根据最新行业数据，美国生物制药公司目前有37款用于帕金森病的新药正在研发之中，其中23个用于该疾病治疗，11个用于相关症状，还有3个是诊断试剂。据来自PhRMA的报道，所有23个新产品目前正处于临床试验或FDA审评中。
全球大约有1000万人患有这种慢性及进行性神经疾病，其中美国多达150万人。每年大约有60万新增病例，这一数字并未反映更多未被检测的病例，据预测，如果干预措施没有任何变化，到2040年帕金森病的流行程度将是现在的三倍多，PhRMA如是说道。
最新报道罗列了一系列被用来治疗和处理这种疾病的方法。其中包括：
基因治疗药物，药物中含有一种腺相关病毒(AAV)载体，它可以将芳香族氨基酸脱羧酶(AADC)基因输送到控制运动的大脑部分细胞中；
一种潜在的一线药物，该药物以大脑中一种受体为靶点，帕金森病患者大脑中这种受体常发生异常；
分子影像剂，它通过单光子发射计算机断层扫描(SPECT)来帮助诊断帕金森病；
一种潜在的一线治疗药物，用于帕金森病精神错乱(PDP)；
新的给药机制，包括一种鼻腔制剂和一种肠道凝胶。
PhRMA的报道也回顾了帕金森病的经济影响。仅在美国，每年预计至少花费144亿美元，81亿美元用于医疗，63亿美元归因于这种疾病间接成本。美国帕金森病患者承受的疾病相关费用平均每人每年为22800美元，这一数字相比不患这种病的人高12800美元，预测表明如果这种疾病的进程可以减缓50%，这将减少35%的额外成本，PhRMA报道说。
“帕金森病的经济负担及服务与支持的需求正因世界人口老龄化而成指数增长。更多合作将会更好地帮助满足帕金森病患者的需求，让我们继续努力应对这一全球性的公共卫生挑战。”PhRMACEOJohnJCastellani说。
US biopharma: 37 new Parkinson's disease medicines now in R&D
US biopharmaceutical companies now have 37 new medicines in development for Parkinson’s disease – 23 to treat the disease, 11 for related conditions and three diagnostics, according to new industry data.
All 23 new products are now either in clinical trials or under by the US Food and Drug Administration (FDA), says the report, from research-based industry group the Pharmaceutical Research and Manufacturers of America (PhRMA).
Around 10 million people worldwide are estimated to be living with this chronic and progressive neurological condition, including up to 1.5 million in the US. Each year, approximately 600,000 additional patients are newly diagnosed, which does not reflect the thousands of cases that go undetected, and it is forecast that, without any change, the preva1ence of Parkinson’s will more than double by 2040, says PhRMA.
The new report looks at the range of approaches being pursued to tackle the disease. Examples include:
- a gene therapy comprising an adeno-associated virus (AAV) vector that delivers the gene for aromatic L-amino acid decarboxylase (AADC) to cells in a part of the brain that controls movement;
- a potential first-in-class medicine that targets a receptor found in the brain where degeneration and abnormality are often seen in Parkinson’s disease;
- a molecular imaging agent that uses single photon emission computed tomography (SPECT) to aid in diagnosis of Parkinson’s:
- a potential first-in-class treatment for Parkinson’s disease psychosis (PDP); and
- new delivery mechanisms of currently-approved treatments, including an intranasal formulation and an intestinal gel.
PhRMA's report also examines the economic consequences of Parkinson’s. These are estimated, in the US alone, to be at least $14.4 billion a year, with $8.1 billion in medical expenses and $6.3 billion in indirect costs attributed to the disease.
Also, US patients with Parkinson’s incur annual disease-related expenses averaging $22,800 per capita, which is $12,800 higher than someone who does not have the disease, and estimates suggest that if the condition’s progression could be slowed by 50%, this would lead to 35% reduction in excess costs, it says.
“The economic burden of Parkinson’s disease and the need for services and support are increasing exponentially due to the world’s aging population. More collaboration throughout the biomedical ecosystem will help better meet the needs of people living with Parkinson’s, and allow us to continue to make strides against this global public health challenge,” said PhRMA CEO John J Castellani.
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