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Diamond-Blackfan Anemia - Market Insight, Epidemiology and Market Forecast - 2034

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    Report

  • 95 Pages
  • September 2024
  • Region: Global
  • DelveInsight
  • ID: 5979503
UP TO OFF until Dec 31st 2024

Key Highlights

  • Esophageal cancer occurs in the esophagus - a long hollow muscular tube that transports food from the neck to the stomach. Esophageal cancer, also known as esophagus cancer, develops in the cells that line the esophagus and occurs when a malignant tumor arises in the esophagus lining.
  • Esophageal malignancies are often discovered as a result of a person’s indications or symptoms. Examinations, testing, and a biopsy (a sample of esophageal cells) will be required to confirm the diagnosis if esophageal cancer is suspected. If cancer is discovered, more tests will be performed to establish the stage of the malignancy. The gold standard for identifying esophageal cancer is Gastroscopy.
  • Histologically, there are two forms of primary esophageal cancer: squamous cell carcinoma and adenocarcinoma; small cell carcinoma is an uncommon type of Esophageal Cancer. These several types of cancer arise in various types of cells in the esophagus. They evolve in distinct ways, necessitating therapeutic techniques tailored to each individual.
  • The staging system most often used for esophageal cancer is the American Joint Committee on Cancer (AJCC) TNM system, which is based on three key pieces of information, that is the extent of the tumor (T), spread to nearby lymph nodes (N) and spread (metastasis) to distant sites (M).
  • The exact cause of esophageal cancer is unknown; however, it is thought to be related to abnormalities (mutations) in the DNA of esophageal cells. The DNA of Esophageal Cancer cells frequently shows changes in many different genes; however, it is unclear if specific gene changes can be found in all Esophageal Cancers.
  • In 2023, the market size of esophageal cancer was highest in the US, accounting for approximately USD 450 million, which is further expected to increase by 2034.
  • In March 2021, the FDA approved KEYTRUDA (pembrolizumab) for use in combination with platinum and fluoropyrimidine-based chemotherapy for patients with metastatic or locally advanced esophageal or gastroesophageal carcinoma who are ineligible for surgical resection or definitive chemoradiation.
  • Esophageal cancer, a relatively uncommon cancer, has very limited information and guidance available, with few opportunities for people to share experiences. This ends up leading to a lack of awareness about the disease. Its symptoms are also often mild until the cancer advances, and symptoms can be easily confused with other illnesses and digestive problems.
  • The emerging pipeline for esophageal cancer patients consists of drugs in different lines of therapies, adjuvant, and neoadjuvant settings. It is estimated that potential drugs that can significantly change the market during the forecast period include zanidatamab, sintilimab, tislelizumab, and sotigalimab. These drugs are in the late stages of clinical development, and other players are evaluating their potential candidates in different stages of clinical development.

Report Summary

  • The report offers extensive knowledge regarding the epidemiology segments and predictions, presenting a deep understanding of the potential future growth in diagnosis rates, disease progression, and treatment guidelines. It provides comprehensive insights into these aspects, enabling a thorough assessment of the subject matter.
  • Additionally, an all-inclusive account of the current management techniques and emerging therapies and the elaborative profiles of late-stage (Phase III and Phase II) and prominent therapies that would impact the current treatment landscape and result in an overall market shift has been provided in the report.
  • The report also encompasses a comprehensive analysis of the Esophageal Cancer market, providing an in-depth examination of its historical and projected market size (2020 - 2034). It also includes the market share of therapies, detailed assumptions, and the underlying rationale for our methodology. The report also includes drug outreach coverage in the 7MM region.
  • The report includes qualitative insights that provide an edge while developing business strategies, by understanding trends, through SWOT analysis and expert insights/KOL views, including experts from various hospitals and prominent universities, patient journey, and treatment preferences that help shape and drive the 7MM Esophageal Cancer market.

Market

Various key players are leading the treatment landscape of Esophageal Cancer, such as Bayer, Hoffman-La Roche, Merck Sharp & Dohme, Eli lilly, Bristol Myers Squibb, Zymeworks, Innovent Biologics, Beigene, and others. The details of the country-wise and therapy-wise market size have been provided below.
  • Among the 7MM countries, the United States comprised the largest market size, accounting for ~40% of the total market size.
  • Among the EU4 and the UK, Italy captured the smallest market size, accounting for ~8% of the total market size of EU4 and the UK.
  • The esophageal cancer market size in Japan is expected to increase at a CAGR of 0.6% during the forecast period (2024-2034).
  • Among the upcoming emerging therapies in the first line, zanidatamab with chemotherapy combination is expected to capture the largest market in the United States.

