‘Major’ challenges require new approaches to rare disease research: Tufts

By Melissa Fassbender

- Last updated on GMT

(Image: Getty/Urupong)
(Image: Getty/Urupong)

Related tags Tufts Rare disease Drug development Wearables Virtual clinical trials

More than half of new drug approvals last year were for rare diseases – which face long cycle times and other operational challenges that are necessitating the rapid adoption of new solutions, per a recent Tufts report.

The drug development space for decades has been characterized by “longer cycle times, very high failure rates, rising development costs and declining revenue potential for commercialized therapies,”​ said Ken Getz, associate professor and director of sponsored research at Tufts CSDD.

“As a result, the overall return on development investment has been declining steadily,”​ he explained, noting that rare diseases represent even smaller market opportunities.

Globally, rare disease drug development currently accounts for nearly one-third of all drugs in active research and development, according to a recent report from the Tufts Center for the Study of Drug Development (CSDD).

The share of new drug approvals worldwide for rare diseases doubled from 29% of all approvals in 2010 to 58% in 2018, per the report.

“The results of our study indicate that rare disease development programs have much longer overall durations, driven in large part by major challenges associated with investigative site identification and patient identification and enrollment,” ​Getz told us.

On average, the duration for rare disease drug development takes four years longer than non-rare diseases. Additionally, Phase I clinical trials for rare diseases involved six times the number of investigative sites to recruit a quarter of the number of patients, according to the report.

To address these issues, Getz noted that the industry has been piloting several initiatives designed to improve site and patient identification. This includes the use of more robust data and analytics, including new risk-assessment approaches and artificial intelligence (AI). 

“The industry has also been deploying new solutions to improve enrollment including convenience-enhancing transportation assistance; home nursing networks; remote and virtual clinical trials; and the use of mobile and wearable devices,”​ he said.

Getz explained, “Although direct development costs for rare disease therapies are lower, the longer cycle times — and operating challenges associated with them — will necessitate more rapid adoption of many of the solutions that companies are only piloting at this time.”

The study results came as no surprise, and confirm the researchers’ hypotheses, said Getz, who noted that the “very high” ​percentage of rare disease therapies among all 2018 new drug and biologic approvals indicates the timeliness of the study.

Related topics Clinical Development

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