Expanded access

Expanded access refers to the use of an investigational drug outside of a clinical trial by patients with serious or life-threatening conditions who do not meet the enrollment criteria for the clinical trial in progress. This type of access may be available, in accordance with United States Food and Drug Administration (FDA) regulations, when it is clear that patients may benefit from the treatment, the therapy can be given safely outside the clinical trial setting, no other alternative therapy is available, and the drug developer agrees to provide access to the drug. The FDA refers to such a program as an expanded access program (EAP).[1] EAPs can be leveraged in a wide range of therapeutic areas including HIV/AIDS and other infectious diseases, cancer, rare diseases, and cardiovascular diseases, to name a few.

There are several types of EAPs allowed in the United States. Treatment protocols and treatment INDs provide large numbers of patients access to investigational drugs. A single-patient IND is a request from a physician to the FDA that an individual patient be allowed access to an investigational drug on an emergency or compassionate use basis.[2] When the FDA receives a significant number of requests (~10 to 100) for individual patient expanded access to an investigational drug for the same use, they may ask the trial sponsor to consolidate these requests, creating an intermediate-size group.[3] “Compassionate use” is a more colloquial term that is not generally used by the FDA.

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FDA regulations

Since 1987, the FDA has had rules in place that have enabled patients, under specific circumstances, to access drugs or biologics that are still in development for treatment purposes. These expanded access program rules were amended in 2009 by the FDA to ensure “broad and equitable access to investigational drugs for treatment.”[4]

The regulations include the following:[4]

The regulations also include general criteria for granting expanded access:[3]

Despite the updated regulations, debate remains over key elements of expanded access:

A number of challenges can exist when patients seek access to investigational drugs:

Outside the United States

Outside the U.S., programs that enable access to drugs in the pre-approval and pre-launch phase are referred to by a variety of names including “named patient programs,” “named patient supply” and “temporary authorization for use.”[10] In the EU, named patient programs also allow patients to access drugs in the time period between centralized European Medicines Agency (EMEA) approval and launch in their home countries which can range from one year to eighteen months.[11]

References

  1. ^ US National Cancer Institute - Access to Investigational Drugs accessed April 22, 2007
  2. ^ FDA Final Rules for Expanded Access to Investigational Drugs for Treatment Use and Charging for Investigational Drugs
  3. ^ a b c d e f Final FDA Rules on Expanded Access to Investigational Drugs for Treatment Use
  4. ^ a b FDA website
  5. ^ Expanded Access to Investigational Drugs Genetic Engineering & Biotechnology News, January 15, 2010.
  6. ^ a b c d e Access to Investigational Drugs Remains Challenge Despite New FDA Rules ‘’The Pink Sheet’’
  7. ^ Managing Access to Drugs Prior to Approval and Launch ‘’Life Science Leader’’
  8. ^ a b c FDA webinar accessed May 5, 2010
  9. ^ FDA Law Blog accessed May 5, 2010
  10. ^ Helene S (2010). "EU Compassionate Use Programmes (CUPs): Regulatory Framework and Points to Consider before CUP Implementation". Pharm Med 24 (4): 223–229. http://adisonline.com/pharmaceuticalmedicine/Fulltext/2010/24040/EU_Compassionate_Use_Programmes__CUPs___Regulatory.4.aspx. 
  11. ^ [Ericson, M., Harrison, K., Laure, N. & De Crémiers, F., Compassionate Use Requirements in the Enlarged European Union. RAJ Pharma, May 2005: 83.

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