보고서 요약
시장
Pfizer, Agios Pharmaceuticals, Editas Medicine, Hoffmann-La Roche 등 다양한 주요 기업들이 SCD 치료제 개발에 참여하고 있습니다. 새로운 치료제 및 기타 치료법의 출시가 예상에 따라 예측 기간(2024-2034) 동안 시장 규모는 크게 확대될 것으로 예상됩니다.
시판 중인 약품
CASGEVY(exagamglogene autotencel) : Vertex Pharmaceuticals/CRISPR Therapeutics
CASGEVY는 CRISPR/Cas9 기술로 BCL11A 유전자의 적혈구 특이적 증강인자 영역을 편집한 자가 CD34+ 조혈모세포(조혈모세포)로 구성된 유전체교정 세포치료제입니다. 태아 헤모글로빈(HbF) 생산을 증가시키도록 변형된 조혈모세포 이식 절차를 통해 단회 투여하는 것을 목표로 하고 있습니다. SCD 환자의 혈관폐쇄성 크리즈를 감소 또는 소실시키는 것으로 나타났습니다.
2023년 12월, Vertex Pharmaceuticals와 CRISPR Therapeutics는 CRISPR/Cas9 유전자가위 세포치료제 CASGEVY(exagamglogene autotemcel(exa-cel)를 혈관폐쇄성 크리즈( VOCs)를 재발하는 12세 이상 겸상적혈구증(SCD) 환자의 치료제로 미국 FDA의 승인을 받았다고 발표했습니다.
이후 CASGEVY는 영국에서 조건부 판매 승인을 받았습니다. 바레인 의약품 및 헬스케어 제품 규제 당국과 국가 보건 규제 당국은 조혈모세포 이식이 적절하고 인간 백혈구 항원이 일치하는 관련 조혈모세포 공여자가 없는 재발성 혈관폐쇄성 크리즈 또는 수혈 의존성 β- 지중해 빈혈(TDT)을 특징으로 하는 12세 이상의 SCD 환자를 대상으로 합니다.
ENDARI(L-글루타민) : Emmaus Life Sciences
ENDARI(L-글루타민)는 경구용 의약품 등급 L-글루타민(PGLG)으로, 성인과 5세 이상의 소아에서 겸상 적혈구 빈혈의 통증, 부종 및 기타 합병증을 완화하는 아미노산 제제로서, 산화의 주요 조절인자로 확인된 보조 효소인 니코틴아미디아데닌디뉴클레오티드(NAD)의 산화 환원 전위를 개선하여 적혈구의 산화적 손상을 감소시킵니다.
ENDARI는 미국 희귀의약품 지정(ODD), EU 희귀의약품 지정, FDA로부터 패스트트랙 지정(FTD)을 받았습니다.
2017년 7월, FDA는 5세 이상 성인 및 소아 환자에서 겸상적혈구증의 심각한 합병증 완화를 목적으로 ENDARI(L-글루타민 경구용 분말)를 승인했습니다.
새로운 치료제
미타피밧 : Agios Pharmaceuticals
미타피밧은 새로운 퍼스트 인 클래스 경구용 저분자 피루브산 키나아제 효소 알로스테릭 활성화제입니다. 미타피바트는 야생형 및 다수의 변이형 적혈구 피루브산 키나아제(PKR)를 유의하게 상향 조절하고, 아데노신 삼인산(ATP) 생성을 증가시키며, 2,3-디포스포글리세린산(2,3-diphosphoglyceric acid) 수치를 낮추는 것으로 나타났습니다. 용혈성 빈혈 치료제로 PYRUKYND(미타피바트)를 승인했습니다.
회사는 겸상적혈구 환자를 대상으로 미타피밧을 평가하는 임상 II/III상 시험을 시작했습니다.
2023년 6월, Agios Pharmaceuticals는 겸상적혈구증 환자를 대상으로 한 미타피바트의 세계 RISE UP 임상 II상 시험에서 미타피바트 50mg 및 100mg 1일 2회 투여(BID) 군 모두에서 주요 평가변수인 헤모글로빈 반응 을 달성했다고 발표했습니다.