Esophageal Cancer Drug Chapters

The section dedicated to drugs in the Esophageal Cancer report provides an in-depth evaluation of late-stage pipeline drugs (Phase III and Phase II) related to Esophageal Cancer.

The drug chapters section provides valuable information on various aspects related to clinical trials of Esophageal Cancer, 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 Esophageal Cancer.

Marketed Therapies

CYRAMZA (ramucirumab): Eli Lilly and Company

CYRAMZA, as a single agent or in combination with paclitaxel, is indicated for the treatment of patients with advanced or metastatic, gastric or gastroesophageal junction (GEJ) adenocarcinoma with disease progression on or after prior fluoropyrimidine- or platinum-containing chemotherapy. The recommended dosage of CYRAMZA as a single agent or combined with weekly paclitaxel is 8 mg/kg every 2 weeks, administered by IV infusion over 60 min. Ramucirumab is a VEGFR2 antagonist that binds VEGFR2 explicitly and blocks the binding of VEGFR ligands, VEGF-A, VEGF-C, and VEGF-D. As a result, ramucirumab inhibits ligand-stimulated activation of VEGFR2, thereby inhibiting ligand-induced proliferation and migration of human endothelial cells.

VITRAKVI (larotrectinib): Bayer

VITRAKVI (larotrectinib) is an oral TRK inhibitor for the treatment of adult and pediatric patients with solid tumors with an NTRK gene fusion without a known acquired resistance mutation that is either metastatic or where surgical resection will likely result in severe morbidity and have no satisfactory alternative treatments or have progressed following treatment. It is indicated for the treatment of adult and pediatric patients with solid tumors that have a neurotrophic receptor tyrosine kinase (NTRK) gene fusion without a known acquired resistance mutation, are metastatic or where surgical resection is likely to result in severe morbidity, and have no satisfactory alternative treatments or that have progressed following treatment.

Note: Detailed current therapies assessment will be provided in the full report of esophageal cancer…

Emerging Therapies

Zanidatamab: Zymeworks

Zanidatamab is given with chemotherapy plus or minus Tislelizumab as a combination therapy in HER2-Expressing Gastrointestinal (GI) Cancers, including Gastroesophageal Adenocarcinoma (GEA). Zanidatamab is given along with oxaliplatin (CAPOX) or 5-fluorouracil (5-FU) and cisplatin (FP) with or without tislelizumab. This emerging asset has a novel mechanism of action. It uses biparatropic binding as it targets two HER2 epitopes. The drug has also received fast-track designation in combination with SoC chemotherapy for first-line Gastroesophageal Adenocarcinoma. As far as safety is concerned, no severe treatment-related adverse events (TRAE) were observed. The most common Grade =3 TRAE was diarrhea which was manageable in the outpatient setting.

Sintilimab: Innovent Biologics

Sintilimab is an investigational PD-1 inhibitor developed by Innovent and Eli Lilly. The drug is currently in the Phase III stage of clinical development in combination with chemotherapy as first-line treatment in subjects with unresectable, locally advanced recurrent or metastatic esophageal squamous cell carcinoma. It is a first-line treatment option with cisplatin + paclitaxel or cisplatin + fluorouracil regimen for patients with unresectable, locally advanced recurrent or metastatic esophageal squamous cell carcinoma, as per the clinical assessment of sintilimab, a 12-month overall survival of 64% was found as compared to the 88% overall survival rate of zanidatamab. The Objective Response Rate (ORR) was also slightly lesser than zanidatamab. The safety profile of Sinitilimab was not as good as zanidatamab. A decrease in neutrophil count, WBCs, and hypokalemia was observed.

Esophageal Cancer Market Outlook

The therapies used for esophageal cancer include chemotherapy, targeted therapy, and immunotherapy. Chemotherapy uses medications to eradicate cancer cells, often by preventing cancer cells from growing, dividing, and proliferating. Targeted therapy for esophageal cancer includes HER2-targeted therapy and anti-angiogenesis therapy.

A chemotherapy regimen, or schedule, typically consists of a predetermined number of cycles administered over a predetermined period. A patient may be administered one medicine at a time or a mixture of drugs simultaneously. As previously stated, chemotherapy and radiation therapy are frequently used concurrently to treat esophageal cancer, a procedure known as chemoradiotherapy. Immunotherapy uses the body’s natural defenses to fight cancer by improving your immune system’s ability to attack cancer cells.

