본 보고서는 미국, EU 4개국(독일, 스페인, 이탈리아, 이탈리아, 프랑스), 영국, 일본의 수표성 표피 박리증, 역사적 및 예측 역학, 수표성 표피 박리증 시장 동향에 대한 상세한 이해를 제공합니다.
이 보고서는 현재 치료법, 신약, 개별 치료법별 시장 점유율, 2020년에서 2034년까지 주요 7개국 수표성 표피 박리증 시장 규모 현황 및 예측을 제공합니다. 또한, 현재 수표성 표피 박리증 치료법/알고리즘과 미충족 의료 수요도 다루고 있으며, 최적의 기회를 발굴하고 시장 잠재력을 평가했습니다.
수표성 표피 박리증은 극도의 피부 취약성과 약간의 마찰이나 외상에도 물집이 생기는 것이 특징인 유전성 피부 질환입니다. 이 질환은 피부의 구조적 완전성을 유지하는 역할을 하는 단백질에 영향을 미치는 유전자 돌연변이로 인해 발생합니다. 수표성 표피 박리증의 중증도는 경증부터 생명을 위협하는 심각한 증상까지 다양한 아형에 따라 달라집니다.
수표성 표피 박리증 환자의 증상은 잦은 피부 물집 형성 및 상처 치유 지연과 같은 증상 발현으로 시작됩니다. 피부의 취약성과 수포의 분포를 평가하기 위해 의료 전문가가 임상 검사를 실시합니다. 진단을 확정하고 특정 수표성 표피 박리증의 아형을 분류하기 위해 피부 생검과 유전자 검사를 시행합니다. 진단 후에는 피부과 전문의, 상처 관리 전문가, 영양사, 물리치료사가 참여하는 다학제적 관리 계획이 수립됩니다. 수표성 표피 박리증과 함께 살아가는 데 필요한 정신적 지원과 상담이 제공됩니다. 피부 건강 상태를 관찰하고 합병증을 예방하기 위해서는 장기적인 관리와 정기적인 경과 관찰이 필요합니다. 의료 자원과 전문 지식에 대한 접근성은 경과 기간과 결과에 영향을 미칩니다. 조기 진단은 적시에 개입하고 환자의 삶의 질을 향상시키기 위해 매우 중요합니다.
수표성 표피 박리증의 치료는 주로 증상 관리와 합병증 예방에 중점을 둡니다. 여기에는 상처 관리, 감염 관리, 통증 관리 및 적절한 영양 공급이 포함됩니다. 심한 수표성 표피 박리증의 경우 피부 외상을 최소화하기 위해 특수 드레싱, 붕대 및 보호복이 필요합니다. 또한 손의 변형과 같은 합병증에 대처하기 위해 외과적 개입이 필요할 수도 있습니다.
표피 수포증은 가려움증을 효과적으로 억제하는 코르티코스테로이드로 치료할 수 있습니다. 그러나 국소 또는 경구용 코르티코스테로이드를 장기간 사용하면 시상하부-뇌하수체 축을 억제할 수 있으며, 이는 소아에서 흔히 볼 수 있습니다.
현재 승인된 치료법은 3가지 밖에 없습니다. 따라서 현재로서는 효과적이고 표적화된 치료법이 이 질환의 치료에 있어 중요한 미충족 수요로 남아 있습니다.
시판 중인 약품
VYJUVEK(beremagene geperpavec) : Krystal Biotech
2023년 5월, VYJUVEK(beremagene geperpavec-svdt)은 생후 6개월 이상의 DEB 환자를 대상으로 한 최초의 재사용 가능한 유전자 치료제로서, 생후 6개월 이상의 DEB 환자를 치료하기 위해 승인되었습니다. VYJUVEK은 최초의 재사용 가능한 유전자 치료제이자 열성 및 우성 DEB 치료제로 FDA 승인을 받은 최초이자 유일한 의약품입니다.
2023년 11월, Krystal Biotech는 유럽의약품청(EMA) 산하 약물사용자문위원회(CHMP)에 제출한 영양장애성수표성 표피 박리증(DEB) 치료제 VYJUVEK의 판매승인신청(MAA)이 유효하며, 현재 CHMP의 심사 중이라고 발표했습니다. CHMP의 의견은 2024년 하반기에 나올 것으로 예상됩니다. 한편, 일본에서의 승인은 2025년 초로 예상되고 있습니다.