인클라크맙: Pfizer
인클락맙은 P-selectin을 선택적으로 표적하는 새로운 완전 인간형 단일클론항체입니다. 이 단백질은 세포 접착을 매개하여 SCD 환자의 VOC로 인한 통증을 감소시키는 것으로 임상적으로 검증되었습니다. 전임상 결과에 따르면, 인클릭맙은 SCD 환자의 VOC를 완화하는 데 있어 동급 최강의 선택이 될 수 있으며,월1회 투여가 아닌 분기별로 투여할 수 있는 가능성을 시사합니다. 회사는 1건의 임상 2상 시험을 완료했으며, 현재 겸상적혈구증 치료제로 임상 3상 단계에 있습니다.
2022년 10월, 화이자는 겸상 적혈구 질환을 비롯한 소외된 환자 커뮤니티에 희망을 주고, 삶을 변화시키는 치료법을 발견, 개발 및 제공하는 바이오 제약사 Global Blood Therapeutics(GBT)를 인수하기로 결정했습니다. 제약 기업입니다.
겸상적혈구증 환자들에게 효과적인 치료법의 확립은 가장 중요한 과제입니다. 현재 치료법은 통증과 염증 완화, 혈관폐쇄성 크리즈 빈도 감소, 산소 공급 개선 등 삶의 질을 높이는 대증요법에 머물러 있습니다. 또한, 반복적인 수혈과 세포 치료를 통해 SCD 환자의 증상 관리를 유지하고 있으며, SCD는 미국 전역의 많은 사람들이 겪고 있는 현실입니다. 그러나 환자들은 특히 비전문 의료 서비스 제공업체로부터 양질의 종합적인 치료를 제공하도록 훈련받지 못한 경우가 많아 질이 낮은 치료를 경험하는 경우가 많습니다. 이는 전체적인 치료를 제공하기 위한 의료 인프라가 부족하여 만족스럽지 못한 증상 관리에 기여하고 있다는 것을 보여줍니다.
현재 치료법은 크게 약물요법과 비약물요법으로 나뉩니다. 약리요법으로는 DROXIA (hydroxyurea), ENDARI (L-glutamine), ADAKVEO (crizanlizumab), OXBRYTA (voxelotor) CASGEVY (exagamglogene autotemcel [exa-cel]) (Vertex Pharmaceuticals/CRISPR Therapeutics), LYFGENIA (Lovo-cel) (bluebird bio) 등이 있습니다. 통증 관리 약물은 오피오이드, 비스테로이드성 항염증제(NSAIDs), 아세트아미노펜, 코르티코스테로이드 등으로 분류됩니다. 급성 혈관폐쇄성 크리즈에는 일반적으로 오피오이드와 비스테로이드성 항염증제(NSAIDs)가 사용됩니다. 또한 비약물적 치료로는 인지행동치료, 바이오피드백, 이완법, 침술, 최면요법 등이 있습니다.
하이드록시 우레아는 경구용 약물로 겸상 적혈구를 감소시키고 SCD의 여러 합병증을 완화하거나 예방하는 것으로 나타났습니다. 하이드록시우레아는 SCD 치료제로서 최초로 FDA의 승인을 받았으며, 현재도 1차 선택 약물로 사용되고 있습니다. 하이드록시우레아로 증상이 충분히 조절되지 않는 경우, FDA가 승인한 새로운 약물을 하이드록시우레아 치료와 함께 병용하는 것이 일반적입니다.
2023년 12월, CRISPR 기반 유전자 치료제인 CASGEVY와 LYFGENIA가 겸상 적혈구 빈혈에 대해 FDA의 승인을 받았습니다. 이 두 치료법은 서로 다른 방식으로 작용하지만, 두 치료법 모두 노벨상을 수상한 CRISPR/Cas 9 유전체 편집 기술을 활용하고 있습니다.
오피오이드 진통제는 VOC 관리에서 통증 완화를 위한 첫 번째 선택으로 권장됩니다. 오피오이드 진통제에는 codeine, hydrocodone/paracetamol (hydrocodone/acetaminophen), hydrocodone/ibuprofen, oxycodone (and with codeine), morphine, hydromorphone, oxymorphone, methadone, diamorphine, fentanyl 등이 있습니다. 오피오이드는 일반적으로 의료기관에서 구입할 수 있으며, 4-6시간마다 모르핀을 정맥주사로 투여하는 경우가 많습니다. 그러나 진통제 오피오이드는 호흡억제, 변비, 구토, 메스꺼움, 가려움증, 두드러기, 중독, 금단증상 등의 부작용이 있어 처방이 제한되어 있습니다.
