On one side of the equation pharmaceutical companies maintain that if drug prices are restricted it will have a negative impact on innovation. Health economists, on the other hand, maintain that it is possible to have it both ways. They claim that investing in research and innovative companies will lower prices.
A recent article in Biospace reports that rare diseases afflict roughly 300 million people in the world, one-third of whom live in the United States. As of January 2023, approximately 95% of rare diseases did not have a treatment approved by the FDA.
A little-known fact is that a 2019 study found that orphan drug trials cost researchers a total of $166 million for each approved drug. In addition, in 2023 general market prices increased 35%.
There was an encouraging increase in biotech investment during 2020 and 2021 but a downward spiral in 2022. Although 50% of rare disease patients in the United States are children, between the years 2010 and 2018, only 33% of orphan drugs were approved for pediatric patients.
His Son Was Diagnosed with Spastic Paraplegia 50
Elpida CEO Terry Pirovolakis founded the company soon after his son received a diagnosis of spastic paraplegia 50, a neurological disorder. Elpida’s SPG50 drug called Melpida is now in clinical trials.
U.S. Government and Industry
A sizable amount of early-stage R & D originates with the U.S. government. The number of rare disease treatments, namely small molecule drugs, gene and cell therapies, and antibodies has increased.
The Alliance for Regenerative Medicine keeps a watchful eye on acquisitions and investments worldwide and reports that a total of $11.7 billion has been invested in gene and cell therapies as of 2023.
About the California Institute for Regenerative Medicine (CIRM)
The CIRM funds clinical trials, research, and development of drugs for rare conditions. It has a membership in the Bespoke Gene Therapy Consortium (BGTC).
BGTC is a public-private partnership that includes the Food and Drug Administration (FDA), National Institute of Health (NIH), patient advocacy groups (PAG), pharmaceutical companies and venture funds.
A Glimmer of Hope
Currently there are over 700 treatments for rare diseases seeking approval according to PhRMA. Takeda, Genethon, and Sanofi each have a minimum of 10 drugs under review in their pipeline.
As of 2023, a total of 1,329 gene and cell therapy active trials involving rare diseases were being reviewed. Forty-four percent of those trials are in Phase I.
Although the number of families seeking treatment has increased, Pirovolakis cautioned that fewer companies are targeting rare diseases, and many programs are being discontinued. Pirovolakis emphasized that treatments for these young patients are critically underfunded, especially with respect to biotech investment. He added that support from the biotech community, philanthropists, and governments is urgently needed.
