What are the challenges of Compassionate Use of unapproved drugs?
Obtaining informed consent from patients seeking access to unapproved drugs on a compassionate use basis may also be difficult. Other important problems include the qualifications of the physician who is to perform treatment, and patient selection criteria.
What should be included in the ethical review of compassionate use?
The independent ethical review of compassionate use should consider protocols (in cases of programs involving groups of patients), available drug safety and efficacy evidence, informed consent forms, medical justifications for using an unapproved drug, and the administering physician’s qualifications.
What is compassionate drug use?
According to guidelines from the National Cancer Institute, most compassionate drug use is for patients who meet all of these conditions: Have used standard treatments and they have not worked. Are not eligible for any current clinical trial that’s using the drug. Have no other treatment options.
Is compassionate use of investigational drugs ethical?
They reasoned that compassionate use might be ethical, provided that it contributes to our knowledge of investigational drugs. However, they acknowledged significant shortcomings of compassionate use as a source of data on the efficacy and safety of investigational drugs.
Is compassionate use ethical?
Compassionate use programs could be justified by claiming that they allow for a fair distribution of experimental drugs to patients with no other means to access the drug. Such compassionate use programs could also be justified by appealing to the ethical principle of beneficence.
What is compassionate use of a drug?
Sometimes called “compassionate use”, expanded access is a potential pathway for a patient with an immediately life-threatening condition or serious disease or condition to gain access to an investigational medical product (drug, biologic, or medical device) for treatment outside of clinical trials when no comparable ...
Should an oncology patient be allowed to try an experimental treatment or should the patient not be allowed to try it?
First, the patient must be informed that the treatment is experimental, the outcome unpredictable and possibly harmful. Second, the patient's participation must be voluntary or, if the patient is unable to voluntarily consent, participation must be approved by the patient's surrogate decision maker.
Is compassionate use research?
IRB agreement that a particular case meets FDA criteria for emergency use applies to the treatment of one patient only and is not the same as IRB approval to conduct a research study. Expanded access, sometimes called "compassionate use," is the use outside of a clinical trial of an investigational medical product.
What is the difference between compassionate use and emergency use?
Compassionate use means that the use does not meet the criteria for emergency use, and therefore prior FDA approval (IDE) is required before the device can be used. Standard IRB review and approval (like a research study) is required for neither emergency nor compassionate use.
Is compassionate use the same as right to try?
What is the Food & Drug Administration's (FDA) Expanded Access Program? Also known as “compassionate use,” this program allows people with immediately life-threatening conditions or serious diseases or conditions to gain access to investigational medical products.
Can an oncology patient be allowed to try an experimental treatment?
Right to try, like expanded access, allows the use of an experimental drug outside of clinical trials to treat people with serious or life-threatening diseases. But unlike expanded access, it is not regulated by the FDA. You have an illness that is life-threatening. There are no standard treatments available for you.
Are patients entitled to refuse experimental drugs?
A qualifying individual may petition to access an experimental therapy, but that does not mean they will receive it. Drug companies still have the authority to refuse any request.
Do dying patients have a right to experimental treatments?
The requirements for granting approval of experimental drug treatment under Right-To-Try laws are: (1) a terminally ill patient has exhausted all other treatment options and is ineligible to participate in clinical trials, (2) the experimental drug passes FDA phase 1 clinical testing, (3) the patients' health-care ...
What is the difference between expanded access and compassionate use?
Expanded access, also called “compassionate use,” provides a pathway for patients to gain access to investigational drugs, biologics, and medical devices used to diagnose, monitor, or treat patients with serious diseases or conditions for which there are no comparable or satisfactory therapy options available outside ...
How do you dispense compassionate drugs?
Requests for the compassionate use of an investigational drug must be made via a request from the patient's doctor to the drug company requesting their permission for its use and their willingness to supply the drug for the patient's use.
When did compassionate use start?
In the US, compassionate use started with the provision of investigational medicine to certain patients in the late 1970s, and a formal program was established in 1987 in response to HIV/AIDS patients requesting access to drugs in development.
What is the difference between expanded access and compassionate use?
Expanded access, also called “compassionate use,” provides a pathway for patients to gain access to investigational drugs, biologics, and medical devices used to diagnose, monitor, or treat patients with serious diseases or conditions for which there are no comparable or satisfactory therapy options available outside ...
What is a compassionate use IND application?
Expanded access, sometimes called "compassionate use," is the use of investigational new drug products outside of clinical trials to treat patients with serious or immediately life-threatening diseases or conditions when there are no comparable or satisfactory alternative treatment options.
When did compassionate use start?
In the US, compassionate use started with the provision of investigational medicine to certain patients in the late 1970s, and a formal program was established in 1987 in response to HIV/AIDS patients requesting access to drugs in development.
What is the Texas compassionate use Act?
The Texas Compassionate-Use Act established requirements for the licensing and registration of dispensaries who provide low-THC cannabis to qualified patients. This chapter of Texas law allows qualified physicians to prescribe low-THC cannabis to certain qualifying patients.