SCD 파이프라인에는 후기 및 중기 개발 단계에 있는 잠재적 약물이 있습니다. 현재 주요 진입기업과 각 신약 후보물질에는 Global Blood Therapeutics/Pfizer (Inclacumab), Forma Therapeutics/Novo Nordisk (etavopivat), Novo Nordisk (NDEC), Bluebird Bio (lovo-cel), Global Blood Therapeutics/Pfizer (GBT-601), Agios Pharmaceuticals (mitapivat), Bausch Health Americas (rifaximin), Editas Medicine (EDIT-301) 등이 있습니다.
간단히 말해서, SCD 치료제로서 몇 가지 가능성이 검토되고 있다는 것입니다. 예측 기간(2024-2034년) 동안 시장에 진입하는 위의 유망한 후보에 대해 언급하기에는 아직 이르지만, 이 시장의 미래는 밝다고 볼 수 있습니다. 궁극적으로, 이 약물은 향후 몇 년동안 SCD의 상황에 큰 변화를 가져올 것으로 보입니다. 이 치료 분야는 전 세계 의료비 지출 증가의 개선으로 인해 향후 몇 년동안 긍정적인 영향을 받을 것으로 예상됩니다.
겸상적혈구증(SCD)은 헤모글로빈에 영향을 미치는 일군의 평생 유전성 질환입니다. 적혈구 내 헤모글로빈 분자가 중합되어 적혈구를 낫 모양(또는 초승달 모양)으로 변형시키고(Hb S), 그 결과 특징적인 혈관 폐쇄성 사건과 용혈이 가속화되는 경향을 특징으로 하는 만성 용혈성 질환입니다.
SCD는 대혈관염으로 분류되지만, 중동맥염과 소동맥염도 동반됩니다. 겸상적혈구증은 상염색체로 유전되며, 동형접합체 또는 이중 이형접합체로 유전됩니다. 겸상적혈구증은 동형접합체 유전의 경우 겸상적혈구빈혈(SCA)이라고 합니다. 다른 SCD 유전자형으로는 헤모글로빈 SC병, 겸상적혈구 플러스 지중해빈혈, 겸상적혈구 제로 지중해빈혈(겸상적혈구 빈혈과 비슷한 중증도를 가짐), 헤모글로빈 SD 펀자브병, 헤모글로빈 SO 아랍병 등이 알려져 있습니다. 헤모글로빈 S(Hb S)는 B글로빈 유전자의 6번 위치가 글루탐산에서 발린으로 대체되어 정상 헤모글로빈(Hb A)과 다릅니다.
미국에서는 현재 출생 시 HbS 검사가 의무화되어 있습니다. 생후 6개월 동안 태아 헤모글로빈(HbF) 수치의 상승으로 인해 유아는 거의 보호받을 수 있으며, SCD는 보통 소아기 초기에 다양한 징후와 증상을 동반하여 나타납니다. 급성 및 만성 통증, 특히 혈관폐쇄성 크리즈는 SCD의 가장 특징적인 임상 증상이며, 종종 골수경색으로 인한 사지 장골의 골통이 나타납니다.
겸상적혈구증의 증상은 보통 생후 4개월까지는 나타나지 않습니다. 이 통증은 몇 시간에서 며칠 동안 지속될 수 있으며, 이러한 통증의 에피소드를 크리즈(crease)라고도 합니다. 매년 한 번씩 발병하는 사람도 있고, 매년 여러 번 발병하는 사람도 있습니다. 크리즈 증상은 심해져 입원해야 하는 경우도 있습니다. 겸상적혈구증의 모든 증상은 산소 부족으로 인한 것입니다.