Two types of immunotherapy drugs are approved to treat adenocarcinoma and squamous cell carcinoma of the esophagus and the gastroesophageal junction. This cancer grows where the stomach and esophagus meet. Pembrolizumab (KEYTRUDA) and nivolumab (OPDIVO) are both checkpoint inhibitors that target the PD-1/PD-L1 pathway.

As few potential therapies are being investigated to manage esophageal cancer, predicting that the treatment space will experience significant reconstitution during 2024-2034 is safe.

Esophageal Cancer Disease Understanding and Treatment

Esophageal Cancer Overview

Esophageal cancer develops when cancer cells form in the esophagus, a tube-like tissue that connects the throat and stomach. The esophagus transports food from the mouth to the stomach. The cancer begins in the esophagus’s inner layer and can spread to other layers of the esophagus and other organs of the body (metastasis).

Typically, symptoms of esophageal cancer do not appear until the tumor has grown large enough to impede eating, swallowing, or digesting food. The most common symptom of esophageal cancer is difficulty swallowing, particularly a sensation that food is lodged in the throat; in some individuals, choking on food occurs. These symptoms develop with time, with greater discomfort while swallowing as the esophagus narrows due to cancer growth.

Esophageal Cancer Diagnosis

Esophageal malignancies are often discovered by indications or symptoms. Exams, testing, and a biopsy (a sample of esophageal cells) will be required to confirm the diagnosis; if cancer is identified, more tests will help establish the extent (stage). When diagnosed late, esophageal cancer has a terrible prognosis; however, if diagnosed early, curative therapy is feasible. Before symptoms cause individuals to seek medical assistance, the condition slowly advances. Effective presymptomatic screening procedures may enhance disease outcomes. Recent research has shed light on the early detection of esophageal cancer using blood testing, sophisticated endoscopic imaging, and artificial intelligence.

Esophageal Cancer Treatment

Everyone who has esophageal cancer receives some form of therapy. Several factors, including personal preferences, cancer stage, and overall health, determine the appropriate course of therapy for each patient. Many medical professionals regularly collaborate in cancer care to construct a patient’s comprehensive treatment plan, including various treatments.

Esophagectomy is the principal treatment for early-stage esophageal cancer, albeit its precise significance in superficial (T1A) cancers is still unclear, given the introduction of endoscopic mucosal therapy. A multimodal strategy for treating locally advanced cancers, comprising neoadjuvant chemotherapy or combination chemoradiotherapy (CRT) followed by surgery, is strongly recommended.

The cornerstone of contemporary esophageal cancer management is preoperative assessment. Preoperative staging accuracy is critical since the tumor board’s decisions on the use of multimodal therapy will be dependent on the precision and specifics of the clinical staging evaluation. Upper endoscopy, high-resolution contrast CT scan, FDG-PET scan, and EUS are all part of the standardized evaluation of a patient undergoing curative treatment for early-stage or advanced esophageal cancer.

Doctors typically recommend combining radiation treatment, chemotherapy, and surgery for a tumor that has not spread beyond the esophagus and lymph nodes. Locally advanced esophageal cancer is usually treated with radiation treatment, chemotherapy, and surgery. Radiation treatment and chemotherapy are often combined in “chemoradiotherapy.” Radiation treatment, chemotherapy, and other drug-based therapies are commonly used to treat metastatic esophageal cancer.

Esophageal Cancer Epidemiology

The Esophageal Cancer epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by total diagnosed incident cases, age-specific cases, histology-specific cases, gender-specific cases, mutation-specific cases, stage-specific cases and line wise treated cases of esophageal cancer in the United States, EU4 countries (Germany, France, Italy, Spain) and the United Kingdom, and Japan from 2020 to 2034.
  • The total diagnosed incident cases of esophageal cancer in the US comprised ~21,000 cases in 2023 and are projected to increase by 2034 at a CAGR of 1.9%, accounting for the second-highest diagnosed incident cases in the 7MM.
  • In 2023, as per the age-specific cases, the 65 and above segment accounted for the highest number of cases of esophageal cancer. In contrast, the < 45 age group accounted for the least number of cases in the United States.
  • In the United States, ~16,000 cases of esophageal cancer were found in case of males, whereas females accounted for ~4,000 cases of esophageal cancer in 2023.
  • As per the Histology-specific incident cases of esophageal cancer, non-squamous esophageal cancer cases accounted for ~7,500 cases of esophageal cancer, whereas ~3,000 cases of squamous esophageal cancer were found in 2023 in the United Kingdom.

