The right to try movement and recent passage of the federal right to try law have renewed public interest in how patients might gain access to investigational therapies. FDA’s expanded access program (also known as “compassionate use”) has for decades served as the main method by which patients can request access to investigational products outside of participating in a clinical trial. Right to try legislation offers an alternative for those who feel the FDA pathway is unnecessarily burdensome to patients, their treating physicians and families.

So what’s the difference between these two programs? Let’s take a look at the basics of what each program does and does not do for patients seeking to access investigational therapies.

Obtaining the Investigational Product

It is entirely up to the company developing the investigational product (also known as the sponsor or manufacturer) whether they will participate in an expanded access or right to try request. This might be the most important thing to understand regarding both pathways: neither requires the sponsor/manufacturer to provide the product to the requesting patient.

Some sponsors/manufacturers have published policies online outlining organizational policy on expanded access and/or right to try. The federal right to try law also removes any liability on the part of the sponsor or manufacturer for providing access or for choosing to not provide access via this pathway. There are many reasons a sponsor or manufacturer might choose not to provide access, some of which I’ve outlined below in the “Clinical Trials Impact” section.

Eligible Patients

Both pathways have very specific patient eligibility criteria. The patient must have a serious or life-threatening disease or condition. He or she also must have already exhausted available approved treatment options, and the patient must be unable to enroll in a clinical trial to access the investigational product. This all must be confirmed by the patient’s physician.

Expanded access goes on to also require that the potential benefit to the patient justifies the potential risks of treatment. Additionally, providing the investigational product must not interfere with clinical trials that could support the product’s development or marketing approval.

Physician Involvement

A physician must assist the patient in accessing the investigational product via either pathway. Patients cannot directly request access from the company developing the investigational product.

Expanded Access

If the physician determines the patient meets the criteria for expanded access, the physician must contact sponsor or manufacturer to find out if the company will provide the product via expanded access. To access investigational drugs, and if the sponsor/manufacturer agrees, the physician will submit either Form 1571 “IND Application” or the streamlined Form 3926 “Individual Patient Expanded Access IND Application” and provide FDA with information on the intended use and monitoring plans, the patient(s) and his/her disease or condition, and the investigational product itself. Access to investigational medical devices requires submission of an IDE (Investigational Device Exemption) which can take the form of an original IDE or an amendment to an existing IDE. Otherwise, the requirements are very similar to accessing investigational drugs.

The physician must report any adverse drug events to the sponsor, ensure informed consent requirements are met, ensure IRB review is obtained appropriately, and maintain and retain accurate records.

Right to try

The federal law states that the patient’s physician must be in good standing with the licensing organization or board. The physician cannot be compensated directly by the sponsor or manufacturer for certifying a patient is eligible for right to try. The law also removes any physician liability for helping the patient gain access, except in situations of serious misconduct.

The physician must receive “written informed consent regarding the eligible investigational drug,” but no guidance is given on the content of such informed consent or what other regulations might impact the consent process.

Regulatory Involvement and Oversight

With expanded access, a physician must submit a request to FDA as described above, and IRB oversight must be obtained. Sponsors must also submit safety reports and annual reports to FDA, and the agency outlines specific responsibilities that sponsors and investigators must follow.

The federal right to try law requires the sponsor or manufacturer submit an annual summary to the Department of Health and Human Services (DHHS) Secretary regarding any drug used under the law. Under the federal right to try law, there is no requirement to obtain prior approval from FDA or to obtain review from an IRB.

Impact on Clinical Trials

The federal right to try law explicitly states that clinical outcomes associated with right to try access cannot be used to adversely affect the review or approval of the investigational product except in special circumstances. FDA has made similar statements regarding clinical outcomes from expanded access: “The potential for expanded access to negatively impact a product’s development program is remote. FDA considers adverse event data in the context in which the adverse events occurred.”

Even so, some sponsors may still have concerns about providing access outside of the clinical trial setting. Possible issues include:

  • Supply of the investigational product – Because the product has not yet been approved, the sponsor might have only a limited supply and cannot allocate additional product beyond what has already been distributed for research purposes.
  • Clinical trial recruitment and data collection – Sponsors may feel that providing access outside the clinical trial realm will make it more difficult to recruit study participants. Not having enough participants can also weaken the study data, which may also weaken the sponsor’s eventual application for approval.

 Choosing a Pathway

For patients or physicians considering these options, you may want to start by contacting the sponsor/manufacturer to see if they will provide the investigational product. The company may prefer one pathway over the other; if so, this simplifies your decision. There is nothing in the law that requires patients and physicians choose one pathway over the other. However, it may be a good idea to choose one pathway and stick with it. Attempting both approaches simultaneously could create confusion as to which requirements and responsibilities apply and slow down the process overall.

For sponsors and manufacturers, if you don’t already have policies in place regarding expanded access and right to try, now may be a good time to establish those. Consult with your legal counsel and other trusted advisors to understand how each pathway might affect your organization.

 

 

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