FILSUVEZ(oleogel-S10) : Chiesi Farmaceutici
자작나무 껍질에서 추출한 birch triterpenes을 함유한 생약제제인 FILSUVEZ(oleogel-S10/birch triterpenes/구 AP101)는 2022년 6월 21일 EU 전역에서 판매 허가를 획득했습니다. 2022년 2월, Amryt는 영양성 수표성 표피 박리증에 수반되는 표재성 상처 치료제로서 6개월 이상의 환자를 대상으로 한 OleoGel-S10에 대한 신약허가신청(NDA)을 미국 FDA에 제출했습니다. FDA는 Amryt에게 수표성 표피 박리증에 대한 Oleogel-S10의 효능에 대한 추가 확인 증거를 제출할 것을 요청했으며, 2023년 4월 Chiesi Farmaceutici가 Amryt Pharma 인수 완료를 발표했습니다.
2023년 12월, 미국 FDA는 6개월 이상의 접합형 수표성 표피 박리증(JEB) 및 영양장애형 수표성 표피 박리증(DEB) 환자의 부분적 두께의 상처 치료제로 FILSUVEZ 국소용 젤을 승인했으며, FILSUVEZ는 JEB에 수반되는 상처 치료제로는 처음으로 승인되었습니다.
신약
EB-101 : Abeona Therapeutics
EB-101은 열성 유전성 수표성 표피 박리증 치료제로 개발 중인 자가 세포 치료제이며, EB-101을 이용한 치료는 유전자 도입을 통해 COL7A1 유전자를 환자의 피부 세포(각질세포 및 그 전구세포)에 도입하여 환자에게 이식하는 방식으로 이루어집니다. 콜라겐의 발현을 가능하게 하고 상처 치유를 촉진하는 능력에 대해 연구되고 있으며, EB-101은 RDEB의 치료제로서 최초로 승인될 가능성이 있으며, 가장 고통스럽고 쇠약하게 만드는 큰 만성 상처에 대한 유일한 내구성 있는 치료제가 될 수 있습니다.
2022년 11월, 주요 임상 3상 시험인 VIITAL 시험에서 상처 치유와 통증 완화라는 두 가지 주요 평가 항목을 달성하고, 대규모 만성 열성 영양결핍성 수표성 표피 박리증에서 통계적으로 유의미하고 임상적으로 유의미한 개선이 확인되었습니다. 101의 승인을 위해 미국 FDA에 생물학적 제제 승인 신청(BLA)을 제출했습니다. 이번 신청에서 Abeona는 우선 심사를 신청했으며, 우선 심사가 승인되면 FDA의 심사 기간이 일반 심사 시 10개월인 반면, BLA 신청 접수 후 6개월로 단축될 것으로 예상하고 있습니다.
D-Fi (dabocemagene autoficel): Castle Creek Biosciences
FCX-007(dabocemagene autoficel)로도 알려진 D-Fi는 진행성, 파괴적인 통증을 동반한 쇠약성 수표성 표피 박리증(DEB)을 치료하는 자가유전자치료제 후보물질로, 현재 열성 유전성 수표성 표피 박리증 환자의 만성 상처의 국소 치료를 위한 임상 3상 단계에 있습니다. 개발 단계에 있습니다. 임상시험 계획에는 D-Fi의 다기관, 환자 내 무작위배정, 대조군, 오픈 라벨 시험이 포함되어 있으며, I/II상 임상시험에서 보고된 데이터를 기반으로 하고 있습니다.
수표성 표피 박리증에 대한 유전자 치료는 FDA가 승인한 최초의 국소 유전자 치료제인 VYJUVEK의 승인으로 현실이 되고 있습니다. 다른 두 가지 유전자 치료 후보물질은 아베오나와 캐슬 크릭 바이오사이언스(Castle Creek Biosciences)의 COL7A1을 체내에 도입하여 VYJUVEK과 유사한 효과를 발휘하는 것입니다.
VYJUVEK은 단순 헤르페스 바이러스 1형(HSV-1)을 유전자 치료 벡터로 사용한 최초의 제품입니다. 유전자 치료에 사용되는 다른 많은 바이러스와 달리, 헤르페스 바이러스는 세포에 감염되어도 숙주의 유전체에 통합되지 않습니다. 이는 통합으로 인해 정상적인 유전자 발현이 억제되어 암을 유발할 위험이 약간 있기 때문입니다.
