The terminally ill and their right to drugs

Excerpts

In the United States people who need medications to treat illness are dependent on the mercy of Food and Drug Administration (FDA). It serves as a gatekeeper to drug services, deciding which medications will and will not be available to consumers. The FDA demands that developers and manufacturers of drugs furnish data on a drug's efficacy and side effects, which it uses to make its determinations. Some organizations are demanding a change in the process to permit terminally ill people to gain speedier access to possible cures. Their demands have so far been rebuffed by regulators. Below I argue the case for liberalizing the approval process.....

Opposition to allowing terminally ill people to bypass the drug testing and approval process involves three criticisms. First, that the court decision creates a "right to experimental drugs"; that is, it creates a positive right that will impose on certain people an obligation to provide other people with experimental medications.

Second, that giving people access to experimental drugs outside of clinical trials will lessen the incentives for patients to participate in randomized, controlled trials that the FDA uses to determine whether drugs are "safe and effective," its criteria for drug approval. Thus, giving access to drugs outside the clinical trials process will undermine the FDA's ability to evaluate the risks and benefits of drugs and decide which ones deserve approval and which ones do not.

Third, that very ill patients are desperate, may not be able to rationally evaluate the choices available to them and will tend to pursue any remedy anyone claims will help them regardless of its risks or effectiveness. It follows, therefore, that such patients need to be protected from the possible adverse consequences of non-rational decisions they might make as a result.

All of these criticisms are flawed. For purposes of this argument, I will leave aside the more fundamental question of whether the FDA should even have a role in deciding which drugs are "safe and effective" and should be available to consumers. Although that is an important issue, which has been well argued by others, [9] [10] the changes sought in this court case could represent an improvement in the system we have to live under now. If objections to it can be addressed, this kind of incremental change could benefit at least some very ill patients who are now being harmed by the current system, and the change sought by the Abigail Alliance can result in a system that is both more just and more beneficial for all patients.

The "right" to experimental drugs

The first argument against access to experimental drugs for the terminally ill springs from the idea that the court victory by the Abigail Alliance creates a positive right to certain drugs. This is erroneous. The right at issue in the court case, and in this article, is a negative right: the right of a willing drug manufacturer and a willing drug consumer to engage in a voluntary transaction for a medication without interference from a third party, such as the FDA.

It has been suggested that requiring the FDA to allow consumers access to certain untested drugs may create a legal avenue to force insurers to pay for them. However, this is a defect created by government intervention in the insurance market, not a logical consequence of the right under consideration.

The drug testing process: patients in conflict?

The second major argument is that the drug testing process needs to be a balancing of opposing interests, with the interests of terminally ill patients who immediately want untested drugs balanced against those of less critically ill patients who must wait for drugs to receive FDA approval. Such approval will not be forthcoming until clinical trials have been completed and the drugs' risks and benefits are defined.

By allowing free access to drugs that have not gone through trials, the argument goes, we undercut the incentives for individuals who want the drugs to participate in trials. This ends up sacrificing the interests of those who will not get access to a drug until it has been further tested. Thus, individuals who might benefit from access to experimental drugs outside of trials benefit at the expense of other patients and society in general, and "The benefit of a few desperate patients would come at a steep cost to the rest of us." But the assumptions underlying these objections are not necessarily true, and basing the drug approval system on them is unjust.

First, it is doubtful that giving patients access to drugs outside the clinical trial system will undercut the incentives to participate in trials. People have different preferences and tolerances for risk. Based on risk preferences, patients seeking treatment for a disease can roughly be divided into two groups. One group consists of those who are willing to try untested or barely tested drugs. This may be because they have run out of meaningful therapeutic options (their disease is refractory to treatment with available therapies), or existing therapies don't do enough for their disease to give them the quality of life they desire.

They may also feel that having people who are willing to try untested drugs first is an important part of advancing the knowledge and science of disease therapy. The second group consists of patients who are more risk-averse. These patients do not want to take drugs unless they have been tested and their risks and benefits are defined to some extent (i.e., the drugs have been tested on other human beings first and the outcomes of this testing are known). Although it tends to be overlooked, there may be seriously or terminally ill patients who fall into this second group.

Some people in the first group of patients might want access to drugs that have not gone through the clinical trial system. But since they are willing to tolerate risk from drugs that have unknown effects, they also provide a pool of people who are willing to participate in clinical trials, for their own benefit and the greater benefit of others with the same illnesses, because they are willing to go first.

The only way the clinical trial system breaks down is if every single patient with a given disease wants immediate access to drugs before they have gone through any trials. Unless every patient with a given disease has the exact same level of risk tolerance, this is an unlikely scenario. Instead, the dynamics of the market can meet the needs both of patients who want immediate access to drugs and those who want better-tested drugs.

It's vital to keep in mind that manufacturers of drugs develop and market them because they see an unmet demand for the drug, and thus a possible profit opportunity. Some of that unmet demand and profit opportunity will be among patients who want drugs immediately, and are willing to take the risks involved in being the first to try them. But meeting the demand only of these patients ignores another profit opportunity: the demand created by individuals who want drugs whose risks and benefits have been defined to a greater extent by testing on other individuals. The more risk-averse second group - the individuals hesitant to try drugs that haven't been tested on others first - will still drive the demand for medications that have been rigorously tested in the clinical trial setting.

Even if drug manufacturers were permitted to sell untested medications to patients who wanted them, they would be marketing to the few people willing to take an untested or barely tested drug. It is doubtful that these people will create a market so profitable that drug companies will abandon the profit opportunities presented by drugs that have gone through the clinical trials process, and simply start selling untested drugs directly.

