Improving Access to Clinical Trials Act Becomes Law

By Kurt R. Karst –      

Earlier this month, President Obama signed into law S. 1674, the Improving Access to Clinical Trials Act (Pub. L. No. 111-255).  The bill, introduced in the Senate by Senator Ron Wyden (D-OR) in September 2009, amends the Social Security Act (42 U.S.C. 1382b & 42 U.S.C. 1396a) to provide for an exclusion under the Supplemental Security Income (“SSI”) program and Medicaid for certain compensation of individuals who participate in clinical trials for rare diseases or conditions.

According to the bill’s findings, “[w]ith a small number of potential trial participants and the possible loss of [SSI] and Medicaid benefits for many who wish to participate, clinical trial research for rare diseases and conditions becomes exceptionally difficult and may hinder research on new treatments and potential cures for these rare diseases and conditions.”  Senator Wyden’s bill, however:

would give people who are eligible for [SSI] and Medicaid the same access to clinical trials as those who are more financially fortunate. . . .  Currently, SSI and Medicaid eligible individuals who want to participate in a clinical trial have to worry about whether or not they will see a loss or a reduction in their benefits for their participation in a clinical trial if the trial offers any sort of research compensation to participants as part of its approved Internal Review Board study design.  This legislation would make it so benefits that these individuals receive from clinical trials are not counted against those who are seeking SSI or Medicaid benefits or those who are already eligible for these benefits.

Specifically, for SSI income exclusion purposes, the new law excludes “the first $2,000 received during a calendar year by such individual (or such spouse) as compensation for participation in a clinical trial involving research and testing of treatments for a rare disease or condition,” provided the clinical trial has been reviewed and approved by an appropriate institutional review board.  For Medicaid exclusion purposes, the new law provides that “[t]he first $2,000 received by an individual (who has attained 19 years of age) as compensation for participation in a clinical trial meeting the requirements of section 1612(b)(26) shall be disregarded for purposes of determining the income eligibility of such individual for medical assistance under the State plan or any waiver of such plan.”

Because, according to Sen. Wyden, there are “some legitimate concerns that research compensation may create the wrong kind of incentives for low-income people,” the new law requires the Comptroller General of the United States to conduct a study to evaluate the effects of S. 1674 on enrollment of SSI beneficiaries in clinical trials for rare diseases or conditions.  The study, which has to be conducted not later than 36 months after the efffective date of the new law, must include an analysis of:

(1) The percentage of enrollees in clinical trials for rare diseases or conditions who were SSI beneficiaries during the 3-year period prior to the effective date of this Act as compared to such percentage during the 3-year period after the effective date of this Act.

(2) The range and average amount of compensation provided to SSI beneficiaries who participated in clinical trials for rare diseases or conditions.

(3) The overall ability of SSI beneficiaries to participate in clinical trials.

(4) Any additional related matters that the Comptroller General determines appropriate.

Not later than 12 months after completion of the study, the Comptroller General must submit a report to Congress with the study results and “with recommendations for such legislation and administrative action as the Comptroller General determines appropriate.”

The new law takes effect within 180 days of enactment, or sooner if the Commissioner of Social Security promulgates final regulations to implement the new law.  S. 1674 is scheduled to sunset in five years, so that Congress can examine the Comptroller General’s report and determine whether the the program is working and should be reauthorized and/or changed. 

The enactment of the Improving Access to Clinical Trials Act is the latest development in a recent flurry of rare disease/orphan drug legislative activity.  As we previously reported (here and here), Congress is considering legislation to streamline the development and regulation of products for rare and neglected diseases and to increase incentives for the development and approval of such products.  We also note that earlier this month the Institute of Medicine issued a 350-page report to help refocus attention on accelerating rare diseases research and product development (see our previous post here), and that FDA and the Drug Information Association (in collaboration with the National Organization for Rare Disorders and Genetic Alliance) recently announced an Orphan Drug Designation Workshop scheduled for November 4-5, 2010, in Lansdowne, Virginia.