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Explainer: How Are Medicines Developed?

The National Organization for Rare Disorders recently launched a course that explains how a potential treatment becomes an approved medicine. We share three takeaways for patients.


Patients wait eagerly for new treatments, but it takes years to fully investigate, conceive of and test a new medicine. The National Organization for Rare Disorders (NORD), which advocates for rare disease patients, recently launched a new course that helps explain the process.

The free course – Rare Disease Drug Development Series: What Patients and Advocates Need to Know – answers questions patients want to know about the timeline and the many stakeholders involved in taking a medicine from idea to approved for use by patients.

NORD says it created the course so patients “can play a more proactive role and share their unique insights at key points along the way.” Understanding more about how medicines are developed can help patients become more impactful advocates. Below are three takeaways from NORD’s course.

1. Many stakeholders are involved.

Understanding the various players and the roles they play is a good place to start. Here’s are four of them:

Sponsors, also known as biotech and pharmaceutical companies

Regulatory agencies, such as the U.S. Food and Drug Administration (FDA) Center for Drug Evaluation and Research (CDER)

Researchers, who do carefully controlled research into a treatment’s safety and effectiveness

Patient organizations, such as the National Organization for Rare Disorders (NORD), the Immune Deficiency Foundation and others, provide essential information about the patient experience.

2. It’s a multi-step process that takes an average of 12 years.

In discovery and development, researchers start by investigating the basic nature of a disease and then do additional studies to zero in on a potential treatment. That’s followed by pre-clinical research. If that phase is successful, clinical research and regulatory review may happen next. If a new medicine is approved for use by patients, post-marketing safety monitoring follows.

3. Patients can and should have a voice.

Patients have an increasing number of opportunities to take part in the process. They might help raise funds for research, share their patient journey, provide medical data in patient registries and participate in FDA Listening Sessions. 

Learn how to enroll in the NORD course.