겸상적혈구증의 진단은 결함 유전자와 헤모글로빈 세포를 분석하는 혈액 검사로 시작됩니다. SCD의 관리는 통증 에피소드 및 기타 합병증 예방과 치료에 중점을 두고 있으며, 태아기 및 영유아기에 조기 진단을 위한 다양한 선별검사 프로그램도 있습니다.
다양한 스크리닝 프로그램도 있어 산전 및 영아기 조기 진단에 도움을 주고 있습니다.
세계 주요 6개국의 겸상적혈구증(SCD) 시장에 대해 조사했으며, 시장 개요, 역학, 환자 동향, 새로운 치료법, 2034년까지 시장 규모 예측, 미충족 의료 수요 등을 조사하여 전해드립니다.
Report Summary
Market
Various key players, such as Pfizer, Agios Pharmaceuticals, Editas Medicine, Hoffmann-La Roche and others, are involved in developing therapies for SCD. The expected launch of emerging therapies and other treatments will lead to a significant increase in the market size during the forecast period [2024-2034].
The section dedicated to drugs in the SCD report provides an in-depth evaluation of pipeline drugs (Phase III and Phase II) related to SCD.
The drug chapters section provides valuable information on various aspects related to clinical trials of SCD, such as the pharmacological mechanisms of the drugs involved, designations, approval status, patent information, and a comprehensive analysis of the pros and cons associated with each drug. Furthermore, it presents the most recent news updates and press releases on drugs targeting SCD.
Marketed Therapies
CASGEVY (exagamglogene autotemcel): Vertex Pharmaceuticals/CRISPR Therapeutics
CASGEVY is a genome-edited cellular therapy consisting of autologous CD34+ hematopoietic stem cells (HSCs) edited by CRISPR/Cas9 technology at the erythroid-specific enhancer region of the BCL11A gene. CASGEVY is intended for one-time administration via a hematopoietic stem cell transplant procedure where the patient's own CD34+ cells are modified to reduce BCL11A expression in erythroid lineage cells, leading to increased fetal hemoglobin (HbF) production. HbF is the form of the oxygen-carrying hemoglobin that is naturally present during fetal development, which then switches to the adult form of hemoglobin after birth. CASGEVY has been shown to reduce or eliminate vaso-occlusive crises for patients with SCD.
In December 2023, Vertex Pharmaceuticals and CRISPR Therapeutics announced that the US FDA had approved CASGEVY (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 genome-edited cell therapy, for the treatment of sickle cell disease (SCD) in patients 12 years and older with recurrent vaso-occlusive crises (VOCs).
CASGEVY was later granted conditional marketing authorization in Great Britain by the UK. Medicines and Healthcare Products Regulatory Agency and by the National Health Regulatory Authority in Bahrain for patients 12 years of age and older with SCD characterized by recurrent vaso-occlusive crises or transfusion-dependent beta-thalassemia (TDT), for whom hematopoietic stem cell transplantation is appropriate and a human leukocyte antigen matched related hematopoietic stem cell donor is not available.
ENDARI (L-glutamine): Emmaus Life Sciences
ENDARI (L-glutamine) is an oral-administered pharmaceutical grade L-glutamine (PGLG), an amino acid formulation to relieve pain, swelling, and other complications of sickle cell anemia in adults and children 5 years and older. ENDARI reduces oxidant damage to red blood cells by improving the redox potential of nicotinamide adenine dinucleotide (NAD), a coenzyme identified as the primary regulator of oxidation.
ENDARI received Orphan Drug designation (ODD) in the US, Orphan Medicinal Product designation in the EU, and Fast Track designation (FTD) from the FDA.
In July 2017, the FDA approved ENDARI (L-glutamine oral powder) to reduce the severe complications of sickle cell disease in adult and pediatric patients aged 5 and older.
Emerging Therapies
Mitapivat: Agios Pharmaceuticals
Mitapivat is a novel, first-in-class oral small molecule allosteric activator of the pyruvate kinase enzyme. It has been shown to significantly upregulate both wild-type and numerous mutant forms of erythrocyte pyruvate kinase (PKR), increasing adenosine triphosphate (ATP) production and reducing levels of 2,3-diphosphoglycerate. In February 2022, the FDA approved PYRUKYND (mitapivat) to treat hemolytic anemia in adults with pyruvate kinase deficiency.