The larger drug market, and hence unexploited profit opportunities, will still be people who want drugs that have been through the clinical trial process. The market will adapt to meet this demand for testing and information, sustaining a system of clinical trials done to meet FDA approval standards. In this way, giving access to experimental drugs outside of trials does not actually undercut the clinical trial-based FDA approval system.....

Paternalism for the Terminally Ill

The third major argument against access to experimental drugs - that of protecting the very ill from their own choices - is a thornier problem because it rests on the same attitude of paternalism that is the entire underlying justification for the FDA. It assumes that certain individuals possess the ability to decide when other individuals are incapable of making good or rational decisions on a subject, and should be able to substitute their own judgment accordingly, forcing the original person to live with the substitute decision-maker's choice.

Note that by "ability to decide" I mean the supposed capability of one person to make a better decision for another person than that person would have made for themselves, not simply to the power Congress has conferred on some people to make other people's decisions for them. The government can give you the ability to make choices for other people. That does not mean that your choices are automatically superior; it simply imposes your own risk preferences on another person. [9] But the argument for substituting the "more rational" judgment of regulators for the "less rational" judgment of terminally ill patients is flawed.

If we are using the standard of rationality to justify paternalism, we must immediately confront the question of how we judge a patient's ability to decide rationally. Bioethicist Ezekiel Emanuel defines "the desire for unproven treatments by a few patients" as "the height of irrationality born of desperation." The first major problem with this is that individuals make real choices to try untested drugs in the context of clinical trials all the time. If they did not, these trials would not exist!.....

So is it possible to assess terminally ill patients' choices at all? Here, we can borrow the central economic insight that all value is subjective. This is usually applied in the context of individuals evaluating economic goods. But we can also apply this insight when we consider how people form valuations of the risks and benefits of disease treatment options: ".both the expected benefit of using a product and the burden of risk bearing are subjectively experienced and knowable only to the individual actor."

Thus, even the way that different people value the quality of their own lives, the value of a life with or without disability, the value of a risk to their life or health, and the value of a tradeoff between the two, is subjective. The value of those kinds of tradeoffs is not the same for every disease, or even for every individual with the same disease. ....

If the value of competing therapeutic alternatives for individual patients is subjective, then there is no objective standard an expert can use to determine how other individuals do value or should value their lives and their health. Yet we allow regulators to carry out this kind of valuation for patients all the time, without asking them if the patients themselves want it done for them.....

The value attached to different risks and benefits cannot be measured or defined in any way that would make it possible for one person to claim that they can make a more rational decision about them than another person. How, then, can we base people's access to drugs on the idea that some people can, and will, make better decisions for patients than those patients would make for themselves? We cannot, and should not, allow access to medical innovation for the sickest people to rest on such a weak and unjust foundation.

Conclusion

Although it may seem like a small victory, reforming the system for the most seriously ill is progress not only for them, but for every patient. As fundamentally flawed as the FDA drug approval system is, it is entrenched, and the majority of people are convinced that we cannot survive without it. Even so, the situation is not hopeless. We can take on the arguments justifying the FDA one at a time. We can argue convincingly that incremental reforms making drugs more widely accessible to more patients will not end in disaster, bring medical progress to a halt, or leave a trail of dead and dying people in their wake.

Allowing terminally ill people to bypass the drug testing and approval process will not create a "right to experimental drugs." It will not destroy the incentives for patients to participate in the clinical trial system. It will not make it impossible to gather scientific data on how drugs work. What it will do is allow individuals who are fully capable of rational choice to make the most important choices of all according to their own values. For the sickest people, it is not only beneficial and just, it is a matter of life itself.

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Your regulators will protect you (again)

Employing a loony as a psychiatrist was a good one!

Vincent Berg, the Russian immigrant exposed as an allegedly bogus psychiatrist at Queensland's "Dr Death" medical inquiry, is undergoing treatment in a psychiatric ward at the Gold Coast Hospital. Mr Berg, 54, was scheduled to face a committal hearing in Southport Magistrates Court on the Gold Coast yesterday on a charge of indecently dealing with a boy under 16. But the hearing was adjourned to January 25 next year after Mr Berg's lawyer, David Gilmore, told the court his client had voluntarily admitted himself to the psychiatric ward.

Mr Berg, who was in court for the brief hearing, is accused of sexually molesting the teenage son of one of his patients when he was employed as a psychiatrist at Townsville Hospital in 2000. The allegation was revealed during the Queensland hospitals inquiry by commissioner Tony Morris. Magistrate Ron Kilner expressed frustration that committal proceedings had still not begun, despite Mr Berg's arrest in September last year. Mr Gilmore said the defence required a psychiatric assessment of Mr Berg before he could face a committal hearing.

But prosecutor Mark Whitbread said: "He has had an opportunity to see a large number of psychiatrists, and no one has been able to give him the report he desires." Outside court yesterday, Mr Gilmore said he expected Mr Berg to apply to have his case heard before the Mental Health Tribunal. Mr Berg could face further charges resulting from his 12-month tenure as a psychiatrist at Townsville Hospital, using allegedly bogus qualifications. Geoff Davies, who took over from Mr Morris as health inquiry commissioner, recommended in his final report that police should investigate whether Mr Berg should be charged with fraud, forgery and "attempts to procure unauthorised status".

Source

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For greatest efficiency, lowest cost and maximum choice, ALL hospitals and health insurance schemes should be privately owned and run -- with government-paid vouchers for the very poor and minimal regulation. Both Australia and Sweden have large private sector health systems with government reimbursement for privately-provided services so can a purely private system with some level of government reimbursement or insurance for the poor be so hard to do?

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