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Why the pharma industry is digging deep in rare disease

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The U.S. rare disease market is cultivated by a set of regulatory incentives like the Orphan Drug Act and others that allow pharmaceutical companies to reap the benefits of aiming at targets with very small patient populations. Many pharmas are taking the bait, which is good for patients and adds up to a quickly growing segment of the life sciences sector.

It comes down to this: While each rare disease is only present in a small number of patients, about 10% of the U.S. population is afflicted with one of these diseases. With 30 million Americans and many more globally suffering from rare conditions, the market is wide and deep, accounting for more than 700 medicines in the biopharma pipeline.

AstraZeneca, one of the leaders in the space, saw rare disease drug sales contribute $7.1 billion to its revenue total in 2022, growing 10% from the previous year. Pfizer has a strong pipeline of rare disease candidates with 10 currently in the clinic, three of which are late-stage gene therapies, and executives have cited the space as a major contributor to future growth.

The global rare disease treatment market reached more than $195 billion in 2022, according to a report from GMI Insights. With a conservatively estimated compound annual growth rate (CAGR) of more than 8.5%, GMI expects the market value to reach $435 billion by 2032. Other reports in the past have estimated the decade’s CAGR to surpass 12%.

That growth is less of a surprise when considering how wide open the rare disease space really is. With more than 7,000 rare diseases and 400 million people affected by them worldwide, about 90% of these conditions have no treatment, according to another rare disease leader, Roche.

By the numbers



Rare disease medicines in the biopharma pipeline


~350 million

People with rare diseases around the world



Of rare diseases are genetic

New approaches

Plenty of rare disease research happens outside of the Big Pharma realm, but with the growth of the market, giant drugmakers are bringing some of their assets in through M&A. AstraZeneca’s $39 billion purchase of Alexion in 2021 is one of the biggest in recent years. Amgen’s $28 billion acquisition of Horizon Therapeutics sent the M&A watchers abuzz as the largest merger of the year in 2022.

And while companies like Pfizer have been pushing into this rare disease market growth through their pipeline, the company has also pivoted to a more nuanced approach to the wide and unfocused area.

“We have rethought our approach to rare disease and will move from having a standalone research unit to aligning key programs with other therapeutic areas,” said Dr. Mikael Dolsten, president of worldwide R&D and medical at Pfizer, on the company’s most recent quarterly earnings call. “We plan to externally advance rare disease programs that do not fit into a core therapeutic area of focus — at the same time, we plan to tap into the expanding external innovation ecosystem by actively pursuing biotech innovation and emerging innovation that fits strategically and accessing external assets that are differentiated.”

Fueling the growth of rare disease research is the faster pace of innovation and lower costs of genetic solutions. For example, the cost of genomic sequencing has fallen from billions of dollars for a single human genome in the early 1990s to less than $1,000 currently. Genetic conditions account for about 80% of all rare diseases, according to a report from IQVIA.

A long-term study of the FDA’s orphan drug program found that 5,099 treatments were granted the designation between 1983 and 2021, and each decade grew stronger, more than doubling in each era. About 37% of these were for oncology, 13% in neurology and 9% in infectious diseases. Across rare diseases, almost a third were granted for pediatric designations.

“The dramatic rise in overall orphan designations over the past four decades suggests we may continue to see an upward trajectory in designations leading to an increased number of approvals for drugs and biologics designed specifically for diagnosing, preventing, and treating rare diseases in the coming decades,” wrote the authors of the 2021 article published in the Orphanet Journal of Rare Diseases.

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