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 Esophageal Cancer, 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 connected with more than 15 KOLs across the 7MM. We contacted institutions such as the Atrium Health’s Levine Cancer Institute, National Cancer Center Hospital, Complexo Hospitalario Universitario de Ourense, etc., among others. By obtaining the opinions of these experts, we gained a better understanding of the current and emerging treatment patterns in the Esophageal Cancer 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; for instance, in trials for Esophageal Cancer, important primary endpoints are overall survival rate, event-free survival, progression free survival, etc. Based on these parameters, 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, a final weightage score is decided, based on which the emerging therapies are ranked.

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.

Esophageal Cancer Report Insights

  • Patient Population
  • Therapeutic Approaches
  • Esophageal Cancer Market Size and Trends
  • Existing Market Opportunity

Esophageal Cancer Report Key Strengths

  • Ten-year Forecast
  • The 7MM Coverage
  • Esophageal Cancer Epidemiology Segmentation
  • Key Cross Competition

Esophageal Cancer Report Assessment

  • Current Treatment Practices
  • Reimbursements
  • Market Attractiveness
  • Qualitative Analysis (SWOT, Conjoint Analysis, Unmet needs)

Key Questions

  • Would there be any changes observed in the current treatment approach?
  • Will there be any improvements in Esophageal Cancer management recommendations?
  • Would research and development advances pave the way for future tests and therapies for Esophageal Cancer?
  • Would the diagnostic testing space experience a significant impact and lead to a positive shift in the treatment landscape of Esophageal Cancer?
  • What kind of uptake will the new therapies witness in coming years in Esophageal Cancer patients?

