ACCESS Act Reintroduced by Sen. Brownback; Previous Tiered Approval Nomenclature Scrapped and New Immunity Provision Added

June 4, 2008

In late May, Senator Sam Brownback (R-KS) introduced S. 3046 – the “Access, Compassion, Care, and Ethics for Seriously Ill Patients Act” (the “ACCESS Act”).  The bill, which is similar to legislation Sen. Brownback introduced in the 109th Congress with the same title (i.e., S. 1956), is intended to increase terminally ill patients’ access to investigational therapies (i.e., drugs, biological products, and medical devices) prior to FDA approval or clearance, and is one component of the ongoing debate over patient access to investigational therapies.  In December 2006, FDA announced the issuance of proposed regulations to amend and clarify the Agency’s current treatment IND regulations on access to investigational new drugs and to add new types of expanded access for treatment use, as well as a proposal to amend the Agency’s regulations on charging for investigational products.  FDA has not yet issued final regulations with respect to either proposal.  Also, on August 7, 2007, the U.S. Court of Appeals for the District of Columbia Circuit held in Abigail Alliance for Better Access to Developmental Drugs v. von Eschenbach in an 8-2 opinion “that there is no fundamental right ‘deeply rooted in this Nation’s history and tradition’ of access to experimental drugs for the terminally ill.”  The decision was appealed; however, earlier this year, the Supreme Court declined to hear the appeal, thereby letting the appellate court decision stand.

The previous version of the ACCESS Act introduced in November 2005 would have amended the FDC Act to create a three-tier approval system, similar to that advocated by the Abigail Alliance, for products for serious or life-threatening diseases or conditions.  Under that proposed bill, Tier I and Tier II approvals would be conditional approvals based on either clinical evaluation and not statistical analyses (Tier I) or information showing that the investigational product has an effect on a clinical or a surrogate endpoint (Tier II).  Tier III approval would be full approval. 

The new version of the ACCESS Act scraps the tiered approval nomenclature and replaces Tiers I , II, and III with “Compassionate Investigational Access,” “Accelerated Approval,” and “Final Approval” levels, respectively.  Sen. Brownback explained when announcing the new ACCESS Act that:

terminally ill patients who have exhausted all available treatment options would have access to treatments currently undergoing FDA approval.  On the Compassionate Investigational Access level, terminally ill patients whose medical needs are unmet by currently available options would be granted access to promising, investigational treatments that have shown evidence that the treatment may be effective against a serious illness considering the risk to the patient from the disease.  On the Accelerated Approval level, ACCESS Act improves the existing system, which allows patients with life-threatening diseases access to treatments that have shown effectiveness upon completion of Phase II clinical trials.  The ACCESS Act also establishes a five-year demonstration program that would offer Medicare coverage for drugs that receive Compassionate Investigational Access approval and makes a technical correction that will increase patient access to drugs used off-label to treat life-threatening diseases.

In contrast to the previous version of the ACCESS Act, which would have made inapplicable to Tier I and Tier II approvals the FDC Act provisions concerning user fees, Hatch-Waxman benefits, and generic drug competition, the new version of the bill does not include similar provisions.  Also, the new bill includes an immunity provision that would make a manufacturer, distributor, sponsor, or physician who manufactures, supplies, distributes or prescribes a product under a Compassionate Investigational Access application immune from suit or liability with respect to the product.

A companion bill to S. 3046 will be introduced in the House of Representatives by Representative Dianne Watson (D-CA).  Given the short amount of time left in the 110th Congress, it seems unlikely that Congress will move forward with the new ACCESS Act this session.

By Kurt Karst

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