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Research and new methods

How a new medicine is born: from target to pharmacy

Modern medicines are often designed for a specific target: scientists find a protein that works wrongly in the cancer cell and create a molecule that blocks it.

Last updated: ~1 min read
Ribbon model of the ABL kinase protein with the drug imatinib bound in its pocket
Figure 1.A medicine on its target: imatinib in the pocket of the ABL protein (3D model)

01Short answer

The stages are: choosing a target; testing thousands of molecules (screening); improving the best one; checking safety and effect in cells and animal models; developing a form of the medicine (tablet or infusion); and then the clinical phases. All the data go to a regulator, the FDA in the USA or the EMA in Europe, which independently weighs benefit and risk. After approval, rare side effects continue to be tracked.

02Simple explanation

Choose a target.

Screening.

Clinical phases.

Regulator review.

03Scientific explanation

FDA: a new medicine is approved only when clinical data show that its benefits outweigh its risks.

NCI: new treatments are tested step by step, from the laboratory to clinical trials; only those proven safe and beneficial become standard care.

04What happens in the body?

Targeted design

The molecule is shaped to fit a specific protein.

Thousands of candidates
Figure 2.Thousands of candidates.

Benefit and risk

The regulator weighs both sides.

Scales
Figure 3.Scales — How a new medicine is born: from target to pharmacy

How to read the news

Research news brings hope, but not every advance suits everyone. Look at who it was tested in, at what phase and in how many people.

Talk to your doctor

Show your doctor what you have read; they will explain how it relates to your diagnosis and treatment. Do not change your treatment on your own.

05From research news to a conversation: four steps

Read, check, ask, decide together.

Read — From a trusted source.1ReadFrom a trusted source.
Check — Phase, participants, result.2CheckPhase, participants, result.
Ask — Show your doctor.3AskShow your doctor.
Decide — Together, without rushing.4DecideTogether, without rushing.
SourceA trusted site.
DoctorA personal explanation.
SafetyNot changing treatment yourself.

06Key points at a glance

TopicIn brief
Targeted designThe molecule is shaped to fit a specific protein.
Benefit and riskThe regulator weighs both sides.
How to read the newsResearch news brings hope, but not every advance suits everyone. Look at who it was tested in, at what phase and in how many people.
Talk to your doctorShow your doctor what you have read; they will explain how it relates to your diagnosis and treatment. Do not change your treatment on your own.

07Key terms

Screening
Rapid testing of many substances.
Regulator
The government body that authorises medicines.
Clinical trial
Testing a new treatment with volunteers.
Evidence
A result confirmed by reliable research.

08Key points

  • Choose.
  • Test.
  • Approve.
  • Check the source.
  • Talk to your doctor.

09Frequently asked questions

Does approval mean it suits everyone?

No; only for specific uses and patients.

Why is it not available in my country yet?

Each country approves medicines separately.

Is this advance right for me?

Only your treating doctor can say, based on your diagnosis, stage and condition.

10Questions for your doctor

Take these questions to your appointment. The ones you save are collected on the “My questions” page and stay on this device only.

  1. Does approval mean it suits everyone?

  2. Why is it not available in my country yet?

  3. Is this advance right for me?

My questions

12Sources

  1. U.S. Food and Drug Administration. The Drug Development Process
  2. U.S. Food and Drug Administration. FDA Drug Review Process (continued)
  3. European Medicines Agency. Clinical trials in human medicines
  4. National Cancer Institute. What Are Clinical Trials?
  5. National Cancer Institute. Phases of Clinical Trials
  6. National Cancer Institute. Using Trusted Resources
  7. Cancer.Net (ASCO). Phases of Clinical Trials
  8. Cancer.Net (ASCO). Placebos in Cancer Clinical Trials
  9. Cancer.Net (ASCO). Questions to Ask About Clinical Trials
  10. American Cancer Society. Clinical Trials
Medical disclaimerThis information is for educational purposes only and does not replace medical advice.
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