
“Eczema affects more people in the United States than diabetes” by this statement, it looks that patient recruitment in dermatology trials is easy compared to clinical trials in diabetics. But at the ground level, the scenario is different. Patient recruitment presents the same or more challenges compared with clinical trials in other therapeutic areas.
Let’s understand few challenges that researchers go through.
Patient awareness in dermatology trials is very low and a survey report shows that many of the patients are unaware of dermatology clinical trials. One solution for this is to utilize social and advocacy groups to create awareness among the patient population.
There are several patient recruitment platforms that may add on benefits to recruit patients for dermatology trials.
Lack of physicians training
In most cases, the physicians fail to refer clinical trials to patients. It may be due to a lack of training and lack of time to read and understand the complete protocol by the physicians.
One solution for this might be to train the physician/clinical investigators to educate the complete theme of the clinical study.
Brochures, flyers, and other literature materials can help physicians pass it to patients and improve recruitment.
Always there shouldn’t be any surprises around possible complications, risks, side effects, and/or responsibilities for the patient.
Good communication between the patient and the principal investigator or another qualified professional may help enroll the patient and avoid early dropout. Here, the patient-centric approach works where all the patient related documents are made easy to read. These documents should clearly outline all the clinical trial-related important information, including benefits, risks, inconveniences, side effects, responsibilities, and what to expect, during a procedure or appointment to the patients.
Patient site visits
Patient visit is another challenging factor that hinders patients from recruitment. The dermatological trials require regular site visits as the changes in the skin can be rapid. These regular visits affect the patient’s daily routine.
One solution for this is Virtual clinical trials (VCT). The VCTs are a relatively new and underutilized method to conduct clinical trials which use technologies (apps, electronically monitoring devices, trackers, etc.) and online social engagement platforms. Making correct diagnoses based on photographs and patient symptomatology has always been part of the dermatologist’s routine.
Virtual clinical trials (VCT) helps to minimize the number of patient visits to sites. It, in turn, provides the advantage in recruiting patients spread out in wide geographical locations.
Unlike other trials, dermatology trials require special attention from physicians toward patients. Depression, low self-esteem, poor performance due to psychological effects, lack of sleep, etc., are commonly seen in patients. So, along with effective treatment, patients may also require counseling and motivation. It helps to improve the patient retention rate.
Clinical trial sites in dermatology trials
COVID 19 pandemic has disrupted many health care sectors along with clinical trials. But, interestingly only, the dermatology sites reported less burden due to shutdown and lockdown. And the reason was that the site operated the clinical trials through online channels and app trackers. This process is known as decentralization.
Decentralized studies and sites
The decentralized studies improve patient recruitment as the trial site distance will not be a hindering factor anymore. These decentralized trials use technology for televisits (popularly known as teledermatology), remote electronic consent in order to minimize site visits.
Traditional trials require patients to visit investigator sites for all physician interactions. Decentralized trials leverage a variety of digital tools and technologies to enable the completion of trial tasks that normally involve face-to-face interactions remotely without compromising efficiency.
For example, dermatology trails provide easy access to gain experience with digital solutions, including e-consent, televisits, and remote assessment using a variety of digital tools.
This approach has a remarkable effect on patient retention, however, the clinical trial sites need to have such infrastructure to manage the trials remotely.
Intense compition between dermatology clinical trial sites
In some skin diseases, such as atopic dermatitis, there is intense competition for eligible patients. The sites with experience reach these patients more rapidly, evaluate and reach enrollment targets faster than the competitors.
So, for this, new and less experienced sites require training to meet dermatology clinical trial demands.
Feasibility study for site selection in dermatology trials
A feasibility study is a critical aspect before you plan a clinical trial. It helps you to choose an experienced and potential site. Unlike clinical trials in other therapeutic areas, dermatology sites are few and require efficient effort in site selection.
Before initiating any dermatology clinical trial, one needs to understand the site experience in patient recruitment and retention. It reduces the trials failures and delays, which in turn have an impact on the budget. The feasibility study can ease the site selection process and also helps to minimize the study start-up and site activation timelines.
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