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Back in June, we reported here briefly that both chambers of the General Assembly unanimously passed “Right to Try” legislation. Now that Governor Pat McCrory has signed the bill into law, North Carolina becomes the 22nd state to enact measures aimed at overcoming the federal Food and Drug Administration’s barriers to terminally-ill patients accessing a wider-variety of potentially life-saving medications.
The Regulatory Affairs Professionals Society’s Legislation Tracker, a resource for regulatory professionals and patients to keep track of legislation as it moves through various state legislative bodies, reports that as of June 25, 2015, 21 states had enacted laws protecting the right of individuals to try experimental drugs not yet approved for public use.
House Bill 652, North Carolina’s new “Right to Try” law, grants terminally ill patients the right to have access to experimental therapies that remain unapproved by the FDA — including investigative drugs, biologic products, and medical devices. House Bill 652 stipulates that these therapies need only have passed Phase I trials before they can be made available to patients as potentially life-saving treatment.
The new law becomes effective on October 1.
There are four test phases that any new medicine or treatment must undergo before it can be made available to patients. Phase I trials are designed to only test experimental therapies for safety and toxicity, to determine the maximum tolerated dose, and to provide an estimate for the dose to use in more advanced phases of clinical trials.
Under the current system, even after an investigational drug has passed the FDA’s Phase I, it can take an another six years or more for the drug to be approved for market — even if initial trials yield promising results.
Since early 2014, more than 20 states have introduced legislation that would allow terminally ill patients to access experimental treatments more readily. These bills are modeled off a federal policy known as Compassionate Use, but contain several key changes meant to make it faster and easier for patients to obtain experimental therapies.1
Under current FDA regulations, a manufacturer wanting to conduct clinical trials on a new drug must first obtain regulatory approval to do so. The manufacturer does this by submitting an Investigational New Drug (IND) Application to the FDA, which offers an exemption from federal law allowing an investigational new drug to be manufactured and investigated. Otherwise, federal law bans unapproved drugs from entering the market.
Under the terms of the exemption, sponsors are tightly regulated and are only able to use the drug on patients enrolled in the clinical trial. This is done to ensure that the drug is used safely, that the correct patients are enrolled in the trial, and that all side effects can be monitored.
Over the last 20 years, the FDA has broadened its policy, through “early access programs” or “compassionate use exemptions,” to allow manufacturers to expand access to their investigational products while they’re still undergoing clinical trials.2
“Right to Try” laws enacted by individual states takes this policy shift even further. They remove barriers that limit medical practitioners from providing care they are otherwise trained and qualified to provide and put access to care for terminally ill patients in the hands of patients and their doctors.3
“Americans shouldn’t have to ask the government for permission to try to save their own lives,” says Darcy Olsen, President & CEO of the Goldwater Institute. “They should be able to work with their doctors directly to decide what potentially life-saving treatments they are willing to try. This is exactly what Right To Try does.”
The current “Right to Try” laws were inspired by the real-life battle with the FDA that Texan Ron Woodroof had with the FDA in 1988 in a desperate effort to save his own life and the lives of others infected with HIV. His story was made famous in the critically-acclaimed 2013 Oscar-winning film Dallas Buyers Club.
Woodroof was diagnosed with AIDS in the mid-1980s, when its cause (HIV) was not understood, effective treatments were largely unavailable, and people with the disease were highly stigmatized and not likely to live longer than just a few years. As part of the experimental AIDS treatment movement, Woodruff smuggled non-FDA-approved pharmaceuticals from Mexico into Texas to treat the disease.
Woodruff subsequently distributed the medication to his own network of people with AIDS through a black market pharmacy he called the “Dallas Buyers Club.” At the time, Woodruff’s club faced relentless opposition from the FDA and he ultimately sued the FDA over a ban on a drug he was using.
Woodruff was not alone. “Buyers Clubs” popped up all over the country during that dark time in response to what many perceived was a slow-moving medical bureaucracy and an unresponsive federal government.
In a clinical trial, participants receive specific interventions according to the research plan or protocol created by the investigators. These interventions may be medical products, such as drugs or devices; procedures; or changes to participants’ behavior, such as diet.
Clinical trials may compare a new medical approach to 1) a standard therapy that is already available, 2) a placebo that contains no active ingredients, or 3) no intervention. Some clinical trials compare therapies that are already available to one another. When a new product or approach is being studied, it is not usually known whether it will be helpful, harmful, or no different than available alternatives (including no intervention).
The investigators try to determine the safety and efficacy of the intervention by measuring certain outcomes in the participants. For example, investigators may give a drug or treatment to participants who have high blood pressure to see whether their blood pressure decreases.