What Is Compassionate Drug use?
Medical professionals use the term “compassionate use” to refer to the treatment of a seriously ill patient using a new, unapproved drug when no ot...
Is Compassionate Drug Use legal?
Compassionate drug use is legal, but it’s tightly restricted to people who meet certain conditions. For people who aren’t in clinical trials, a dru...
Who Might Benefit from Using Unapproved Drugs?
According to guidelines from the National Cancer Institute, most compassionate drug use is for patients who meet all of these conditions: 1. Have u...
Is It Difficult to Get Drugs For Compassionate use?
Perhaps the biggest problem with compassionate drug use is that it’s often hard to get the drug. The simplest way to get an unapproved drug is thro...
What Should I Ask My Doctor About Compassionate Drug use?
Here are some questions you may want to ask if your doctor is thinking about compassionate drug use: 1. Are there any approved treatments that I ha...
Where Do I Start If I Want to Apply For Compassionate Drug use?
Your doctor or one of the office staff will work with you on this process. The US Food and Drug Administration, Center for Drug Evaluation and Rese...
What is compassionate drug use?
Compassionate drug use means making a new, unapproved drug available to treat a seriously ill patient when no other treatments are available. Drugs that are being tested but have not yet been approved by the US Food and Drug Administration (FDA) are called investigational drugs. These drugs are normally available only to people who are taking part ...
What is the right to try act?
The Right to Try Act. A federal law passed in 2018 gave patients another path to access unapproved drugs, without needing the approval of the FDA. This pathway is commonly referred to as Right to Try. Right to Try laws do not replace EAPs but provide another way to access unapproved drugs.
How to contact the FDA?
If so, the next step is to contact the FDA's Center for Drug Evaluation and Research (CDER) at 1-301-796-3400 or fda.gov/drugs or the after-hours FDA Emergency Call Center at 1-866-300-4374 (toll free). See the FDA's information on EAPs to learn more.
Why are people not in clinical trials?
This could be because they live too far from locations where the studies are being done, or because they don't meet the requirements. People who aren’t in clinical trials might be able to get access to an unapproved drug from the company that makes it in 2 ways: Through expanded access programs (EAPs).
How long does it take for an expanded access program to be approved?
Expanded access programs (EAPs) The FDA accepts applications for expanded access every day of the week. It usually takes 4 days to process a non-emergency request, and less than one day to process an emergency request. Requests are made through your doctor.
Why is access denied?
There are a couple of reason access may be denied, including: If the patient does not meet the criteria spelled out in the law. If the drug company refuses to provide the drug, or they aren't able to make enough of it for all of the people requesting it.
Can patients get compassionate treatment?
Patients with serious or life-threatening conditions who can't get treatment with an unapproved drug through a clinical trial might benefit from compassionate use, if it's available.
Why are compassionate medical qualifications important?
First, patients who wish to be treated with investigational drugs on a compassionate use basis have very serious medical conditions. Second, any use of drugs with unproven safety and efficacy is associated with a high risk of side effects or other complications. Furthermore, when considering a patient for compassionate use, the physician should take into account not only medical aspects, but also the complex ethical dilemmas inherently connected with the use of unapproved drugs. Therefore, the physician should possess not only high professional qualifications, but also have some background of ethical training, or at least have an awareness of the ethical aspects of compassionate use. In this regard, note that the Declaration of Helsinki states that, “medical research involving human subjects must be conducted only by individuals with the appropriate ethics and scientific education, training and qualifications” (General Principles section, paragraph 12 [ 12 ]). The Good Clinical Practice standards [ 14] also state that the qualifications of the principal investigator should be evaluated during the independent ethical review of a clinical trial proposal (Institutional Review Board/Independent Ethical Committee section, paragraphs 3.1.2 and 3.1.3).
What should be considered in ethical review of compassionate use?
The independent ethical review of compassionate use should consider protocols (in cases of programs involving groups of patients), available drug safety and efficacy evidence, informed consent forms, medical justifications for using an unapproved drug, and the administering physician’s qualifications.
What is compassionate use?
Compassionate use is the use of unapproved drugs outside of clinical trials. So far, compassionate use regulations have been introduced in the US, Canada, many European countries, Australia and Brazil, and treatment on a compassionate use basis may be performed in Japan and China. However, there are important differences between relevant ...
Why is fair patient selection important?
Fair patients selection is essential for ethical compassionate use. Since it involves the use of investigational drugs, compassionate treatment is not considered standard clinical care. Consequently, problems of inequity may arise in terms of patients’ access to compassionate use programs.
What happens if a research is not scientifically valid?
Research results that are not scientifically valid might enter medical practice, leading to patient harm and/or a waste of resources. While the role of RECs in the review of biomedical research is commonly accepted, it is not the case with compassionate use.
What is independent review?
