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USA: Access to medicines

In the USA, leukaemia medicines reach the market after FDA approval, but for patients the key question is how insurance pays for them.

Last updated: ~1 min read
On a dark navy background, a seal of approval.
Figure 1.FDA seal — USA: Access to medicines

01Short answer

The FDA approves medicines, sometimes faster through “accelerated approval”. Insurance plans have a drug list called a “formulary”; expensive oral leukaemia medicines are often on a high tier and are shipped home through a “specialty pharmacy”. Many medicines need prior authorization. If you are refused, you have the right to appeal. Access to an unapproved medicine is sometimes possible through a trial or the FDA “expanded access” programme.

02Simple explanation

FDA: approves.

Formulary: the insurer’s list.

Prior authorization: advance approval.

03Scientific explanation

Expanded access: for serious illness with no other options, a doctor can apply to the FDA.

Help: manufacturer and foundation payment assistance programmes exist; terms vary.

04What happens in the body?

Approval

The FDA can review cancer medicines on a priority basis.

Specialty pharmacy

Expensive medicines arrive by mail, sometimes with a cold chain.

On a dark navy background, a cold-chain box.
Figure 2.Box — USA: Access to medicines

Tiers

The higher the tier, the larger your share may be.

On a dark navy background, steps of light.
Figure 3.Tiers — USA: Access to medicines

05Getting your medicine

Four steps.

List — Check the formulary.1ListCheck the formulary.
Approval — Your doctor requests it.2ApprovalYour doctor requests it.
Help — Ask about payment programmes.3HelpAsk about payment programmes.
Refill — Order on time.4RefillOrder on time.
Medicine in handNo gaps.
Cost is clearYour share is known.

06Key points at a glance

TopicIn brief
ApprovalThe FDA can review cancer medicines on a priority basis.
Specialty pharmacyExpensive medicines arrive by mail, sometimes with a cold chain.
TiersThe higher the tier, the larger your share may be.

07Key terms

Formulary
The insurer’s drug list.
Prior authorization
Advance approval.
Specialty pharmacy
A pharmacy for complex medicines.
Expanded access
A programme for unapproved medicines.

08Key points

  • Learn the medicine name and tier.
  • Ask about the approval status.
  • See the financial counsellor.
  • Order refills a few days early.
  • Appeal if refused.

09Frequently asked questions

What if insurance refuses?

Ask for the reason in writing; internal and independent external appeals are possible.

Are there generics?

Some leukaemia medicines have generics; your doctor and pharmacy will tell you.

Can I bring medicine from abroad?

This is risky legally and for safety; talk to your team.

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. What if insurance refuses?

  2. Are there generics?

  3. Can I bring medicine from abroad?

My questions

12Sources

  1. U.S. Food and Drug Administration (FDA). Drugs
  2. U.S. Food and Drug Administration (FDA). Oncology (cancer) / hematologic malignancies approval notifications
  3. U.S. Food and Drug Administration (FDA). Expanded access
  4. National Cancer Institute (NCI). Drugs Approved for Leukemia
  5. HealthCare.gov. Appealing a health plan decision
  6. GOV.UK. Cancer Drugs Fund
  7. NHS Blood and Transplant. Give blood
  8. NHS. Blood transfusion
  9. Anthony Nolan. Official website
  10. World Marrow Donor Association (WMDA). Official website
Medical disclaimerThis information is for educational purposes only and does not replace medical advice.
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