Challenges in Orphan Drug Development

Thanks to a combination of a number of scientific, financial, and regulatory hurdles that orphan drugs must overcome in order to treat the rare disease, they perceive themselves as less favorable. Here are some of the foremost difficulties raised:

1️. Cost of Development

• Orphan drugs involve huge laboratories and clinical trials coming into play, which can amount to billions of dollars.

• Given the small patient population, recouping the development costs becomes extremely difficult.

2️. Limited Patient Population

• Fewer than 200,000 people in any one geographical region are affected; hence, recruiting enough subjects for clinical trials becomes practically impossible.

• Other common areas of concern include limited genetic diversity in studies, which influence efficacy rates.

3️. Complex Diagnosis and Delayed Treatment

• Misdiagnosis and undiagnosed cases of rare diseases delay treatments from being applied.

• Some diseases require complex specialized tests unavailable in most countries.

4️. Regulatory and Approval Hurdles

• Getting FDA or EMA approval can take anywhere from several years to decades, requiring lots of clinical data.

• A handful of orphan drugs receive accelerated approval under the condition that they will continue to work according to their nonexistent follow-up studies.

5️. High Cost of Treatment and Limited Access

• Most orphan drugs are exceedingly expensive, running to hundreds of thousands of dollars-a-whole-lot-some-as an annual cost.

• Limited insurance and high out-of-pocket costs deny patients access.

6️. Manufacturing and Supply Chain Issues

• Many orphan drug developers require manufacturing away from a conventional system.

• There are logistical difficulties due to disparate regions in different locations.

7. Lack of Awareness and Financial Support

• Low public and medical awareness of rare diseases tends to slow the flow of research and ensuing funding.

• Only a handful of pharmaceutical companies consider investing in orphan drugs because they usually do not earn profits at least equal to nonorphans.