Table of Contents

1. Key Insights2. Report Introduction
3. Diamond Blackfan Anemia Market Overview at a Glance
3.1. Market Share (%) Distribution of Diamond Blackfan Anemia in 2023
3.2. Market Share (%) Distribution of Diamond Blackfan Anemia in 2034
4. Methodology5. Executive Summary
6. Disease Background and Overview
6.1. Introduction
6.2. Symptoms of Anemia
6.3. Congenital Anomalies in Dba
6.4. Clinical Features of Dba
6.5. Genetic Heterogeneity of Dba
7. Diagnosis
7.1. Diagnostic Algorithm of Diamond Blackfan Anemia
7.2. Diagnostic Recommendations
8. Treatment of Diamond Blackfan Anemia
8.1. Treatment Algorithm
8.2. Treatment Recommendations
9. Conclusion
10. Epidemiology and Patient Population
10.1. Key Findings
10.2. Assumptions and Rationales: 7MM
10.3. Total Incident Cases of Diamond Blackfan Anemia in the 7MM
10.4. United States
10.4.1. Total Diagnosed Prevalent Cases of Diamond Blackfan Anemia in the US
10.4.2. Cases of Dba by Mutated Genein the US
10.4.3. Cases of Dba by Congenital Anomaly in the US
10.4.4. Total Treated Cases of Dba in the US
10.5. EU4 and the UK
10.5.1. Total Diagnosed Prevalent Cases of Diamond Blackfan Anemia in EU4 and the UK
10.5.2. Cases of Dba by Mutated Gene in EU4 and the UK
10.5.3. Cases of Dba by Congenital Anomaly in EU4 and the UK
10.5.4. Total Treated Cases of Dba in EU4 and the UK
10.6. Japan
10.6.1. Total Diagnosed Prevalent Cases of Diamond Blackfan Anemia in Japan
10.6.2. Cases of Dba by Mutated Gene in Japan
10.6.3. Cases of Dba by Congenital Anomaly in Japan
10.6.4. Total Treated Cases of Dba in Japan
11. Patient Journey
12. Market Analysis
12.1. Key Findings
12.2. Total Market Size of Diamond Blackfan Anemia in the 7MM
12.3. US Market
12.3.1. Total Market Size of Diamond Blackfan Anemia in the US
12.3.2. Market Size of Diamond Blackfan Anemia by Therapies in the US
12.4. EU4 and the UK Market
12.4.1. Total Market Size of Diamond Blackfan Anemia in EU4 and the UK
12.4.2. Market Size of Diamond Blackfan Anemia by Therapies in EU4 and the UK
12.5. Japan Market
12.5.1. Total Market Size of Diamond Blackfan Anemia in Japan
12.5.2. Market Size of Diamond Blackfan Anemia by Therapies in Japan
13. Kol Views14. Market Barriers15. Market Drivers16. SWOT Analysis17. Unmet Needs
18. Appendix
18.1. Bibliography
18.2. Report Methodology
19. Publisher Capabilities20. Disclaimer21. About the Publisher
List of Tables
Table 1: Summary of DBA Market and Epidemiology (2020-2034)
Table 2: Congenital Anomalies in DBA
Table 3: Clinical Features in Patients With DBA
Table 4: Various Types of DBA and Their Associated Genetic Mutations
Table 5: Recommended diagnostic tests in patients with suspected DBA
Table 6: Differential Diagnoses of DBA Syndrome
Table 7: Treatment Algorithm of DBA
Table 8: Recommendations for Transfusion Support in DBA patients
Table 9: Recommendations for Steroid Treatment in DBA Patients
Table 10: Recommendations for chelation therapy in DBA patients
Table 11: Recommendations for Allogeneic HSCT
Table 12: Total Diagnosed Prevalent of DBA in the 7MM (2020-2034)
Table 13: Total Diagnosed Prevalent Cases of DBA in the United States (2020-2034)
Table 14: Cases of DBA by Mutated Gene in the United States (2020-2034)
Table 15: Cases of DBA by Congenital Anomaly in the United States (2020-2034)
Table 16: Total Treated Cases of DBA in the United States (2020-2034)
Table 17: Total Diagnosed Prevalent Cases of DBA in EU4 and the UK (2020-2034)
Table 18: Cases of DBA by Mutated Gene in EU4 and the UK (2020-2034)
Table 19: Cases of DBA by Congenital Anomaly in EU4 and the UK (2020-2034)
Table 20: Total Treated Cases of DBA in EU4 and the UK (2020-2034)
Table 21: Total Diagnosed Prevalence Cases of DBA in Japan (2020-2034)
Table 22: Cases of DBA by Mutated Gene in Japan (2020-2034)
Table 23: Cases of DBA by Congenital Anomaly in Japan (2020-2034)
Table 24: Total Treated Cases of DBA in Japan (2020-2034)
Table 25: Total Market Size of DBA in the 7MM, in USD million (2020-2034)
Table 26: Total Market Size of DBA in the United States, in USD million (2020-2034)
Table 27: Market Size of DBA by Therapies in the United States, in USD million (2020-2034)
Table 28: Total Market Size of DBA in EU4 and the UK, in USD million (2020-2034)
Table 29: Market Size of DBA by Therapies in EU4 and the UK, in USD million (2020-2034)
Table 30: Total Market Size of DBA in Japan, in USD million (2020-2034)
Table 31: Market Size of DBA by Therapies in Japan, in USD million (2020-2034)
List of Figures
Figure 1: Child Suffering With DBA
Figure 2: Symptoms of Anemia
Figure 3: Pathophysiology of DBA
Figure 4: Diagnostic Algorithm for DBA
Figure 5: Total Diagnosed Prevalent of DBA in the 7MM (2020-2034)
Figure 6: Total Diagnosed Prevalent Cases of DBA in the United States (2020-2034)
Figure 7: Cases of DBA by Mutated Gene in the United States (2020-2034)
Figure 8: Cases of DBA by Congenital Anomaly in the United States (2020-2034)
Figure 9: Total Treated Cases of DBA in the United States (2020-2034)
Figure 10: Total Diagnosed Prevalent Cases of DBA in EU4 and the UK (2020-2034)
Figure 11: Cases of DBA by Mutated Gene in EU4 and the UK (2020-2034)
Figure 12: Cases of DBA by Congenital Anomaly in EU4 and the UK (2020-2034)
Figure 13: Total Treated Cases of DBA in EU4 and the UK (2020-2034)
Figure 14: Total Diagnosed Prevalent Cases of DBA in Japan (2020-2034)
Figure 15: Cases of DBA by Mutated Gene in Japan (2020-2034)
Figure 16: Cases of DBA by Congenital Anomaly in Japan (2020-2034)
Figure 17: Total Treated Cases of DBA in Japan (2020-2034)
Figure 18: Total Market Size of DBA in the 7MM, in USD million (2020-2034)
Figure 19: Total Market Size of DBA in the United States, in USD million (2020-2034)
Figure 20: Market Size of DBA by Therapies in the United States, in USD million (2020-2034)
Figure 21: Total Market Size of DBA in EU4 and the UK, in USD million (2020-2034)
Figure 22: Market Size of DBA by Therapies in EU4 and the UK, in USD million (2020-2034)
Figure 23: Total Market Size of DBA in Japan, in USD million (2020-2034)
Figure 24: Market Size of DBA by Therapies in Japan, in USD million (2020-2034)
Figure 25: Unmet Needs
Figure 26: Health Technology Assessment
Figure 27: Reimbursement Process in Germany
Figure 28: Reimbursement Process in France
Figure 29: Reimbursement Process in Italy
Figure 30: Reimbursement Process in Spain
Figure 31: Reimbursement Process in the United Kingdom
Figure 32:Reimbursement Process in Japan