确保患者获得罕见疾病的基因疗法:解决补偿和保险的挑战
Diane Berry1,2, Carolyn Hickey2, Lisa Kahlman3
1Sarepta Therapeutics, Inc., Cambridge, MA, USA.
Molecular therapy. Methods & clinical development
|February 3, 2025
概括
创新的基因疗法为罕见的遗传疾病提供了希望,但后勤和支付方面的挑战阻碍了患者的获取. 解决这些问题对于可持续的治疗和持续的治疗进步至关重要.
科学领域:
- 遗传学 遗传学 是一个
- 药物经济学 药物经济学
- 医疗物流 医疗物流
背景情况:
- 全球已发现超过1万种罕见疾病,其中80%是遗传性疾病.
- 目前,只有5%的罕见遗传疾病有FDA批准的治疗方法.
- 基因医学的进步需要改善获得创新疗法的机会.
相关概念视频
Gene Therapy
25.1K
Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...
25.1K
Issues And Trends In Healthcare Delivery System
5.6K
The issues and trends in healthcare delivery are constantly changing. The COVID-19 pandemic is one recent issue that wreaked havoc on healthcare systems, causing a shortage of healthcare workers, high demand for medicines and supplies, and increased medical expenditure due to a lack of insurance. Other issues include rising healthcare costs and care fragmentation.
Cost Containment
Payment for healthcare services has historically promoted adoption of costly and often unnecessary or inefficient...
Cost Containment
Payment for healthcare services has historically promoted adoption of costly and often unnecessary or inefficient...
5.6K
Combination Therapies and Personalized Medicine
4.8K
Combining two or more treatment methods increases the life span of cancer patients while reducing damage to vital organs or tissue from the overuse of a single treatment. Combination therapy also targets different cancer-inducing pathways, thus reducing the chances of developing resistance to treatment.
The combination of the drug acetazolamide and sulforaphane is a good example of combination therapy to treat cancer. The cells in the interior of a large tumor often die due to the hypoxic and...
The combination of the drug acetazolamide and sulforaphane is a good example of combination therapy to treat cancer. The cells in the interior of a large tumor often die due to the hypoxic and...
4.8K
What is Genetic Engineering?
73.4K
Overview
73.4K
Targeted Cancer Therapies
7.4K
The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
There are several types of targeted therapies against...
There are several types of targeted therapies against...
7.4K
iPS Cell Differentiation
2.6K
The ability of induced pluripotent stem cells or iPSCs to differentiate into most body cell types has stimulated repair and regenerative medicine research over the past few decades. iPSC-derived blood cells, hepatocytes, beta islet cells, cardiomyocytes, neurons, and other cell types can repair injuries or regenerate damaged tissue in diseases such as diabetes and neurodegenerative disorders.
2.6K


