Developing treatments for rare diseases can be challenging. This is because rare diseases affect a relatively small number of people. Medications that are developed to treat or prevent rare cancers and rare diseases are called orphan drugs.
In this article, you’ll learn what orphan drugs are, how they are developed, and how they advance care for people with rare cancers and other rare diseases.
What are orphan drugs?
Orphan drugs are medications or treatments developed for rare diseases. In the United States, a disease is considered rare if it affects fewer than 200,000 people. There are many rare diseases that still do not have an FDA-approved treatment.
It is expensive to develop new medications. When there are few people with a disease, pharmaceutical companies may not invest in developing those medications. This is because it is difficult to make the money spent investing in the drug back from sales of the medication.
The term orphan drug comes from the phrase “pharmaceutical orphan.” This refers to patients who do not have treatment options.
What is the Orphan Drug Act?
In 1983, Congress passed the Orphan Drug Act (ODA). The purpose of this law is to encourage more research and development of treatments for rare diseases. It does this by giving financial assistance to pharmaceutical companies developing medications for rare diseases.
What is orphan drug designation?
A drug company can ask the U.S. Food and Drug Administration (FDA) to grant a treatment orphan drug designation if it meets certain requirements. FDA orphan drug designation can make it easier for researchers and companies to pursue treatments for smaller patient populations. They may receive tax credits for certain clinical trials, exemptions from some FDA fees, and the possibility of market exclusivity after a drug is approved.
It is important to note that orphan drug designation does not mean that a treatment has been approved by the FDA. It is a status given during the drug development process.
A treatment still has to go through research and clinical trials to determine whether it is safe and effective. The FDA must review the evidence before the treatment can be approved for patients.
What happens when an orphan drug is approved?
Once an orphan drug receives FDA approval, doctors may be able to use it to treat the specific rare disease or condition included in its approval.
Researchers may collect additional information about how well a treatment works, its side effects, and how patients respond over time. In some cases, researchers may also study the drug for other diseases or patient populations.
This continued research can provide doctors and patients with more information to consider when making treatment decisions.
Why do orphan drugs matter?
For people living with rare diseases, treatment options can be limited or may not exist at all. Smaller patient populations can also make research and clinical trials more challenging.
Orphan drug programs are designed to encourage researchers and drug developers to continue studying diseases that might otherwise receive less attention. Since the Orphan Drug Act was passed, hundreds of treatments for rare diseases have received FDA approval. Still, most rare diseases do not have an approved treatment.
For patients and families affected by rare diseases, continued research into orphan drugs can mean more opportunities for new treatment options in areas where they are greatly needed.
Orphan drugs for rare blood cancers
Because most blood cancers are rare diseases, many of the approved treatments are orphan drugs. Recent examples of orphan drugs approved to treat blood cancer include:
Rusfertide (Mimrylo) for polycythemia vera
Ropeginterferon alfa-2b-njft (Besremi) for essential thrombocythemia
T cells-vldq (Tregzi) to prevent graft-versus-host-disease (GVHD) after a stem cell transplant
D-VRd for multiple myeloma
Pivekimab sunirine-pvzy (Decnupaz) for BPDCN
Orphan drugs for other rare cancers
Other recent examples of orphan drugs approved to treat other rare cancers include:
Daraxonrasib (Rasonque) for metastatic pancreatic cancer
Sotorasib (Lumakras) for KRAS-mutated non-small cell cancer
Belzutifan (Welireg) for rare VHL-associated cancers, including kidney cancer
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