The unfolding effort to contain the Ebola virus in the U.S. is revitalizing the issue of giving people access to experimental medication and the ethical wrangling involved in making those decisions, referred to as Compassionate Use or Right to Try. Cue a timely talk by Kenneth Moch, the former CEO of biotechnology company Chimerix, which recently received Emergency Investigational New Drug Applications clearance from the FDA for its experimental drug brincidofovir to treat Ebola.
At the Life Sciences Future conference by PA Bio this week, he gave a riveting talk on how the debate over a denied compassionate use request on behalf of 8-year old Josh Hardy erupted into a media firestorm, spurred and amplified by an intense social media campaign by the boy’s relatives and supporters.
Despite being a relatively small biotechnology company keen to get its Phase 3 antiviral drug, brincidofovir, to as many people who needed it as possible the company was likened by its critics to a big pharma company depriving a child of a much-needed drug. It was demonized for its initial decision to deny the child access to its experimental antiviral drug, brincidofovir, even as it held talks with the FDA behind the scenes. Even the lives of Moch and his colleagues were threatened. The talks ultimately led to the setting up of a 20-patient, open-label Phase III trial, and Josh became the first participant in that trial. But it certainly illustrates the ugly side of this complex issue.
Among the points Moch highlighted was the problem of who pays for Compassionate Use. Small biotechnology company face lots of expenses driven by the high cost of doing clinical trials. Insurance companies aren’t usually keen to cover the cost of an experimental drug. So who does that leave? Government? Moch said several states are reviewing proposals, but does not think they’ve been well thought out. In his talk, he mentioned that he and Dr. Arthur Caplan, who is director of medical ethics at NYU Langone Medical Center in the department of population health, are looking at forming a policy group that could work with lawmakers more closely on this issue.
Earlier this year in Congress, Rep. Griffith H. Morgan (R-VA) introduced the Compassionate Freedom of Choice Act of 2014 and it was referred to the Subcommittee on Health. According to BioCentury,Rep. Michael McCaul (R-Texas), a co-chair of the congressional childhood cancer caucus, told the publication that he is drafting compassionate access legislation and is planning to introduce it next year.
BIO emailed a list of the states that are considering or have passed Right to Try legislation. Although a couple of them differ, they would establish an individual’s right, under state law, to have access to investigational drugs, devices, biologics, after they have cleared a Phase 1 clinical trial. The bills also take on health professional licensure, which falls under state authority, and impose penalties on state officials who block access. In most cases these bills don’t obligate manufacturers to provide the investigational treatment and health insurers aren’t obligated to cover these treatments either. Given the high rate of failure of many drugs in clinical trials, some are skeptical about how effective this legislation will be.
Colorado became the first state to pass the legislation earlier this year. Similar bills in Louisiana and Missouri also became law.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
Michigan‘s House and Senate each passed Right to Try bills, SB 991 and HB 5649, but Gov. Rick Snyder has not yet signed it into law as this post was published.
Arizona voters will choose yes or no on a Right to Decide proposal in a referendum this November. Arizona HCR 2005 wants to take the FDA out of the equation by providing access to potentially life saving drugs to patients who are terminally ill. It wants to make it about a decision between a patient and their doctor. It even goes so far as to say that an official, agent or employee of the state who blocks access of an eligible patient to the drug is guilty of a misdemeanor. It also wants to make patients with a nonterminal illness eligible for these investigational treatments.
Delaware SB 270 was introduced in the state Senate in July. At press time it was in the Senate’s Health and Social Services committee, according to the General Assembly’s website.
New Jersey introduced a proposal on July 1 that was subsequently referred to the Senate Health and Social Services Committee. The bill requires the patients who benefit from the drug to use an investigational drug, biological product, or device, the patient would be required to fulfill certain criteria. They have to have a medical condition with a life expectancy of under 12 months; consult with a physician and considered all other treatment options currently approved by the FDA. They also have to have received a prescription or recommendation from a physician for the investigational drug,