The company has initiated a Phase II/III trial to evaluate mitapivat in sickle cell patients.
In June 2023, Agios Pharmaceuticals announced that the Phase II portion of the global RISE UP study of mitapivat in sickle cell disease had met its primary endpoint of hemoglobin response for patients in both the 50 mg and 100 mg twice daily (BID) mitapivat arms.
Inclacumab: Pfizer
Inclacumab is a novel, fully human monoclonal antibody that selectively targets P-selectin. This protein mediates cell adhesion and is clinically validated to reduce pain due to VOCs in people with SCD. Preclinical results suggest that inclacumab can be a best-in-class option for reducing VOCs in people with SCD, with the potential for quarterly rather than monthly dosing. The company has completed one Phase II study, and it is currently in the Phase III stage of clinical development for the treatment of Sickle cell disease.
In October 2022, Pfizer competed the acquisition of Global Blood Therapeutics (GBT), a biopharmaceutical company dedicated to the discovery, development, and delivery of life-changing treatments that provide hope to underserved patient communities, starting with sickle cell disease.
An effective cure for a disease is the utmost requirement in SCD patients. Current therapies only provide symptomatic treatment such as relief in pain crises, inflammation, reduction in the frequency of vaso-occlusive crisis, improved oxygen supply, etc., enhancing quality of life. Recurring blood transfusion and cell therapies also sustain symptom management in SCD patients. SCD is the reality of many people across the US. Yet, patients often experience poor care, especially from non-specialist healthcare providers who may lack the training to provide good, comprehensive care. It shows the scarcity of healthcare infrastructure to provide holistic treatment, which fuels unsatisfactory symptom management.
The treatment pattern currently consists of different approaches classified into pharmacologic and nonpharmacological therapies. The pharmacological therapies, including DROXIA (hydroxyurea), ENDARI (L-glutamine), ADAKVEO (crizanlizumab), OXBRYTA (voxelotor) CASGEVY (exagamglogene autotemcel [exa-cel]) (Vertex Pharmaceuticals/CRISPR Therapeutics) and LYFGENIA (Lovo-cel) (bluebird bio). Pain management agents are segregated into opioids, nonsteroidal anti-inflammatory drugs (NSAIDs), acetaminophen, corticosteroids, etc. Acute vaso-occlusive crisis is generally managed using opioids and nonsteroidal anti-inflammatory drugs (NSAIDs). Further, non-pharmacological therapies include cognitive behavioral therapy, biofeedback, relaxation techniques, acupuncture, hypnosis, etc.
Hydroxyurea is an oral medicine that has been shown to reduce sickling and reduce or prevent several complications of SCD. This was the first medication approved by the FDA to treat SCD and is still used as a first-line treatment. If hydroxyurea does not control symptoms enough, the newer FDA-approved drugs are typically added on top of hydroxyurea treatment for combination therapy.
In December 2023, CASGEVY and LYFGENIA, CRISPR-based gene therapies, received approval from the FDA for sickle cell anemia. These therapies work in different ways; however, both therapies utilize the Nobel-winning CRISPR/Cas 9 genome editing technology.
Opioid analgesics are recommended as the primary choice of pain relief in VOC management. They include codeine, hydrocodone/paracetamol (hydrocodone/acetaminophen), hydrocodone/ibuprofen, oxycodone (and with codeine), morphine, hydromorphone, oxymorphone, methadone, diamorphine, and fentanyl. Opioids are generally available in healthcare settings and are often delivered as intravenous (IV) morphine every 4-6 h. However, the adverse effects of analgesic opioids include respiratory depression, constipation, vomiting, nausea, pruritus and hives, addiction, withdrawals, etc., limit their prescriptions.
The SCD pipeline possesses potential drugs in the late and mid-development stages. The current key players and their respective drug candidates include Global Blood Therapeutics/Pfizer (Inclacumab), Forma Therapeutics/Novo Nordisk (etavopivat), Novo Nordisk (NDEC), Bluebird Bio (lovo-cel), Global Blood Therapeutics/Pfizer (GBT-601), Agios Pharmaceuticals (mitapivat), Bausch Health Americas (rifaximin), Editas Medicine (EDIT-301), and others.