Independent review is a generally accepted ethical and legal principle for biomedical research [ 11 ]. All major international ethical and legal guidelines contain this principle, including the Declaration of Helsinki (World Medical Association) [ 12 ], the Additional Protocol to the Convention on Human Rights and Biomedicine Concerning Biomedical Research (Council of Europe) [ 13 ], the Guideline for Good Clinical Practice (International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use) [ 14 ], and the International Guidelines for Health-Related Research Involving Humans (Council for International Organizations of Medical Sciences with the World Health Organization) [ 15 ].
Where are compassionate use regulations already in place?
Compassionate use regulations have already been introduced in the USA, Canada, most European Union countries (e.g. France, Italy, Spain, and Germany), Australia, and Brazil [ 7, 8, 9 ].
What are the challenges of compassionate use of drugs?
However, there are challenges in ethically implementing compassionate use of the drugs due to limited sound scientific evidence, probability of therapeutic misconception, challenges related to autonomy and that of fairness, possibility of misuse for commercial gains, or being a substitute for clinical trials.
What is compassionate drug use?
Compassionate drug use is an important regulatory provision that is being proposed as an amendment to the New Drugs and Clinical Trial Rules, 2019 and will facilitate making provisions for unapproved interventions to needy patients in emergency situations and is a welcome step proposed by the drug regulator.
What are the requirements for clinical trials?
Clinical trials governed under the New Drugs and Clinical Trial Rules 2019 have mandated and described the requirements for having insurance policies, reporting of adverse events, medical management, and payment of compensation for related injuries.
What is the benefit of expanded use of drugs?
Benefit with expanded use of drug justifies the potential risks of treatment. Using the investigational drug will not interfere with clinical trials that were conducted for marketing approval of drug for the treatment indication.”.
Can compassionate use lead to clinical trials?
Compassionate use can however lead to difficulty in conducting clinical trials in the future for said drug as facilitation of expanded use of drug to large patients making requests can deplete supply of drugs for clinical research. [41] .
What is Ebola virus?
Ebola virus disease (EVD), formerly known as Ebola haemorrhagic fever is a severe form of zoonoses with fatal illnesses in humans. The 2014 outbreak of Ebola viral disease in some West African countries such as Guinea, Liberia, Mali, Nigeria, Senegal, and Sierra Leone, spread beyond the African borders to Spain and the United States.
How long does it take for a person to recover from Ebola?
This is usually followed by vomiting, diarrhea, skin rash, red eyes, impaired kidney and liver function, and in some cases, both internal and external bleeding occur.6Symptoms may appear between 2 to 21 days after exposure to Ebola virus. Some who fall sick are able to recover while others do not.
What is monitored compassionate use?
Monitored compassionate use has the goal of saving human life, reducing the suffering of the patients and promoting human dignity in health care systems. To deny access on such ground would amount to a grave injustice.
What is the FDA's focus?
The patient, however, is the central focus and such uses are without control groups .25The US Food and Drug Administration (FDA), which regulates drugs administration and evaluates clinical trials sponsored by US agencies, also allows the use of untested drugs on compassionate ground.
What is the categorical imperative?
The commands of morality must be categorical imperatives, by which he means a moral law that must apply to all rational beings, regardless of their wants and feelings.11His formulation of the categorical imperative is to establish that a morally good action is based duty and not on consequentialist considerations.
Why is preventive health important in Africa?
Africans need to be pro-active and more health conscious just as the failing states in Africa should see as urgent, overhauling of their existing health care systems.
Is compassionate use ethical?
Given that the principle behind compassionate use primarily is about the patients, it then becomes ethical to allow patients to access any available drugs first. However, in a case that the medical personnel are patients and ‘first-spreaders’, the qualification for access becomes higher and thus ethical.
What does compassionate use mean?
Under “Compassionate Use” or “Expanded Access”, the Food and Drug Administration allows an unapproved drug still in development to go to a patient with few alternatives remaining, also absolving the drug maker of liability should the drug not work or cause harm.
How does PARP inhibitor work?
PARP inhibitors work by preventing DNA repair in cancer cells, and are thought to work especially well in patients with a particular gene mutation.
What is Andrea Sloan's treatment for?
Andrea Sloan is dying of ovarian cancer. Having exhausted all standard treatment options, her doctors say her best hope now is a new class of cancer drugs called PARP inhibitors. The California pharmaceutical company BioMarin makes one version of these drugs called BMN 673.
When did Sloan get cancer?
But Sloan says she’s willing to accept the risks – to roll the dice now that her doctors have tried everything else they can to save her life, including multiple rounds of surgery, chemotherapy, and radiation since she was first diagnosed with cancer in 2007. And her doctor supports her.
Where is Sloan's BMN 673?
Sloan says there are doses of BMN 673 sitting on shelves in the same hospital where she’s seeking treatment: M.D. Anderson Cancer Center. Sloan is a 45 year-old attorney in Austin, Texas, who has ovarian cancer. But she’s not getting the treatment because the company is refusing to give it to her.