JACE는 2018년 수표성 표피 박리증에 대한 식약처 허가를 받은 최초의 세포치료제입니다. 줄기세포를 이용한 치료법은 특히 불치병으로 여겨졌던 질환에 대한 치료법이 점점 더 중요해지고 있으며, ABCB5+간엽간질세포(ABCB5+MSC)는 특별한 면역조절 및 항염증 작용으로 인해 수표성 표피 박리증을 포함한 다양한 만성 염증성 질환에 대한 새로운 유망한 치료 접근법입니다. 새로운 유망한 치료 접근법입니다. 이 세포치료제는 이미 만성정맥상처(CVU)에서 줄기세포가 체내 면역체계와 국소적으로 상호 작용하여 만성창상(CVU)을 봉합할 수 있다는 것을 보여주었습니다. 다른 두 가지 세포치료제에는 이신제약의 3상 제품인 ISN001과 Holostem Terapie Avanzate사가 개발한 ALLO-RV-LAMB3 도입 표피 줄기세포가 있습니다.
다양한 돌연변이로 인한 수표성 표피 박리증의 아형에 대한 치료법은 없으며, 현재 치료법은 상처와 통증 관리에만 초점을 맞추었습니다. 따라서 새로운 효과적인 치료법이 시급히 요구되고 있습니다. 표피 수포증의 치료는 지지 요법과 상처 관리, 수포 형성 및 감염 예방, 통증과 가려움증에 대한 대증요법, 합병증 예방, 모니터링 및 치료에 중점을 둡니다. 상처 관리는 여전히 치료의 기본이며, 통증을 줄이고 물집 형성, 흉터 형성 및 감염을 줄이고 예방하기 위해 환부에 반창고로 보호 붕대를 사용하여 통증을 완화하고 물집 형성, 흉터 형성 및 감염을 예방합니다. 드레싱 재료 교체 및 상처 치료는 시간과 비용이 많이 들고 환자와 간병인에게 부담이 될 수 있습니다. 많은 열성 수표성 표피 박리증 환자의 경우, 하루 4시간 이상의 상처 관리가 필요합니다. 종합적이고 평생에 걸친 치료가 필요하기 때문에 환자와 의료 시스템에 큰 임상적, 재정적 부담을 안겨줍니다.
본 보고서는 주요 7개국의 수표성 표피 박리증 시장에 대해 조사했으며, 시장 개요, 역학, 환자 동향, 새로운 치료법, 2034년까지 시장 규모 예측, 미충족 의료 수요 등을 정리하여 전해드립니다.
DelveInsight's "Epidermolysis Bullosa - Market Insights, Epidemiology and Market Forecast - 2034" report delivers an in-depth understanding of the Epidermolysis Bullosa, historical and forecasted epidemiology as well as the Epidermolysis Bullosa market trends in the United States, EU4 (Germany, Spain, Italy, and France) and the United Kingdom, and Japan.
The Epidermolysis Bullosa market report provides current treatment practices, emerging drugs, market share of individual therapies, and current and forecasted 7MM Epidermolysis Bullosa market size from 2020 to 2034. The report also covers current Epidermolysis Bullosa treatment practices/algorithms and unmet medical needs to curate the best opportunities and assess the market's underlying potential.
Study Period: 2020-2034
Epidermolysis Bullosa Overview
Epidermolysis Bullosa is a genetic skin disorder characterized by extreme skin fragility and blistering in response to minimal friction or trauma. The condition is caused by genetic mutations that affect the proteins responsible for maintaining the structural integrity of the skin. The severity of Epidermolysis Bullosa can vary widely, ranging from mild to life-threatening, depending on the specific subtype.
Epidermolysis Bullosa Diagnosis
The patient journey of Epidermolysis Bullosa begins with the onset of symptoms, such as frequent skin blistering and slow wound healing. Concerned caregivers seek medical attention, leading to a clinical examination by a healthcare professional to assess skin fragility and blister distribution. Skin biopsy and genetic testing are performed to confirm the diagnosis and classify the specific Epidermolysis Bullosa subtype. Upon diagnosis, a multidisciplinary management plan is developed involving dermatologists, wound care specialists, nutritionists, and physiotherapists. Emotional support and counseling are provided to cope with the challenges of living with Epidermolysis Bullosa. Long-term care and regular follow-ups are necessary to monitor skin health and prevent complications. Access to medical resources and expertise can influence the journey's duration and outcomes. Early diagnosis is crucial for timely intervention and improving the patient's quality of life.
