By Kurt R. Karst –
Citing FDA approval standards that “may deny the benefits of medical progress to seriously ill patients who face morbidity or death from their disease,” Representative Diane Watson (D-CA) introduced H.R. 4732, the Compassionate Access Act of 2010, earlier this week. The bill would amend the FDC Act to create a new conditional approval system for drugs, biological products, and devices for seriously ill patients.
Specifically, the bill would amend FDC Act § 561, titled “Expanded Access to Unapproved Therapies and Diagnostics,” and which was added in 1997 by the FDA Modernization Act, to create new subsection (d) – “Compassionate Investigational Access.” Under this new subsection:
upon submission by a sponsor of an application intended to provide widespread access to an investigational drug, biological product, or device for eligible patients (referred to in this subsection as ‘Compassionate Investigational Access’), the Secretary shall permit such investigational drug, biological product, or device, to be made available for expanded access under a treatment investigational new drug application or treatment investigational device exemption if the Secretary determines that [certain] requirements . . . are met with respect to Compassionate Investigational Access.
In particular, a sponsor must submit to FDA an application for Compassionate Investigational Access containing:
Data and information from completed Phase I clinical investigations and any other nonclinical or clinical investigations;
Preliminary evidence (that may be based on uncontrolled data and on a small number of patients or a subset of a patient population) that the product may be effective in humans against a serious or life-threatening condition or disease; and
Evidence that the product is safe at the dose and duration proposed consistent with the level of information needed to initiate a Phase II clinical trial.
A sponsor must also state that it is actively pursuing marketing approval with due diligence.
FDA would then review the application and provide Compassionate Investigational Access approval, or refer the application to a new “Accelerated Approval Advisory Committee” for further review and recommendation. In making an approval decision, FDA must “consider whether the totality of the information available to the Secretary regarding the safety and effectiveness of an investigational drug, biological product, or device, as compared to the risk of morbidity or death from a condition or disease, indicates that a patient (who may be representative of a small patient subpopulation) may obtain more benefit than risk if treated with the drug, biological product, or device.” And “[i]f the potential risk to a patient of the condition or disease outweighs the potential risk of the product, and the product may possibly provide benefit to the patient, the Secretary shall provide Compassionate Investigational Access approval of the application.”
The bill would also create FDC Act § 561A – “Accelerated Approval” – to permit the sponsor of a drug, device, or biologic application to submit an application containing:
data and information that the drug, biological product, or device has an effect on a clinical endpoint or on a surrogate endpoint or biomarker that is reasonably likely to predict clinical benefit to a patient (who may be representative of a small patient subpopulation) suffering from a serious or life-threatening condition or disease.
FDA would have 120 days to provide Accelerated Approval of the application or refer the application to the Accelerated Approval Advisory Committee. If referred to the Accelerated Approval Advisory Committee, the committee would have 90 days to make an approval recommendation, which FDA would need to act on within 30 days thereafter – either with an approval decision or an explanation as to why approval is not granted. That non-approval decision could be appealed.
In addition, the bill would create FDC Act § 561B – “Expanded Access to Investigational Drugs and Devices” – requiring FDA to “establish a new program to expand access to investigational treatments for individuals with serious or life threatening conditions and diseases.” Among other things, FDA would be required to “establish policies, regulations, and guidance designed to most directly benefit seriously ill patients,” implement training programs with respect to the expanded access programs, and “establish a program or expand upon an existing program to encourage the development of surrogate endpoints and biomarkers that are reasonably likely to predict clinical benefit for serious or life-threatening conditions for which there exist significant unmet patient needs.” In a similar vein, the bill would amend the FDC Act to add § 568 – “Policies Related to Study Evaluation Information” – requiring FDA to “give consideration to clinical judgment and risks to the patient from the disease or condition involved in the evaluation of the safety and effectiveness of drugs, biological products, and devices that treat serious or life-threatening diseases or conditions” (i.e., non-statistical measures).
Rep. Watson’s bill comes several months after FDA issued its final rule on Expanded Access to Investigational Drugs for Treatment Use in August 2009. As we previously reported, those regulatons clarified FDA’s existing expanded access regulations and added new types of expanded access for treatment use.
Rep. Watson’s bill is not the first legislative attempt to create a new approval system for patients to access drugs for serious or life-threatening diseases or conditions. In 2008, Senator Sam Brownback (R-KS) introduced S. 3046 - the “Access, Compassion, Care, and Ethics for Seriously Ill Patients Act” (the “ACCESS Act”). That bill proposed to create a three-tiered approval system – “Compassionate Investigational Access,” “Accelerated Approval,” and “Final Approval” – for products for serious or life-threatening diseases or conditions (see our previous post here). Sen. Brownback's bill was preceded by the U.S. Court of Appeals for the District of Columbia Circuit's August 7, 2007 8-2 opinion in Abigail Alliance for Better Access to Developmental Drugs v. von Eschenbach in which the court held “that there is no fundamental right ‘deeply rooted in this Nation’s history and tradition’ of access to experimental drugs for the terminally ill.” (see our previous post here)