‘Screw the FDA (US Food and Drug Administration), I’m going to be DOA’ were the immortal words of a dying Ron Woodroof, played by Matthew McConaughey, in the 2014 movie The Dallas Buyers Club. The movie told the true life story of an AIDS patient, desperate to save his own life and those of his friends, who began smuggling illegal unapproved drugs from Mexico into America. Thirty years later, some of the medication Woodroof was smuggling is widely and legally available to treat HIV and AIDS and has, in the main, turned this once deadly disease into a largely chronic and manageable illness.
The efforts of patients such as Ron Woodroof led to new legislation, known as Right to Try (RTT), which gives terminally ill people the legal right to try unapproved medication. RTT legislation is a workaround of America’s lengthy, often decades-long, process of government drug approvals, and has now spread to 37 states. Hundreds of lives have been saved in the process, and a range of drugs have become available on the back of terminally ill patients utilising RTT legislation.
However, no comparable legislation exists in Australia. The Leukaemia Foundation’s submission into a 2015 Senate Inquiry into the availability of new, innovative and specialist cancer drugs in Australia states that:
Australian blood cancer patients are among the last in the G20 to access affordable drugs. Hundreds of patients are trying to access new drugs in a bid for life. For most, these drugs are either too expensive or not yet approved in Australia.
The Leukaemia Foundation found that 76 per cent of patients taking new blood cancer drugs experienced life extension, and half are in remission or disease free. But only 1 in 5 patients could access new drugs. This is a tragedy. Like in America, Australian states should return the fundamental human right of saving one’s own life to patients.
RIGHT TO TRY IN AMERCIA
In both America and in Australia, a new drug must go through several phases of clinical trials before it can be approved and sold. According to the US Council of Advisors on Science and Technology, the time from drug discovery to drug approval is roughly 14 years. In Australia, approvals take 2 years longer than that—that’s 16 years where terminally ill patients do not have access to potentially life-saving medication.
Whilst there were ways for the terminally ill in America to circumvent these approvals process—in the form of expanded access processes—the Phoenix based Goldwater Institute devised a legal workaround for the 97 per cent of terminally ill patients who can’t access clinical trials for experimental drugs.
Their solution was simple: bypass the FDA approvals process through Right to Try (RTT) legislation. Patients could now opt-out of the bureaucratic system and negotiate directly with their doctors and pharmaceutical companies. Under RTT, patients sign waivers saying there will be no liability on either the drug company or the prescribing doctor should the experimental medication harm them. The legislation therefore de-risks the provision of unapproved drugs for pharmaceutical companies, enabling and incentivising their legal supply.
The application of RTT legislation has been successful across America. For instance, last year a Texan doctor used the RTT laws to successfully treat 78 terminally ill neuroendocrine cancer patients—the deadly cancer that claimed the life of Apple founder and CEO Steve Jobs in 2011. These people would simply not be alive without RTT.
To be clear, RTT legislation isn’t a free-for-all. The option is only available to the terminally ill—those who have been given less than a year to live—and its application is limited to drugs which have successfully passed human testing, establishing the correct dosage and whether any side effects outweigh potential benefits.
Kurt Altman, the National Policy Adviser for the Goldwater Institute, has pointed out that RTT laws return control of medical decisions ‘back to a local level’. That is, they return choice back to where it belongs, in the hands of doctors and their patients. In this way it taps into a resurgent libertarian movement advocating for the fundamental right of the individual to make informed decisions about their own treatment.
The movement has gained momentum after being endorsed by the Trump administration, including the explicit support of Vice President Mike Pence. Speaking at a meeting with pharmaceutical industry executives in February 2017, Trump expressed his concern that the FDA was standing in the way of patients accessing unapproved drugs as treatments of last resort:
One thing that’s always disturbed me, they come up with a new drug for a patient who is terminal, and the FDA says ‘we can’t have this drug used on the patient.’ But they say ‘But the patient within four weeks will be dead.’ They [the FDA] say ‘Well, we still can’t approve the drug and we don’t want to hurt the patient.’ But the patient is not going to live more than four weeks.
However, the laws have not escaped controversy. Some critics say the FDA’s expanded access program would have given people access to drugs anyway—a curious claim given the extensive bureaucratic process involved, when timeliness is critical to the patient. Other opponents suggest that RTT is not a right to receive, and as such could provide false hope. Indeed, the laws do not compel a doctor to prescribe experimental drugs, insurance companies to pay for them, or a pharmaceutical company to provide them. There’s also the argument that there is not enough safety data for patients to possess enough knowledge to have truly informed consent. And then there is the old paternalistic argument that opening the floodgates to circumnavigate the federal bureaucracy will lead to quacks with medical licenses and ‘big pharma’ exploiting desperate and vulnerable patients and their families.
These are all valid criticisms. What they ignore, however, is that without RTT individuals are denied the fundamental human right to save their own life. Terminally ill people can’t wait years for all of the safety trials to be conducted, for legislation to prevent exploitation, or to see if they can access the drugs through expanded access programs. Given the spread of RTT legislation across America, it is timely to ask if it could work here in Australia.
AUSTRALIA’S RIGHT TO TRY
Australia’s Therapeutic Goods Administration (TGA) has a scheme whereby terminally ill patients can access unapproved drugs if their doctors prescribe them. This process occurs on a case-by-case basis—requiring applications from doctors and institutions, and noting that the approval of a Special Access Scheme (SAS) request by the TGA does not overrule state and territory laws which govern hospitals, doctors, and pharmacists. In making approvals, the TGA states that it has a responsibility to balance the rights of the individual—to gain timely access to new therapeutic agents—against the broader community interest of having therapeutic products available in Australia that are properly evaluated for quality, safety, and efficacy.
However, particularly for those with rare diseases, the SAS does not go far enough. Applications on a case-by-case basis are bureaucratic. The system doesn’t allow pharmaceutical companies to waive their liabilities—something which is necessary for patients seeking access to drugs which haven’t been tested for their condition. There is also a significant delay in approving drugs developed to treat rare diseases because of the small patient population size and the associated difficulty and cost impediment to obtaining the required data. Further, to encourage drug companies to open compassionate schemes they need to be reassured that any off-trial adverse effects won’t prevent their future approval.
The TGA’s claim that in restricting access to drugs it is balancing the needs of the individual with the community is absurd. In having the courage to take on the risk of using unapproved drugs, terminally ill patients discover new uses for them which can save the lives of other patients in the future. It is difficult to imagine a more valuable contribution to the community than that.
Think how many thousands of drugs are in the process of clinical trials that could be saving people’s lives and helping to find new, undiscovered, applications. The use of experimental drugs is fundamentally a discovery process—of determining what drugs are useful for what problem—and complex red tape slows down this process.
There are more than 1000 cancer drugs currently in development in America. These new drugs provide the opportunity for many cancers to be curable or at the very least manageable in the near future.
Limiting access to drugs through complex approvals processes does not only do individuals a disservice, but the community as a whole. In the absence of an entire overhaul of the approvals process, RTT legislation liberates the system through a workaround. Australia must have a debate on why we aren’t able to access these life-saving drugs, and we should follow the lead of America and adopt Right to Try legislation as a means of empowering patients and their doctors.