Epidermolysis Bullosa Treatment
Treatment for Epidermolysis Bullosa primarily focuses on symptom management and complication prevention. This includes implementing wound care, infection control, pain management, and providing appropriate nutritional support. For individuals with severe Epidermolysis Bullosa, specialized dressings, bandages, and protective clothing may be necessary to minimize skin trauma. Surgical intervention might also be required to address complications like hand deformities.
Epidermolysis bullosa can be treated with corticosteroids, which effectively reduce itching. However, extended use of topical or oral corticosteroids may suppress the hypothalamic-pituitary axis, which can be more common in children.
Currently, only three therapies have been approved; therefore, as per the current situation, effective and targeted treatment remains a significant unmet need to treat this disease.
As the market is derived using a patient-based model, the Epidermolysis Bullosa epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by total prevalent cases of Epidermolysis Bullosa, total diagnosed prevalent cases of Epidermolysis Bullosa, gender-specific cases of Epidermolysis Bullosa, age-specific cases of Epidermolysis Bullosa, and type-specific cases of Epidermolysis Bullosa in the 7MM covering the United States, EU4 countries (Germany, France, Italy, and Spain), the United Kingdom, and Japan from 2020 to 2034. The total prevalent cases of Epidermolysis Bullosa in the 7MM comprised approximately 46,500 cases in 2023 and are projected to increase during the forecasted period.
The drug chapter segment of the Epidermolysis Bullosa report encloses a detailed analysis of Epidermolysis Bullosa marketed drugs and late-stage (Phase III and Phase II) pipeline drugs. It also helps understand the Epidermolysis Bullosa pivotal clinical trial details, recent and expected market approvals, patent details, advantages and disadvantages of each included drug, the latest news, and recent deals and collaborations.
Marketed Drug
VYJUVEK (beremagene geperpavec): Krystal Biotech
B-VEC is a non-invasive, topical, redosable gene therapy designed to deliver two copies of the COL7A1 gene when applied directly to DEB wounds. In May 2023, VYJUVEK (beremagene geperpavec-svdt) was approved for treating patients 6 months of age or older with DEB. VYJUVEK is the first-ever redosable gene therapy and the first and only medicine approved by the FDA for treating DEB, both recessive and dominant; a healthcare professional can administer that in either a healthcare professional setting or in the home.
In November 2023, Krystal Biotech announced that the Company's Marketing Authorization Application (MAA) to the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) for VYJUVEK for the treatment of dystrophic epidermolysis bullosa (DEB) has been validated and is now under CHMP review. A CHMP opinion is anticipated in the second half of 2024. Whereas approval in Japan is expected in early 2025.
FILSUVEZ (oleogel-S10): Chiesi Farmaceutici
FILSUVEZ (oleogel-S10/birch triterpenes/formerly known as AP101) is an herbal medicinal product that contains birch triterpenes from birch bark. FILSUVEZ received a marketing authorization valid throughout the EU on 21 June 2022. FILSUVEZ gel is indicated for patients 6 months and older to treat superficial wounds associated with Junctional Epidermolysis Bullosa and Dystrophic Epidermolysis Bullosa. In February 2022, Amryt announced it received a Complete Response Letter (CRL) from the US FDA regarding its New Drug Application (NDA) for Oleogel-S10 for the treatment of the cutaneous manifestations of dystrophic and Junctional Epidermolysis Bullosa. The FDA asked Amryt to submit additional confirmatory evidence of effectiveness for Oleogel-S10 in epidermolysis bullosa. In April 2023, Chiesi Farmaceutici announced the completion of the acquisition of Amryt Pharma.
In December 2023, the US FDA approved FILSUVEZ topical gel for the treatment of partial thickness wounds in patients 6 months and older with Junctional Epidermolysis Bullosa (JEB) and Dystrophic Epidermolysis Bullosa (DEB). FILSUVEZ is the first approved treatment for wounds associated with JEB.
Emerging Drug
EB-101: Abeona Therapeutics
EB-101 is an autologous, engineered cell therapy currently being developed to treat Recessive Dystrophic Epidermolysis Bullosa. Treatment with EB-101 involves using gene transfer to deliver the COL7A1 gene into a patient's skin cells (keratinocytes and its progenitors) and transplanting those cells back to the patient. EB-101 is being investigated for its ability to enable normal Type VII collagen expression and to facilitate wound healing. EB-101 has the potential to be the first approved therapy for RDEB and the only durable treatment to address large chronic wounds, which are the most painful and debilitating.