Clinical trials used in drug development are sometimes described by phase. These phases are defined by the FDA.4
Clinical Trial Phases
FDA categories for describing the clinical trial of a drug based on the study’s characteristics, such as the objective and number of participants. There are five phases:
- Phase 0: Exploratory study involving very limited human exposure to the drug, with no therapeutic or diagnostic goals (for example, screening studies, microdose studies).
- Phase 1: Studies that are usually conducted with healthy volunteers and that emphasize safety. The goal is to find out what the drug’s most frequent and serious adverse events are and, often, how the drug is metabolized and excreted.
- Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition). For example, participants receiving the drug may be compared with similar participants receiving a different treatment, usually an inactive substance (called a placebo) or a different drug. Safety continues to be evaluated, and short-term adverse events are studied.
- Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.
- Phase 4: Studies occurring after FDA has approved a drug for marketing. These including postmarket requirement and commitment studies that are required of or agreed to by the sponsor. These studies gather additional information about a drug’s safety, efficacy, or optimal use.5
House Bill 652 allows manufacturers to make an investigational medications available to eligible patients if they have met the following criteria:
- The patient must have a progressive medical or surgical condition that (i) entails significant functional impairment, (ii) is not considered by a treating physician to be reversible even with the administration of available treatments approved by the FDA, and (iii) will soon result in death without life-sustaining procedures. This diagnosis of a terminal illness must be attested to by a physician.
- In consultation with the treating physician, the patient must have considered all other treatment options currently approved by the FDA.
- The patient must have received a recommendation by the treating physician for use of the investigational drug, biological product, or device.
- The patient must have given written, informed consent (see below) to use of the investigational drug, biological product, or device.
- There must be documentation from the treating physician that the patient meets all of the criteria above. This documentation must include an attestation from the treating physician that the treating physician was consulted in the creation of the required written, informed consent.
House Bill 652 requires that the informed consent must be a written document that is signed by an eligible patient; or if the patient is a minor, by a parent or legal guardian; or if the patient is incapacitated, by a designated healthcare agent pursuant to a healthcare power of attorney. The following must be included in a written informed consent:
- An explanation of the currently approved products and treatments for the eligible patient’s terminal illness.
- An attestation that the eligible patient concurs with the treating physician in believing that all currently approved treatments are unlikely to prolong the eligible patient’s life.
- Clear identification of the specific investigational drug, biological product, or device proposed for treatment of the eligible patient’s terminal illness.
- A description of the potential best and worst outcomes resulting from use of the investigational drug, biological product, or device to treat the eligible patient’s terminal illness, along with a realistic description of the most likely outcome.
- A statement that eligibility for hospice care may be withdrawn if the eligible patient begins treatment of the terminal illness with an investigational drug, biological product, or device, and that hospice care may be reinstated if such treatment ends and the eligible patient meets hospice eligibility requirements.
- A statement that the eligible patient’s health benefit plan or third party administrator and provider are not obligated to pay for any care or treatments consequent to the use of the investigational drug, biological product, or device, unless specifically required to do so by law or contract.
- A statement that the eligible patient understands that he or she is liable for all expenses consequent to the use of the investigational drug, biological product, or device and that this liability extends to the eligible patient’s estate, unless a contract between the patient and the manufacturer of the drug, biological product, or device states otherwise.
- A statement that the eligible patient or, for an eligible patient who is a minor or lacks capacity to provide informed consent, that the parent or legal guardian consents to the use of the investigational drug, biological product, or device for treatment of the terminal condition.
House Bill 652 prohibits any liability to the heirs of the eligible patient for any outstanding debt related to the use of investigational drugs, biological products, or devices.
House Bill 652 also contains provisions that would prohibit certain punitive actions from being taken against healthcare providers based upon a recommendation to an eligible patient regarding investigational drugs, biological products, or devices. No official, employee, or agent of the State would be able to take action to block or attempt to block an eligible patient’s access to investigational drugs, biological products, or devices.
Under House Bill 652, no private right of legal action can be brought against a manufacturer of an investigational drug, biological product, or device, or against any other person or entity involved in the care of an eligible patient using an investigational drug, biological product, or device, for any harm caused to the eligible patient resulting from use of the investigational drug, biological product, or device as long as the manufacturer or other person or entity has made a good faith effort to comply with the provisions of the act and has exercised reasonable care in undertaking his or her actions.
- Regulatory Affairs Professionals Society, “‘Right to Try’ Legislation Tracker“
- Regulatory Affairs Professionals Society, “Regulatory Explainer: FDA’s Expanded Access (Compassionate Use) Program“
- The Goldwater Institute, “FDA Pledges to Streamline ‘Compassionate Use’ Process in Response to ‘Right To Try’ Laws”
- ClinicalTrials.gov, “Learn About Clinical Studies“
- ClinicalTrials.gov, “Glossary Definition“