Gene therapies will provide a valid treatment option for patients that have developed resistance to chemotherapies across several cancer indications. With patient access set to increase over the next decade, these therapies are expected to eliminate the need for further treatments and reduce the cost of disease management. Total sales of in-vivo gene therapies are forecast to reach over $7bn by 2027 across the 8MM*, with the therapeutic oligonucleotide market forecast to reach $4.5bn by 2027 globally.

Scope

Overview of gene therapy including classification of therapy and technologies, regulatory and market access details, product & company profiles. Quotes from US- and 5EU-based key opinion leaders and researchers. Key topics covered for gene therapy in oncology in the 8MM include trends, value chain, market analysis, opportunities, challenges and unmet needs and high-value deals. Pipeline analysis: Comprehensive data split across different phases and indications, emerging novel trends under development, and analysis of the most promising late-stage pipeline drug...

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Fanconi Anemia - Drugs In Development, 2021, provides an overview of the Fanconi Anemia (Hematological Disorders) pipeline landscape.Fanconi anemia (fan-KO-nee uh-NEE-me-uh), or FA, is a rare, inherited blood disorder that leads to bone marrow failure. The disorder also is called Fanconi’s anemia. FA is a type of aplastic anemia. In aplastic anemia, the bone marrow stops making or doesn't make enough of all three types of blood cells. Low levels of the three types of blood cells can harm many of the body's organs, tissues, and systems. Treatment is recommended for significant cytopenias, such as hemoglobin less than 8 g/dL, platelets fewer than 500/μL.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Fanconi Anemia - Drugs In Development, 2021, provides comprehensive information on the therapeutics under development for Fanconi Anemia (Hematological Disorders), complete with analysis by stage of ...

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Hypereosinophilic Syndrome - Drugs In Development, 2021, provides an overview of the Hypereosinophilic Syndrome (Hematological Disorders) pipeline landscape.The hypereosinophilic syndrome (HES) is a disease characterized by a persistently elevated eosinophil count (≥ 1500 eosinophils/mm³) in the blood for at least six months without any recognizable cause, with involvement of either the heart, nervous system, or bone marrow. HES is a diagnosis of exclusion, after clonal eosinophilia (such as leukemia) and reactive eosinophilia (in response to infection, autoimmune disease, atopy, hypoadrenalism, tropical eosinophilia, or cancer) have been ruled out.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Hypereosinophilic Syndrome - Drugs In Development, 2021, provides comprehensive information on the therapeutics under development for Hypereosinophilic Syndrome (Hematological Disorders), complete with analysis b...

Summary

A biomarker is a defined molecular characteristic, for example a genetic mutation, that can tell a physician more about a patient’s disease state, or how they might respond to treatment. Predictive biomarkers indicate how likely a patient is to respond to a treatment, and are used in clinical decision making. In some cases, a patient must possess a particular biomarker to receive a particular drug. For example, breast cancer patients must test positive for HER2 in order to receive anti-HER2 therapy. Companion diagnostics (CDx) are in vitro diagnostics used to measure biomarkers deemed essential for the use of a particular drug, and their use is on the rise, particularly in oncology. In fact, over 90% of FDA-approved CDx are for cancer indications. This is likely because oncology treatments have a relatively high ‘failure’ rate compared to other diseases, and treating a patient with the right drug is often very time-sensitive due to the aggressive nature of some cancers. Biomarkers are of high interest to multiple stakeholders in oncology and have been a...

Summary

Since hitting the market in 2015, drugs that target poly (ADP-ribose) polymerase (PARP) have transformed the clinical management of cancers harboring BRCA-mutations. As more PARP inhibitors have been commercialized, drug developers are turning their attention towards broadening drug labels and exploring combination strategies. This report covers the management of PARP inhibitors in the US, Europe, and Asia, focusing on efficacy and safety comparisons for approved products, current and future indications, and research and development strategies.

Scope

This report combines key opinion leader insight with in-house analyst expertise and research to provide an insight-rich look at PARP inhibitors in the US, Europe, and Asia. Components of the report include -
- Overview of PARP Inhibitors in Oncology: Background, mechanism of action, and clinical development of PARP inhibitors.
- Epidemiology Analysis: Which current and future disease spaces are relevant to PARP inhibitors.
- Marketed PARP Inhibitors: How the currently approved PARP ...

Summary

This report provides an overview of the pipeline landscape for hereditary hematological disorders. It provides comprehensive information on the therapeutics under development and key players involved in therapeutic development for thalassemia, hemophilia B and sickle cell anemia, and features dormant and discontinued products.

Thalassemia refers to a group of inherited blood disorders that affect the body's ability to produce hemoglobin and red blood cells. Symptoms include paleness, frequent infections and jaundice. Predisposing factors include family history. Treatment includes blood transfusions and bone marrow transplant. There are 40 products in development for this indication.

Hemophilia B is a hereditary bleeding disorder caused by a lack of blood clotting factor IX. Symptoms include bleeding into joints and associated pain and swelling, bruising, nosebleeds and prolonged bleeding from injuries and surgery. Treatment includes replacing the defective clotting factor. There are 42 products in development for this indication.

Sic...

Summary

Recent trends in drug development have seen the price of new medicines continually increase, and there is a need for cheaper medicines to achieve global sustainable healthcare. Biologic drugs are some of the most expensive medicines to develop, as they are large complex molecules. Biosimilars are highly similar to approved biologic drugs, with no clinically relevant differences, and aim to achieve the same clinical results at a lower price than their originator molecules. This is a key way in which the cost-savings required to promote sustainability can be achieved.

In oncology, biosimilar versions of six biologic drugs are now available. The EMA has pioneered regulatory acceptance of biosimilars, and has approved many more biosimilars than the FDA. Beyond the major markets of the US and 5EU (France, Germany, Italy, Spain and UK), two emerging markets are important in the oncology biosimilars landscape: India, with the most approved biosimilars globally, and South Korea, home to major biosimilar developer Celltrion.

This report provides an asses...

Summary

Market access of oncology medicines is increasingly challenging due to the rising cost of new and innovative medicines, more stringent scientific and value assessments and economic pressures on national budgets and health systems. Although, 5EU markets have implemented patient access schemed to ensure early access to innovative oncology medicines with high unmet need, the increasing requirements for improved clinical benefits and better cost-effectiveness of medicines has resulted in more stringent pricing and reimbursement of medicines leading to access issues such as delays in patient access due to negotiations.

In this report, GlobalData provides an overview of market access, pricing and reimbursement in the 5EU (France, Germany, Italy, Spain and UK) including key KOL and payers perspectives throughout. KOLs and Payers also provide insights into the market access trends, challenges, drivers and barriers. KOLs and Payers also provide insights on unmet needs in oncology and overall current and future outlook.

Scope

The report comb...