LONDON, Oct. 2, 2023 /PRNewswire/ -- The Hemophilia Disease burden is witnessing a significant rise across a globe. This rare genetic disorder may occur at an early age and progress with time. Epidemiology study indicates that presently, more than 1.1 million individuals are affected with haemophilia and the statistics are reckoned to increase in the coming years. This disease is known to primarily impact men. Rigorous efforts are being taken by medical researchers, biotech and pharma firms, along with healthcare professionals to find effective haemophilia management, diagnostic, and treatment solutions.
Haemophilia is a rare hereditary condition that predominantly impact's an individual's capacity to clot blood. It is characterized by the deficiency of certain proteins known as clotting factors. It is worth mentioning that blood clotting is an essential biological function that primarily prevents excessive bleeding in case of severe injuries. It is a lifelong condition, but proper management may alleviate the symptoms to a large extent. Haemophilia A and Haemophilia B are the two sub-types of this ailment.
How is Haemophilia turning into a global concern?
Haemophilia epidemiological data suggests that more than 400 babies are born with haemophilia each year. This concerning data has escalated the demand for efficient haemophilia healthcare solutions. Once diagnosed, the patient is required to stay under complete medical surveillance throughout their lives. The fact that this ailment has no permanent cure makes it highly dangerous. Even minor injuries and cuts may be life threatening for haemophilic patients. Haemophilia patient care has become a top priority for numerous healthcare organizations worldwide.
Price and Market Access
Delving into the causes and symptoms of this rare disease:
There are two primary haemophilia causes namely genetic inheritance and spontaneous mutation. Also, this disease mainly occurs in men because the genes responsible for clotting factors are located on the X chromosome. But women can be the carrier of this disease by carrying one copy of the mutated gene but have a normal gene on their other X chromosome. Females do not experience any symptoms, but they pass the mutated gene to their offspring, making them highly prone to this genetic disorder.
Haemophilia symptoms may vary from person to person depending on the severity of their conditions. Prolonged bleeding even in case of minor injuries, easy bruising, swelling, joint pain, nose bleeds, blood in stool or urine, and intracranial bleeding are mainly experienced by the affected patients.
How to detect Haemophilia?
Early diagnosis plays a significant role towards ensuring proper haemophilia management. This disorder can be detected through a combination of various tests which are specified below-
Medical history and physical examination- Here the doctors enquire the patient about their personal and family medical history, including any instances of prolonged bleeding after injury or surgery. On the other hand, physical examination involves the proper evaluation of signs and symptoms that may indicate the presence of haemophilia.
Blood Tests- Clotting factor tests and activated partial thromboplastin time test are performed to measure the clotting factors in the blood and the time it takes for the blood to clot respectively.
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Genetic Testing- Through this testing, doctors can confirm the presence of the specific genetic mutation responsible for haemophilia. It allows them to easily determine the severity of the condition, identifying carriers among family members, and providing genetic counselling.
Apart from the aforementioned diagnostic techniques, differential diagnosis and certain specialized tests can be performed to detect this dreadful disease.
Navigating through the treatment landscape
Haemophilia patient care involves the administration of various drugs and therapies, along with physical and emotional support. A permanent cure for this ailment is yet to be developed but various drugs and therapies to alleviate the symptoms have successfully been introduced.
Here is a glimpse of the Haemophilia treatment vertical:
Clotting Factor Replacement therapy:
In this therapy, the deficit clotting factor is replaced through intravenous infusion of clotting factor concentrates. For haemophilia A (factor 8 deficiency), factor VIII concentrates are administered.
Emicizumab:
This monoclonal antibody has immense potential to treat Haemophilia A. It is primarily administered to prevent bleeding episodes in the patients.
Gene therapy:
Gene therapy aims to correct the genetic mutation responsible for hemophilia, potentially providing a long-term or even permanent solution to the condition.
Apart from these, several other FDA and EMA approved drugs and therapies are also prescribed by doctors for the effective treatment of this inherited disorder. DiseaseLandscape Insights has been a frontrunner when it comes to the research & development of advanced treatment entities against Haemophilia. It has been empowering the concerned bodies within depth disease market analysis which in turn assists them with treatment gaps identification tactics. DLI has also been supporting them with clinical trial management, regulatory compliance, and drug pricing strategies while offering them deep dive market access consultancy.
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Role of DLI in post launch scenario:
Many players seek healthcare consulting services from DLI during their drug development and launch stages. By leveraging DLI's product development services and product launch services, they successfully solidify their presence in the market. But their efforts do not end here. Even after the successful launch of the treatment products, it is crucial for them to engage in activities and adopt strategies through which they can ensure long time profits from these products.
DLI empowers them with post launch services that include commercial strategy analysis, product portfolio extension strategies, product pipeline analysis, along with other pharma consulting services. This allows players to get in depth insights about the efficacy of their drugs, thereby helping them with their long-term pricing and reimbursement decisions.
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Final Words:
Haemophilia is a rare genetic disorder than predominantly affect men. Although women can be the primary carriers of this disease. Patients affected with this condition lose the ability to clot blood. It means that in case of injuries, the bleeding won't stop automatically without medical intervention. Medical researchers and other healthcare professionals are making huge R&D investments to improve the diagnostic and treatment landscape pertaining to this ailment. DLI has been assisting them in their endeavours by providing them with disease overview, drug insights, and other related consulting services. The ongoing research activities may result in the development of a permanent cure for this devastating ailment in the coming years.
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