Investigational Medicine

Getting Access to Investigational Medicine

If you or someone you know suffers from a serious or immediately life threatening disease or condition and there does not appear to be a satisfactory therapy to treat the situation, the patient may be able to gain access to medications that are not available to the general public. These medicines are classified as “investigational” by the US Food and Drug Administration (FDA) and pharmaceutical companies. This page gives you some background information on three different ways you may be able to gain access to these medicines.

Pharmaceutical companies invest billions each year to discover and develop new chemical (drug) compounds into pharmaceuticals, the products we get at the pharmacy. To take a new chemical compound from the discovery stage to your pharmacy, pharmaceutical companies must first register it with the FDA as an Investigational New Drug. The Investigational New Drug is then progressed over many years through a series of regulated stages: Phase I, Phase II, and Phase III.  To learn more about Investigational New Drug development click here.

Since it takes so long to bring pharmaceuticals to market, it is important to make investigational new drugs available to critically ill patients that may have run out of options.  Over the last half century, pharmaceutical companies have worked with the US FDA to design a couple of avenues that patients may pursue to gain access to investigational new drugs; clinical trials, expanded access programs, and compassionate use appeals. 

Clinical Trial Access

Clinical trials are usually the most preferred method to gain access to investigational new drugs.  This route is preferred by the FDA and the pharmaceutical companies as it is a well-controlled pathway and limits liability. For most patients the clinical trial pathway is also preferred as the majority of cost and participation are covered by the pharmaceutical company. At the end of the trial, if a patient has benefited from the investigational new drug, they are usually allowed continued access although the drug still may not have been approved by the US FDA yet.  To search for clinical trials that might be beneficial to you or someone you go to www.ClinicalTrials.gov.

Expanded Access Programs

In the later stages of development such as the Phase III clinical trial stage, pharmaceutical companies can offer what the FDA calls Expanded Access Programs. Expanded Access Programs are for critically ill patients who are not able to enroll in a clinical trial. The FDA generally approves these programs if the drug has demonstrated some effectiveness against a specific disease in the on-going clinical trials. Investigational new drug expanded access programs are not allowed for diseases other than the ones being investigated by the pharmaceutical company.  Access to investigational new drugs has allowed tens of thousands of patients with HIV/AIDS, cancer, heart disease, and other conditions to receive promising therapies when no approved alternative is available.

To be able to offer an expanded access program for an investigational new drug:

  1. The compound cannot expose patients to unreasonable risks given the severity of the disease to be treated
  2. The pharmaceutical company must be willing to make the drug available for expanded access use
  3. The pharmaceutical company must offer the expanded access program to provide treatment for a patient’s disease or condition, rather than to collect data about the study drug

If you go the Expanded Access Program route be prepared to be confused as the major players; pharmaceutical companies, patient support/advocacy organizations and the US FDA all may use different terms. If you or a loved one is interested in treatment with an investigational drug under an expanded access program listed in ClinicalTrials.gov, make sure to review the protocol eligibility criteria and use the Contact Information listed.  To search for Expanded Access Programs that might be beneficial to you or someone you know, go to www.ClinicalTrials.gov and enter Expanded Access Programs in the search box. 

No Formal Expanded Access Program?

If there is not an expanded access program listed at ClinicalTrials.gov, the patient or their health care provider may contact a pharmaceutical company that is studying an investigational new drug directly to ask about another form of an expanded access program commonly referred to as a single patient compassionate use.

Patients and health care providers may request the use of an investigational new drug through an appeal to the pharmaceutical company on a compassionate use basis. The best way to approach a compassionate use appeal is to have the patient’s healthcare provider request permission for access to an investigational new drug from a pharmaceutical company. If you call the main switchboard of a company and ask for Medical Affairs, they should be able to help or direct you to the proper person.

If the company agrees, the patient's healthcare provider and the pharmaceutical company apply to the US FDA for approval to use the investigational new drug for the patient. The time to approval or access in a single patient scenario varies. In an emergency, the FDA has been known to process the paperwork in 24 hours.

Most pharmaceutical companies and the FDA generally support requests for compassionate use if the patient has exhausted standard treatment options and is ineligible for clinical trials. Also, due to the high risk and liability involved in the development of investigational new drugs, some pharmaceutical companies may be reluctant to establish an expanded access program for a product.  Be aware that there is no way to force or require the company or FDA to make an investigational new drug available for compassionate use treatment.

Working through a single patient compassionate use for an investigational new drug will be confusing, time-consuming and sometimes frustrating. There are usually several other hurdles that the patient’s healthcare provider will likely have to overcome as well including hospital approval and other issues. Single patient compassionate use is done on a case-by-case basis. There is no single list of drugs currently available through compassionate use as this could be seen as promoting the use of an unapproved product. Also, there is no single process adhered to by the FDA and pharmaceutical companies.