In November 2022, the pivotal Phase III VIITAL study met its two co-primary efficacy endpoints demonstrating statistically significant, clinically meaningful improvements in wound healing and pain reduction in large chronic Recessive Dystrophic Epidermolysis Bullosa. In September 2023, the Company submitted a Biologics License Application (BLA) to the US FDA seeking approval of EB-101. As part of the submission, Abeona requested a Priority Review, which, if granted, would shorten the FDA's review period to six months from the filing acceptance of the BLA, instead of 10 months under standard review.
D-Fi (dabocemagene autoficel): Castle Creek Biosciences
D-Fi, also known as FCX-007 (dabocemagene autoficel), is an autologous gene therapy candidate to treat dystrophic epidermolysis bullosa (DEB), a progressive, devastatingly painful, and debilitating, rare genetic skin disorder. D-Fi is currently in Phase III clinical development for the localized treatment of chronic wounds in individuals with Recessive Dystrophic Epidermolysis Bullosa. The clinical study plan includes a multi-center, within-patient randomized, controlled, open-label study of D-Fi and builds on data reported from the Phase I/II clinical trial.
Drug Class Insights
Preclinical or clinical testing of gene therapies (gene replacement, gene editing, RNA-based therapy, natural gene therapy), cell-based therapies (fibroblasts, bone marrow transplantation, mesenchymal stromal cells, induced pluripotential stem cells), recombinant protein therapies, and small molecule and drug repurposing approaches are all being currently explored for epidermolysis bullosa.
Gene therapies for epidermolysis bullosa are becoming a reality with the approval of VYJUVEK, the first FDA-approved topical gene therapy. Two other gene therapy contenders are Abeona and Castle Creek Biosciences, which operate similarly to VYJUVEK by introducing COL7A1 into the body.
VYJUVEK is the first to use the herpes simplex virus type 1 (HSV-1) as a gene therapy vector. Unlike many other viruses used for gene therapy, the herpes simplex virus does not integrate into its host's genome when it infects a cell. This is an advantage because there is a slight risk that integration can disrupt normal gene expression and cause cancer.
JACE was the first cell therapy to get the regulatory nod for Epidermolysis Bullosa in 2018. Stem cell-based therapies are becoming increasingly important, especially for previously incurable diseases. Due to the special immunomodulatory and anti-inflammatory properties, ABCB5+ mesenchymal stromal cells (ABCB5+ MSCs) represent a new, promising therapeutic approach for various chronic inflammatory diseases, including epidermolysis bullosa. The cell therapy agent has already shown in chronic venous wounds (CVU) that the stem cells interact locally with the immune system in the body, and therefore chronic wounds can be closed. The two other cell therapies include ISN001, a Phase III product by Ishin Pharma, and ALLO- RV-LAMB3-transduced epidermal stem cells, developed by Holostem Terapie Avanzate.
There is no cure for any of the subtypes of epidermolysis bullosa resulting from different mutations, and current therapy only focuses on managing wounds and pain. Novel effective therapeutic approaches are therefore urgently required. Management of epidermolysis bullosa focuses on supportive and wound care, prevention of blistering and infection, symptomatic relief of pain and itch, and prevention, monitoring, and treatment of complications. Wound care remains the cornerstone of treatment and includes the use of semi-occlusive, protective bandages to the affected area to decrease pain and reduce and prevent blistering, scarring, and infection. Dressing changes and wound care can be time-consuming and expensive, making them burdensome to patients and caregivers. For many patients with Recessive Dystrophic Epidermolysis Bullosa, wound care requires more than 4 h per day. Due to the comprehensive and lifelong care needed, the clinical and economic burden can be high for patients and healthcare systems.
In the race for gene therapy treatments for epidermolysis bullosa, Krystal has gained an advantage as it has already received FDA approval for VYJUVEK in both recessive and dominant forms of the condition. On the other hand, Abeona and Castle Creek Biosciences have pursued more conventional gene therapy approaches with their candidates, EB-101 and D-Fi, respectively, targeting Recessive Dystrophic Epidermolysis Bullosa.
Shionogi and Ishin Pharma are exclusively conducting their development efforts in Japan. Shionogi's Redasemtide is currently undergoing Phase II trials for DEB. The company is in discussions with the Pharmaceuticals and Medical Devices Agency (PMDA) to seek drug approval based on the results of the Phase II trial and the follow-up study.