In a nutshell, a few potential therapies are being investigated for the management of SCD. Even though it is too soon to comment on the above-mentioned promising candidate to enter the market during the forecast period (2024-2034), it is safe to assume that the future of this market is bright. Eventually, the drug will create a significant difference in the landscape of SCD in the coming years. The treatment space is expected to experience a positive impact in the coming years owing to the improvement in the rise in the number of healthcare spending across the world.
SCD Disease Understanding and Treatment
SCD Overview
Sickle Cell Disease (SCD) is a group of lifelong inherited conditions that affect hemoglobin. It is characterized as a chronic hemolytic disorder marked by the tendency of hemoglobin molecules within red blood cells to polymerize and deform the red cell into sickle (or crescent) shape (Hb S), resulting in characteristic vaso-occlusive events and accelerated hemolysis.
SCD is classified as a large-vessel vasculitis but also involves medium and small arteritis; sickle cell disease is inherited in an autosomal fashion, whether in the homozygous or double heterogeneous state. Sickle cell disease is called sickle cell anemia (SCA) when there is an inheritance in the homozygous state. Other known SCD genotypes include hemoglobin SC disease, sickle beta plus thalassemia, sickle beta zero thalassemia (which has similar severity with sickle cell anemia), hemoglobin SD Punjab disease, hemoglobin SO Arab disease, and others. Hemoglobin S (Hb S) differs from normal hemoglobin (Hb A) because of the substitution of valine for glutamic acid in the sixth position in the B-globin gene.
Screening for HbS at birth is currently mandatory in the United States. For the first 6 months of life, infants are largely protected by elevated levels of fetal hemoglobin (HbF). SCD usually manifests early in childhood with various signs and symptoms. Acute and chronic pain, particularly vaso-occlusive crises, are the most distinguishing clinical features of SCD, often presenting as bone pain in the long bones of the extremities due to bone marrow infarction
Symptoms of sickle cell disease usually do not occur until the age of 4 months; the prevalent symptom includes painful episodes. This pain can last from a few hours to days; these painful episodes are also known as crises. Some people have one episode every year; others have many episodes each year. Crises can be severe, which leads to hospital stays. All the symptoms of sickle cell disease are because of a lack of oxygen.
SCD Diagnosis
Sickle cell disease diagnosis starts with a blood test that is analyzed for defective genes or hemoglobin cells. Various screening programs also help in the early diagnosis of the disease during the prenatal or infancy period. SCD management focuses on preventing and treating pain episodes and other complications.
Various screening programs are also there that help in early diagnosis of the disease during the prenatal or infancy period.
Blood tests
A person can go for the screening blood test to differentiate sickle hemoglobin (hemoglobin S) or another hemoglobin (such as C, B-thalassemia, E).
Newborn screening
Diagnosing SCD early in a child is very important to prevent further complications. All babies born in most developed countries are offered screening for sickle cell disease shortly following the birth. In newborn screening programs, blood from a heel prick test is collected in "spots" on a special paper. A second test should be done to confirm the diagnosis if the test is positive.
Prenatal diagnosis
Prenatal diagnosing is done on the baby before it is born to know whether the baby is suffering from any particular disease. Different types of tests that are used include:
Chorionic villus sampling
Fetal blood sampling
Amniocentesis
DNA analysis
This test can be used to investigate alterations and mutations in the gene that produces hemoglobin components. This test may be performed to determine whether someone has one or two copies of the Hb S mutation or has two different mutations in hemoglobin genes (e.g., Hb S and Hb C). Genetic testing is most often used for prenatal testing. This is done using a sample of amniotic fluid, the liquid in the sac surrounding a growing embryo, or a tissue taken from the placenta.
SCD Treatment
The treatment goals for sickle cell disease aims to relieve pain, prevent infections, and specifically manage complications. Treatment of Sickle cell disease can be divided into First line treatment and second line treatment. The first line treatment includes management of pain, vaso-occlusive crisis, and chronic symptoms by using various medications, Second line treatment includes gene therapy and bone marrow transplantation.
Patients with SCD use medications to make their disease less severe and treat symptoms. FDA approved medications include Voxelotor, Crizanlizumab, Hydroxyurea, L-glutamine, and others.
