Should Dying Patients Have the Right to Access Experimental Treatments?

“In the last six months Colorado, Louisiana, Missouri, Michigan and, most recently, Arizona have passed ‘right to try’ laws that allow terminally ill patients to access treatments that have only passed FDA Phase I clinical trials. All patients need is permission from a drug company and a prescription from a doctor.

“Right to try laws are designed to ensure that terminally ill patients taking part in clinical trials are true volunteers and have no incentive to cheat the clinical trials system as has happened in the past.

“Recently, these laws have been critiqued as misguided, and the ethics of allowing patients to use experimental drugs are still up for debate. These laws do not guarantee access to experimental treatments and patients may have to pay for them out of pocket.

“Critics of these laws worry that alternative trial designs, or access to such experimental drugs outside the clinical trials system will significantly delay the development of effective therapies.”


‘Early Access’ Scheme Unveiled for Innovative Medicines

“People with advanced cancer and other serious illnesses like dementia could soon benefit from early access to innovative and promising treatments.

“The Early Access to Medicines scheme would enable safe and promising drugs to be ‘fast tracked’ into the NHS before they have even been granted a licence for use.

“The new scheme will allow patients without other treatment options to be given experimental drugs that have not yet been licensed but that have been deemed safe and effective through clinical trials.

“Experts will carry out a rapid analysis of the treatment before labelling it a ‘promising innovative medicine’.

“The Medicines and Healthcare products Regulatory Agency (MHRA) will then offer a scientific opinion based on a medicine’s risks and benefits. If the benefits are found to outweigh the risks, doctors will be given the green light to offer the drug to patients.”

Editor’s Note: This UK program is similar to a program already in place in the US that allows patients to request access to drugs still being tested in clinical trials. Even though so-called “compassionate access” is possible, the process can be quite difficult for patients. Learn more about it  from our blog feature on the topic.


‘Early Access’ Scheme Unveiled for Innovative Medicines

“People with advanced cancer and other serious illnesses like dementia could soon benefit from early access to innovative and promising treatments.

“The Early Access to Medicines scheme would enable safe and promising drugs to be ‘fast tracked’ into the NHS before they have even been granted a licence for use.

“The new scheme will allow patients without other treatment options to be given experimental drugs that have not yet been licensed but that have been deemed safe and effective through clinical trials.

“Experts will carry out a rapid analysis of the treatment before labelling it a ‘promising innovative medicine’.

“The Medicines and Healthcare products Regulatory Agency (MHRA) will then offer a scientific opinion based on a medicine’s risks and benefits. If the benefits are found to outweigh the risks, doctors will be given the green light to offer the drug to patients.”

Editor’s Note: This UK program is similar to a program already in place in the US that allows patients to request access to drugs still being tested in clinical trials. Even though so-called “compassionate access” is possible, the process can be quite difficult for patients. Learn more about it  from our blog feature on the topic.


‘Early Access’ Scheme Unveiled for Innovative Medicines

“People with advanced cancer and other serious illnesses like dementia could soon benefit from early access to innovative and promising treatments.

“The Early Access to Medicines scheme would enable safe and promising drugs to be ‘fast tracked’ into the NHS before they have even been granted a licence for use.

“The new scheme will allow patients without other treatment options to be given experimental drugs that have not yet been licensed but that have been deemed safe and effective through clinical trials.

“Experts will carry out a rapid analysis of the treatment before labelling it a ‘promising innovative medicine’.

“The Medicines and Healthcare products Regulatory Agency (MHRA) will then offer a scientific opinion based on a medicine’s risks and benefits. If the benefits are found to outweigh the risks, doctors will be given the green light to offer the drug to patients.”


Compassionate Use of Unapproved Drugs Raises Difficult Ethical Issues

Patients with terminal illnesses, including late-stage cancer patients, are understandably eager to try still-unapproved experimental drugs once other options have been exhausted. The U.S. Food and Drug Administration (FDA) recently created a program to make it easier for drug companies to grant ‘compassionate use,’ which allows patients in extraordinary need access to otherwise unauthorized treatments. However, the decision to dispense experimental drugs is left to the companies, which are often reluctant to take this risky step. Experimental drugs are just that–experimental; they may have catastrophic side effects or simply not work. When these drugs are given outside the framework of a well-designed clinical trial, it becomes impossible to decisively judge whether they actually work. And if all patients could bypass clinical trials, there would be no incentive for anyone to enroll in these clinical trials, meaning that new drugs would never be properly studied and approved. Compassionate use therefore needs to be approved by experienced medical professionals on a case-by-case basis, and remain an option of last resort only.


Compassionate Use of Unapproved Drugs Raises Difficult Ethical Issues

Patients with terminal illnesses, including late-stage cancer patients, are understandably eager to try still-unapproved experimental drugs once other options have been exhausted. The U.S. Food and Drug Administration (FDA) recently created a program to make it easier for drug companies to grant ‘compassionate use,’ which allows patients in extraordinary need access to otherwise unauthorized treatments. However, the decision to dispense experimental drugs is left to the companies, which are often reluctant to take this risky step. Experimental drugs are just that–experimental; they may have catastrophic side effects or simply not work. When these drugs are given outside the framework of a well-designed clinical trial, it becomes impossible to decisively judge whether they actually work. And if all patients could bypass clinical trials, there would be no incentive for anyone to enroll in these clinical trials, meaning that new drugs would never be properly studied and approved. Compassionate use therefore needs to be approved by experienced medical professionals on a case-by-case basis, and remain an option of last resort only.


Compassionate Use of Unapproved Drugs Raises Difficult Ethical Issues

Patients with terminal illnesses, including late-stage cancer patients, are understandably eager to try still-unapproved experimental drugs once other options have been exhausted. The U.S. Food and Drug Administration (FDA) recently created a program to make it easier for drug companies to grant ‘compassionate use,’ which allows patients in extraordinary need access to otherwise unauthorized treatments. However, the decision to dispense experimental drugs is left to the companies, which are often reluctant to take this risky step. Experimental drugs are just that–experimental; they may have catastrophic side effects or simply not work. When these drugs are given outside the framework of a well-designed clinical trial, it becomes impossible to decisively judge whether they actually work. And if all patients could bypass clinical trials, there would be no incentive for anyone to enroll in these clinical trials, meaning that new drugs would never be properly studied and approved. Compassionate use therefore needs to be approved by experienced medical professionals on a case-by-case basis, and remain an option of last resort only.