Despite the challenges, the focus remains strong among epidermolysis bullosa specialists and researchers who are deeply engaged in researching new treatment modalities. Further investigation is crucial for this patient population, given the severe impact of epidermolysis bullosa on their quality of life and life expectancy, particularly when complicated by chronic wound infections or sepsis. The commitment to finding effective treatments for epidermolysis bullosa persists, emphasizing the urgency of continued research efforts.
As per DelveInsight's estimates, the potential drugs that can mark a significant change in the forecast period includes JACE, FILSUVEZ, VYJUVEK, PTR-01, RV-LAMB3-transduced epidermal stem cells, and others.
This section focuses on the uptake rate of potential drugs expected to be launched in the market during 2024-2034. An expected fast uptake of VYJUVEK in the US as Cheisi's product launched recently in the US after several hurdles, which might gave VYJUVEK some alone time in the US market. Even though we expect a Medium-fast uptake of Abeona's EB-101 and its launch in 2024, the label will only be restricted to RDEB patients, which is approximately 15% of the total EB patient pool comprising of severe patients. Two of the most potential upcoming therapies by Abeona and Castle Creek Biosciences are targeting the same patient pool and are expected to have stiff competition due to launch during the same period. Whereas, this remains quite different in Europe, where we expect FILSUVEZ to have an upper hand in terms of faster adoption and uptake due to first mover advantage and inclusion of a broader patient pool in its EMA label, even though FILSUVEZ has not been launched anywhere except in Germany as of July 2023.
Epidermolysis Bullosa Pipeline Development Activities
The report provides insights into different therapeutic candidates in Phase III and Phase II. It also analyzes key players involved in developing targeted therapeutics.
Pipeline Development Activities
The report covers information on collaborations, acquisitions and mergers, licensing, and patent details for Epidermolysis Bullosa emerging therapy.
KOL Views
To keep up with current market trends, we take KOLs and SMEs' opinions working in the domain through primary research to fill the data gaps and validate our secondary research. Industry experts contacted for insights on Epidermolysis Bullosa evolving treatment landscape, patient reliance on conventional therapies, patient's therapy switching acceptability, and drug uptake, along with challenges related to accessibility, include Medical/scientific writers; Dystrophic Epidermolysis Bullosa Research Association of America; Dermatologist and Professors; National Epidermolysis Bullosa Registry, and Center for Blistering Diseases, St John's Institute of Dermatology, NHS Foundation Trust, Departments of Dermatology, and others.
Delveinsight's analysts connected with 30+ KOLs to gather insights; however, interviews were conducted with 10+ KOLs in the 7MM. Centers such as the Center for Blistering Diseases, Department of Allergology and Dermatology, Department of Dermatology, Medical Center, etc., were contacted. Their opinion helps understand and validate current and emerging therapy treatment patterns or Epidermolysis Bullosa market trends. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the unmet needs.
Qualitative Analysis
We perform Qualitative and market Intelligence analysis using various approaches, such as SWOT analysis and Analyst views. In the SWOT analysis, strengths, weaknesses, opportunities, and threats in terms of disease diagnosis, patient awareness, patient burden, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are provided. These pointers are based on the Analyst's discretion and assessment of the patient burden, cost analysis, and existing and evolving treatment landscape.
The analyst views analyze multiple emerging therapies based on relevant attributes such as safety, efficacy, frequency of administration, route of administration, and order of entry.
In efficacy, the trial's primary and secondary outcome measures are evaluated; for instance, in Epidermolysis Bullosa trials, wound healing/wound closure is one of the most important primary outcome measures.
Further, the therapies' safety is evaluated wherein the acceptability, tolerability, and adverse events are majorly observed, and it sets a clear understanding of the side effects posed by the drug in the trials.
Market Access and Reimbursement
Reimbursement is a crucial factor that affects the drug's access to the market. Often, the decision to reimburse comes down to the price of the drug relative to the benefit it produces in treated patients. To reduce the healthcare burden of these high-cost therapies, many payment models are being considered by payers and other industry insiders. The payment models are based on clinical outcomes, annuity payments, and expanded risk pools. The Institute for Clinical and Economic Review estimates 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. There are various disease advocacy groups, such as DEBRA International, with nearly 50 national DEBRA and epidermolysis bullosa patient support groups that help patients with epidermolysis bullosa for insurance and reimbursement.
The report further provides detailed insights on the country-wise accessibility and reimbursement scenarios, cost-effectiveness scenario of approved therapies, programs making accessibility easier and out-of-pocket costs more affordable, insights on patients insured under federal or state government prescription drug programs, etc.