Epidermolysis bullosa (EB) places a significant socioeconomic burden on patients. Several reports stated that Epidermolysis bullosa had a considerable negative impact on the quality of the lives of patients. Although it is a rare disease, EB is a cause of significant morbidity and mortality among the affected individuals. It is observed that EB affects 1 in 30,000 to 50,000 people. As per DelveInsight estimates, the total Epidermolysis bullosa prevalent population in the 7MM (the US, EU5 (the UK, Germany, France, Spain, and Italy) and Japan) was 41,509 in 2017.
Epidermolysis bullosa is a group of heterogeneous congenital conditions that causes blisters on the skin. It is caused by an error or mutation in the keratin or collagen gene, and it affects the connective tissues. Patients with EB have soft, fragile skin, which in response to rubbing or any other form of frictional trauma, gives rise to blisters at different locations of the body, primarily on the hands and feet. However, there are several subtypes of the condition that differ depending upon depth, severity, and underlying cause of the blisters. However, three major subtypes are Epidermolysis bullosa simplex (EBS), junctional EB (JEB), and dystrophic EB (DEB). Assessments, as per DelveInsight’s analysts, demonstrated EBS to be the most common type, which is also a milder form of EB with lower risks of complications. The form that ranges from mild to severe is DEB, and the rarest and most severe is JEB.
Conventionally, there is a wall of protein that acts as an anchor preventing the two consecutive layers of skin from tearing or shearing. However, EB patients lack such kind of protein anchorage. Milder subtypes of EB do not cause much harm and are easy to cope up with as compared to the more recessive and severe forms. The condition in some cases worsens to such an extent, making it impossible to even swallow solids or urinate, affecting several organs and hampering both life quality and lifespan.
The current Epidermolysis bullosa treatment regimen revolves around the prevention of blisters and managing wounds. Avoidance of intensifying factors that cause blistering is the backbone of management. The primary goal of Epidermolysis bullosa treatment is to guard skin against any frictional trauma and infections. Pruritus is often a common complication in patients with epidermolysis bullosa and is quite bothersome as it interferes with sleep. The present EB market offers antihistamines, gabapentin, pregabalin, TCA, serotonin-norepinephrine uptake inhibitors (SNRIs), and other nontraditional antipruritic agents for itch treatment. Several patients describe EB as an ineffably painful condition. To manage pain, patients are often given paracetamol and ibuprofen for mild pain while opioids and anxiolytics for severe pain. To facilitate easy swallowing of solid foods, anti-inflammatory drugs such as corticosteroids are available in the Epidermolysis bullosa treatment market. Psychological evaluation and treatment and chlorhexidine baths also prove to be beneficial. Nutritional support in the management of EB is paramount. Any dietary intervention that can complicate the condition further is kept at bay with the involvement of highly nutritious supplements. Surgical treatment is opted to minimize deformities and contractures that are a result of repeated blistering. Lastly, motivated spirits, positive attitude, and strong support help patients and their caregivers to sustain the quality of life.
What lies in the future for EB Market
While EB cannot be cured, substantial research has been made in the field to find EB-specific medications, which is the major present unmet need of the market. The scientific community is making strides in understanding this rare group of diseases, which has led to the discovery of novel treatment approaches. Several pharma companies worldwide are diligently working towards the development of novel treatment therapies for Epidermolysis bullosa. Many have reported a considerable amount of success over the years. Key pharma companies in the Epidermolysis bullosa market include Fibrocell Technologies, Castle Creek Pharmaceuticals, RegeneRx, Amryt Pharma, Abeona Therapeutics, Krystal Biotech, Shionogi, among others that are expected to propel the growth of the Epidermolysis bullosa market size in the foreseeable future, which DelveInsight estimated to be USD 2,283.40 million in the 7MM in 2017. The launch of Epidermolysis Bullosa pipeline therapies, expected to be between 2021 and 2024, including FCX-007 (Fibrocell Technologies), CCP-020 (Castle Creek Pharmaceuticals), RGN-137 (RegeneRx/Lenus Therapeutics), AP-101 (Amryt Pharma), EB-101 (Abeona Therapeutics), KB103 (Krystal Biotech), and S-005151 (Redasemtide (Shionogi)) are expected to fuel the Epidermolysis bullosa market during the forecasted period (2020–2030). At present, CCP-020 is the only drug under investigation for the treatment of EBS; thus, it is expected to enjoy its fair share of the market without any tugs from other therapies. The drug is expected to enter the EB Market in the year 2023.
The EB market is witnessing a fair influx of pharma companies with their gene therapies in the market, such as FCX-007, EB-101, and KB103. DelveInsight’s Epidermolysis bullosa market evaluation further demonstrated a promising future for KB103 among all these gene therapies that are under development. Furthermore, an expected increase in the EB patient pool will boost the EB market in the forecasted period (2020–2030). However, the cost-factor can turn the sailing ship of the gene therapies in the ocean of EB market size around. The price of the gene therapies depends on several parameters besides their success rate, such as level of innovation, durable clinical benefit, treatable patient pool, and impact on health systems. The Institute for Clinical and Economic Review estimated that the cumulative budget impact for gene and cell therapies alone could rise to USD 3 trillion in the US when only about 10% of eligible patients are treated with these therapies. However, to reduce the financial burden on patients, there are numerous payment models to provide reimbursements that are under consideration.
To tidy it up, the Epidermolysis bullosa treatment needs to be tailored to suit the needs of the patients. Preventive measures, nutritional support, and wound management must be patient-centric. Skin-scarring and lesions require periodic healing and assessment; thus, it should be a part of the treatment regimen. The pharmacological treatment should aim to reduce the severity and complexity of the condition over the course of time. Further, wound care, which so far is reported to be time-intensive, needs to be addressed. Often there are instances where Epidermolysis bullosa hampers the daily schedule or routine of the patient, faltering the academic, social, and professional momentum. Thus, elevated awareness and social support will further help patients to come out of the condition emotionally and mentally. The unmet needs in the Epidermolysis bullosa market are many, and thus it offers pharma companies to explore the EB market. Further, an increase in research funding is also setting the market growth in motion. Thus, the Epidermolysis bullosa market is hurling towards a better and promising future, which will ultimately bode well for the patients living with painful blisters and wounds associated with EB.