ALAD Porphyria (Aminolevulinic acid dehydratase porphyria) is a rare disease caused by the deficiency of 5-aminolevulinic acid dehydratase in the body. It results in an increase in ALAD in liver, blood, urine and other tissues. It is also associated with an increase in erythrocyte protoporphyrin and urine coproporphyrin. It is an autosomal recessive disease and it mostly affects only male patients. Treatment mainly includes enzyme inhibitors derived from processed red blood cells and symptomatic therapy. In symptomatic therapy, NSAIDS, anti-emetics and others are used to manage the disease.
ALAD Porphyria Treatment Market: Drivers and Restraints
FDA’s Office of Orphan Products Development (OOPD) and Orphan Products Clinical Trials Grants Program (formerly known as Orphan Products Grants) supports the clinical development of products to be used for treating rare diseases or conditions where no current therapy exists or where the proposed product will be superior to the existing therapy and this will fuel the growth of the ALAD porphyria treatment market. Along with this, FDA also provides grants for clinical studies on safety and/or effectiveness that will either result in or substantially contribute to market approval of these products. Other factors, such as the fact that the growing demand for orphan drugs is inelastic, meaning that it is relatively insensitive to any changes in prices, will also add to the growth of the ALAD Porphyria treatment market. Orphan drugs are developed for conditions where there are only a few effective treatment options available. A relatively small fraction of the affected population allows the cost of the drug to not severely strain the healthcare system and this will boost the growth of the ALAD porphyria treatment market. Orphan diseases are supported by well-organized patient-advocacy organizations, including the National Organization for Rare Disorders in the U.S. and EURORDIS in the EU. It may be politically untenable for a payer to refuse coverage for a promising new therapy on cost grounds.
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Healthcare providers do not have adequate information regarding the treatment and management of disease. Most people suffering from rare diseases do not have any treatment options available for their condition. Awareness and understanding of rare diseases is often low and many patients struggle to find adequate information about their condition. This particular factor will restrain the growth of the ALAD porphyria treatment market.
ALAD Porphyria Treatment Market: Segmentation
Segmentation based on Drug Type
- Enzyme Inhibitors
Segmentation based on Distribution Channel
- Hospital Pharmacies
- Retail Pharmacies/Drugstores
ALAD Porphyria Treatment Market: Market Overview
The global ALAD Porphyria Treatment market is witnessing rapid growth due to rising demand for ALAD Porphyria Treatment. There have been a number of initiatives aimed at raising awareness. The Global Genes Project, one of the leading rare and genetic disease patient advocacy organizations in the world, plans to sponsor grassroots activities to increase awareness internationally. The Rare Disease Day is a global initiative aimed at increasing awareness about rare diseases for both healthcare professionals and the population at large. Along with this, many government agencies, educational institutions, clinical centers and corporations have signed on as Rare Disease Day Partners. The future of ALAD Porphyria Treatment market is anticipated to grow with double digit CAGR during the forecast period.
ALAD Porphyria Treatment Market: Region-Wise Overview
The global ALAD Porphyria Treatment Market is segmented into North America, Europe, Asia -Pacific & Japan, Middle East and Africa regions and Latin America. North America dominates the global ALAD Porphyria Treatment Market due to growing high awareness and in North America, the USA is expected to be a major stakeholder due to the high penetration. Economic conditions in the APAC region are set to drive the ALAD Porphyria Treatment market to new heights. European and APAC are expected to be fastest growing regions due to rising awareness about ALAD Porphyria Treatment. Latin America is expected to grow at a stable pace due to increasing acceptance of ALAD Porphyria Treatment. The Middle East and Africa regions are showing significant growth because of growing awareness regarding ALAD Porphyria Treatment. However, developed countries will maintain their position in the ALAD Porphyria Treatment market because of increasing awareness and rising adoption.
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ALAD Porphyria Treatment Market: Key Participants
Some of the key participants in the ALAD Porphyria Treatment market include Recordati Rare Diseases Inc., Teva Pharmaceutical Industries Ltd., Novartis AG, Pfizer Inc., Johnson & Johnson, Mylan N.V., AstraZeneca Plc. and others.