Monday, December 8, 2014
Guest Blogger Associate Dean and Professor of Law Joan H. Krause: The Right to Try Meets the Reality of Drug Approval
Whether it be a social media campaign to convince a company to provide an experimental anti-viral drug to a young cancer patient suffering from a life-threatening infection or the debate over appropriate treatment for high-profile Ebola cases, access to potentially life-saving but unapproved medications remains a controversial issue. Two recent articles, published on the same day, illustrate the difficulty of trying to balance desperate patients’ willingness to try unproven therapies with the very real concerns faced by manufacturers undergoing the drug approval process. The first was a Kaiser Health News article describing the passage of “Right to Try” laws in five states. The second was a brief note in the Los Angeles Business Journal that shares of CytRx Corporation, a biopharmaceutical R&D company, had fallen 9% after the company announced that the FDA had placed a partial clinical hold on its clinical trials after a patient’s death.
Right to Try laws are designed to give patients who have exhausted all other treatment options the right to access investigational medications, devices, and biological products that have met Phase I safety milestones. Right to Try legislation has been enacted in Colorado, Louisiana, Michigan and Missouri, and voters recently approved it by initiative in Arizona. The laws are based on model legislation drafted by the Goldwater Institute, which issued a detailed report on the issue in February 2014. While prohibiting states from blocking patient access to such medications, however, the model legislation does not require manufacturers to provide the products, nor does it require insurance companies to cover the costs.
Proponents of Right to Try legislation argue that the laws are necessary because the FDA’s “expanded access” (or “compassionate use”) programs severely limit patient access to experimental treatments. The FDA process can be a lengthy one, demanding voluminous and time-consuming paperwork from the requesting physician and inviting possible delays by requiring IRB approval. Moreover, proponents argue that the FDA may deny requests on broad grounds, effectively allowing the agency to disregard the “deeply personal” views of patients and their treating physicians regarding the risks they are willing to accept in their unique circumstances. Advocates have not yet been successful in pressing these issues at the federal level. In 2007, the D.C. Circuit held in Abigail Alliance for Better Access to Developmental Drugs v. Von Eschenbach that there was no fundamental right of access to experimental drugs for the terminally ill. While numerous bills have been introduced in Congress since then, none have been passed – leading proponents to turn instead to the states.
Commentators such as Elizabeth Weeks Leonard have elucidated the potential disadvantages of allowing broad access to drugs still in the experimental stage, including the fear that manufacturers would no longer be able to recruit enough patients to participate in the controlled double-blind studies needed to gain final approval. The CytRx situation illustrates a similar conundrum: the concern that poor results in very sick patients who receive the drug outside of clinical trials will delay the approval process. That is precisely what happened to CytRx: the patient who died was not enrolled in one of the company’s clinical studies of the chemotherapeutic agent aldoxorubicin, but instead received the drug under the compassionate use program. While patients currently enrolled in the company’s trials may continue, no new patients may be enrolled until the hold is lifted, and the company has been asked to amend the study protocols to include additional inclusion/exclusion criteria, patient screening, and evaluation of serum electrolytes prior to drug administration. With the company expecting to announce the results of several of Phase 2 trials in 2015, as well as to complete enrollment for a major Phase 3 trial, the hold (and its ensuing publicity) comes at a difficult time.
If Right to Try laws gain more traction (and survive any constitutional challenges), more companies could find themselves in similar situations. These laws, while well-intentioned, raise the potential for significant delays in the drug approval process – and the potential for far-ranging impact on the larger population that awaits a drug’s approval.