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Alagille Syndrome
Industry News

Impact of rare diseases greater than previously estimated

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The number of rare diseases worldwide is greater than the previously estimated 7000 figure, according to Global Genes’ NEXT 2022: Redefining the Possible report. Research conducted by RARE-X, a Global Rare Disease Data Platform concluded there are as many as 10,869 rare diseases globally.

“Looked at collectively—and in particular with the new knowledge that the true number of rare conditions has been significantly undercounted—rare diseases represent a major area of unmet need and, as we move beyond the pandemic, should become more of a global health priority,” said Craig Martin, CEO of Global Genes in a press release. “The NEXT Report also reinforces that rare diseases are becoming an exciting proving ground for promising biotechnologies, such as gene therapy, gene editing, and other regenerative medicine approaches—and a place where new models for collaborative data-sharing and drug development may lead to more productive and efficient pathways to advance therapies to patients.”

A report published earlier this month in the Journal of Rare Diseases also found that the economic burden for rare conditions is as much as 5x times higher than the cost for people without rare diseases.

Although the burden of rare diseases is greater than previously estimated, advances in drug development and treatments have been substantial. In 2021, there was a 28% increase year-over-year in funding for rare disease drug development, which according to the report, outpaced most biotech and health segments. The FDA’s accelerated assessment program has also helped many new drugs gain approval on an accelerated schedule. From 2015 to 2020, 84% of drugs approved under the program had orphan designations.

The report also highlighted the importance patient organizations have in creating “frameworks for rare disease health access to address inequities and gaps” as well as their role in drug development and research. In a recent article for Rare Disease 360, Roberta Smith, Alagille Syndrome Alliance President, underscored the importance of advocacy work in the rare disease space.

“Rare Disease advocacy led by patient groups, communities, individuals, and families has led to remarkable outcomes like new treatments and therapies, new laws and policies that make living with a rare disease easier, improved medical care, advanced science and research, and much more,” she said. “Individuals, families, and caretakers have an extraordinary perspective that pharma, biotech, medical professionals, lawmakers, and scientists and researchers are hungry for. “

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