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A nightmare diagnosis

By Lorna Speid

Your worst nightmare has come to pass. You are given a diagnosis that has left you in a state of shock. The specialist told you there is nothing else that they can do for you.

“What was it that he said?” you ask yourself. “Did I hear him correctly?” you mutter to yourself. You are driving home, but you are on automatic pilot.

You remember back to last year when you began to feel unwell. Nothing very specific, just a general malaise. The general practitioner could find nothing wrong and so you didn’t insist on further investigations. The headaches became progressively worse and now this.

Each day, many people will face just this type of scenario. Diagnoses are given in as caring a manner as possible, and yet the life, which days before was taken for granted, now seems fleeting. Current medical treatments can only go so far.

What do we do when we have reached the end of the road with current medical science? What do patients do? What do their doctors do?

In the case of patients, they must obviously seek second, and maybe even third and fourth opinions to make sure it really is the end of the road for them. They must make sure that a correct diagnosis has been delivered by the appropriately trained and experienced specialists, on the basis of properly and thoroughly conducted tests and evaluations.

If it really is the end of the road in terms of currently available medical treatments, there may be opportunities to take part in clinical trials which may offer some hope. In a situation like the one cited above, the type of clinical trial that would be best would be a Phase 3 trial for which efficacy has already been demonstrated. In this case, the drug may be close to submission to major regulatory authorities for marketing authorization. Earlier studies should usually be avoided because they are less unlikely late stage clinical trials to yield the life-saving results required in this case.

Patients looking for options to save their lives may be easily misled into taking part in clinical trials for unproven experimental drugs. There are many ways that they can be inadvertently influenced to sign up for inappropriate clinical trials. The enthusiasm of the young doctor running the Phase 1 dose-finding study, could lead them to believe that the experimental drug is the next best thing since sliced bread. What’s in it for the investigator? A nice publication and perhaps a promotion. What’s in it for the sick patient? Likely, not very much except a contribution to medical science and the knowledge of how the drug behaves at the dose that they would be administered. What advice would I give to our fictitious but sadly realistic patient in the nightmare?

  1. Get as many opinions as necessary, as quickly as possible before giving up hope.
  2. Find appropriate clinical trials if currently available treatment will not be able to help in your situation.
  3. Don’t be swayed by the enthusiasm of an investigator to take part in a clinical trial. Ask the right questions to determine if the clinical trial is best for you.
  4. Ask for data from other patients that have already taken part in, or are already enrolled in the clinical trial. For instance:
    • What types of side effects and adverse events have been observed?
    • Has anyone died during the study?
    • If the answer to the above is yes, were the deaths considered probably related to the study drug?

Lorna Speid, B.Pharm, MRPharm.S., Ph.D., RAC, is the president of Speid and Associates, Inc., a regulatory affairs and drug development consultancy and the author of Clinical Trials: What Patients and Volunteers Need to Know. Dr. Speid has worked for the international pharmaceutical industry since the late 1980s. In addition to her work as a consultant, her earlier research and clinical trial experience in the pharmaceutical industry has given her invaluable insight into the issues faced by patients and healthy volunteers who take part in, or are considering taking part in, clinical trials. She lives in San Diego.

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Recent Comments

  1. Aidan O'Donnell

    This is a very interesting article, which deserves a careful reply.
    I believe that medical certainty (which is that doctors can decide which treatments work, based on their knowledge and experience) is a seductive illusion. The only way medical knowledge can be advanced at all is by doing research.
    Research is painful. A clinical trial requires randomising patients with a disease into two or more groups, one of which is given the study drug and one which isn’t. As a result, one group is (usually) going to do better than the other. Careful study design and good statistical analysis can give you an answer; without these the answer is only guesswork. The answer might be that the new treatment works, but it can also be that it’s no improvement, or even that it’s harmful. There is no guarantee either way.
    I believe patients view their prognosis in binary terms: either this drug will make me better, or it won’t. But in truth, the new treatment is likely to only be better than the best existing treatment by a few percent.
    Medical trials give answers which apply to populations of patients, not individuals. Yet unless those populations are studied (i.e. unless people volunteer at all phases) the results will be skewed.
    For you to recommend that individuals submit themselves only to phase 3 trials is irresponsible. If everyone did this, research would grind to a halt as no-one enrolled in phase 1 and 2 trials. Later there would be no phase 3 trials because the results of new phase 1 and 2 trials would be found to be meaningless. (If only some people take your advice, then you will introduce a hidden selection bias into the trial which may affect the results).
    You are completely right when you describe “what’s in it” for the investigator and the patient. But your phrase “inappropriate clinical trials” is very misleading. All recent drugs were once “unproven experimental drugs”. You make patients who volunteer themselves in trials sound like ill-informed guinea-pigs, when in fact each one makes a small but irreplaceable contribution to the advance of knowledge.
    There are still some trials which are poorly designed, poorly conducted or poorly analysed. But this doesn’t justify your advice to stay away from early clinical trials!

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