Moreover, NSAIDS, Opioids, Iron chelating agents, Antibiotics, Folic acid and others are used for the pain and other complications associated with SCD.
Acute sickle cell crises are managed primarily with drug therapy, psychologic supportive care, including oxygen. The standard treatment approach includes opioid analgesics, adequate hydration, and rest. Initial management should be aimed at providing rapid pain control. Pain management should follow the three-step "analgesic ladder" recommended by the World Health Organization for treating cancer-related pain. The choice of analgesic and dosage should be based on the severity of pain in the individual patient.
Patients with mild pain can often be treated with oral fluids and non-narcotic analgesics at home. Acetaminophen with or without codeine or oxycodone (Roxicodone), depending on pain severity, is started first. Nonsteroidal anti-inflammatory drugs (NSAIDs) can be used unless specifically contraindicated due to peptic ulcer disease, renal disease, or hepatic dysfunction. Narcotic analgesics can be used in patients with moderate to severe pain. Pain which is sufficiently severe that require an emergency department visit or hospitalization should be treated with stronger opioids.
Supportive care with oxygen therapy, hydration using fluid replacement, antibiotics in case of infection and transfusion should be considered while managing vaso-occlusive crises in SCD patients.
The major goals in chronic disease management are symptom control and prevention of disease complications. Hydroxyurea should be used in patients with severe complications who can reliably follow the regimen. Hydroxyurea reduces the frequency of painful crises and the need for blood transfusions in patients with recurrent painful crises.
The only chance for a cure for Sickle Cell Disease is bone marrow or stem cell transplantation. The bone marrow nurtures stem cells, which are early cells that mature into red and white blood cells and platelets. Normal hemoglobin may be produced by destroying the sickle cell patient's diseased bone marrow and stem cells and transplanting healthy bone marrow from a genetically-matched donor.
The SCD epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by total prevalent cases of SCD trait, total prevalent cases of SCD, diagnosed cases of SCD, age-specific prevalent cases of SCD and type-specific prevalence of SCD cases in the United States, EU4 countries (Germany, France, Italy, Spain) and the United Kingdom, and Japan from 2020 to 2034.
KOL Views
To stay abreast of the latest trends in the market, we conduct primary research by seeking the opinions of Key Opinion Leaders (KOLs) and Subject Matter Experts (SMEs) who work in the relevant field. This helps us fill any gaps in data and validate our secondary research.
We have reached out to industry experts to gather insights on various aspects of SCD, including the evolving treatment landscape, patients' reliance on conventional therapies, their acceptance of therapy switching, drug uptake, and challenges related to accessibility. The experts we contacted included medical/scientific writers, professors, and researchers from prestigious universities in the US, Europe, the UK, and Japan.
Our team of analysts at Delveinsight connected with more than 10 KOLs across the 6MM. By obtaining the opinions of these experts, we gained a better understanding of the current and emerging treatment patterns in the SCD market, which will assist our clients in analyzing the overall epidemiology and market scenario.
Qualitative Analysis
We perform Qualitative and Market Intelligence analysis using various approaches, such as SWOT analysis and Conjoint Analysis. 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.
Conjoint Analysis analyzes multiple approved and emerging therapies based on relevant attributes such as safety, efficacy, frequency of administration, designation, route of administration, and order of entry. Scoring is given based on these parameters to analyze the effectiveness of therapy. In efficacy, the trial's primary and secondary outcome measures are evaluated. Based on these, the overall efficacy is evaluated.
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. In addition, the scoring is also based on the route of administration, order of entry and designation, probability of success, and the addressable patient pool for each therapy. According to these parameters, the final weightage score and the ranking of the emerging therapies are decided.
Market Access and Reimbursement
Because newly authorized drugs are often expensive, some patients escape receiving proper treatment or use off-label, less expensive prescriptions. Reimbursement plays a critical role in how innovative treatments can enter the market. The cost of the medicine, compared to the benefit it provides to patients who are being treated, sometimes determines whether or not it will be reimbursed. Regulatory status, target population size, the setting of treatment, unmet needs, the number of incremental benefit claims, and prices can all affect market access and reimbursement possibilities.
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.