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Effective Global Access Programs within an International Regulatory Landscape

Posted by Lisa Tandy on Jun 23, 2015 3:19:30 PM

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Like most ethical questions in the medical field, the use of Expanded Access Programs eludes simple answers. The decision to grant expanded access requests can be complicated even though the patients making these requests have exhausted many if not all of their other options for survival. Because these drugs are still in their clinical stages, companies might hesitate to grant access for a number of reasons. In clinical trials, for example, companies have complete control over the administration of drugs. The drugs are administered in a highly controlled and regulated manner in hopes of achieving the best results and retrieving the most accurate data.

Expanded access patients can be deemed ineligible for the trial for a number of reasons ranging from geographical location and general level of health to having an illness that is not specifically included in the clinical trial. This means that any adverse effects of the drug that occur with these patients can still work against the drug's approval even though the drug might not have been intended for this particular use and the patient may have been in too poor of health to be considered for the regular trial. The Expanded Access Program poses an ethical dilemma, as drug companies are forced to weigh the benefits of giving a dying patient a chance at treatment with their ethical imperative to get effective drugs onto the market as quickly as possible so that the greatest number of patients can be treated. 

The US has three types of EAPs, each of which is considered on a case-to-case basis.

  • Single Patient (Emergency Use): Provides treatment for individual patients where the physician has reason to believe that the drug might have a positive effect, even though the patient falls outside of the trials intended participants. Their physician must determine that the risk of treatment is not greater than the risk of the disease or condition.
  • Intermediate Size: Provides treatment for groups of patients (less than 100) that do not otherwise qualify to participate in the drug trials.
  • Treatment Protocol: Provides access to experimental drugs that have shown promise during their clinical testing but are still in the final stages of their clinical work and FDA approval process.

The EU provides two types of managed access.

  • Cohort Access: Provides structured and supervised treatment to a group of patients for therapeutic use and the collection of information.
  • Nominative Access: Provides treatment for an individual patient on a case-by-case basis, with various denominations depending on the individual country of access.

The expanded access dilemma becomes even more complicated when considered from a global standpoint. In low and middle-income countries, treatment for non-communicable and chronic diseases is often overshadowed by the treatment of more endemic, communicable diseases. Many fear that by extending treatment opportunities to individuals who might not have access to skilled and trained professionals for administration could end up being more dangerous than beneficial. "Right to Try" laws have been approved in twenty states, which doesn't include states with pending legislation, and there is now legislation pending on a federal level. These laws were developed due to the complicated nature of EAP approval, which in many cases required up to 100 hours for completion. While debate continues about the dangers of these laws, the law has motivated the FDA to design a new form which would take only 45 minutes to complete. This form is now available for public comment and should be released later this year.

While the problems are obvious, so are the benefits. EAPs can provide other benefits for drug companies, such as pre-approval attention and recognition of their drug, additional data about drug interactions and side effects, improved safety information, varied recommendations for use, and early engagement with patients and physicians. Especially with rare diseases, these EAPs can be extremely helpful, as there is often less money for research when it comes to these diseases. As EAPs become more popularized, so will the opportunity to regulate and better facilitate patients while still ensuring their safety and the effectiveness of the drug trials.

Want to learn more about where the industry is headed? Join Expanded Access Programs Summit which takes place July 22-23, 2015 featuring an extended session with the FDA. 

 

SOURCES:

http://www.healthaffairs.org/healthpolicybriefs/brief.php?brief_id=135

http://mapigroup.com/services/real-world-evidence/rare-diseases-expanded-access-programs/

http://www.fda.gov/ForPatients/Other/default.htm

http://www.nlm.nih.gov/services/ctexpaccess.html

http://www.raps.org/regulatoryDetail.aspx?id=18343

 

Topics: Patient Access