Regulation and associated testing procedures vary across the world, but America is home to the biggest pharmaceutical industry, and its regulations are fairly typical of those found in many developed countries. The road from early‑stage research to drugs available to patients can be broken down into six stages:
1. Preclinical Research. Scientists test different chemicals to see if they might have a role in medicine. This is likely to involve preliminary testing on animals to see if the chemical is likely to be sufficiently safe and effective. It will also take into consideration how the chemical might be mass produced if it turns out to be useful. This takes at least five years.
2. Clinical Studies, Phase I. The potential drug is given to between 10 and 100 volunteers to investigate safety in humans. The main goal of Phase I clinical studies is to identify a safe range of dosage. This takes between one and two years and costs roughly $10 million.
3. Clinical Studies, Phase II. The drug is given to between 50 and 500 patients with a relevant disease. The main goal is to gauge the effectiveness of the drug in humans. At the same time it is important to establish the optimum dosage and duration of treatment for the next stage of testing. Phase II takes two years and may cost a further $20 million.
4. Clinical Studies, Phase III. The drug is given to hundreds or thousands of patients to determine its effectiveness and any side‑effects. This usually involves randomized clinical trials and the drug is tested against a control group receiving a placebo or the best existing drug. For thoroughness, Phase III may involve two independent studies. It might generate a further stage of research if the drug seems to be effective only for a subset of patients, such as those in the early stages of the disease. Phase III takes three to four years and may cost $45 million.
5. Review by Food and Drug Administration (FDA). If Phase III has been successful, then it is possible that news of a breakthrough will reach the public. Before the drug is made available, however, the evidence has to be reviewed by the FDA in America, and its counterparts elsewhere, such as the European Agency for the Evaluation of Medicinal Products. This takes a further one or two years.
6. Post‑marketing Surveillance. Even when the drug has passed all the tests and is being prescribed or sold over the counter, doctors will still be alert to any adverse reactions and report them to the FDA. This ongoing monitoring is important just in case there is a small risk that was not identified at Phase III.
These inordinate costs were cited in the journal Scientific American in 2000, so they have probably increased significantly in the last few years. Moreover, only a third of those drugs that enter Phase I trials reach the stage of New Drug Application, which allows a drug to be marketed to the public.
In short, conventional medicines have to leap over extraordinarily high hurdles, which requires a vast amount of money and time, but this level of testing is essential if the public is to be protected from harmful and ineffective drugs. Such rigorous testing should bring reassurance to patients, and it would not be unreasonable to demand similar levels of testing and regulation for alternative medicine.
There is, however, an argument that suggests that the sequence of testing for alternative medicines should be reversed. This is for the simple reason that these treatments are already widely available, whereas the pharmacological approach outlined above was developed for entirely new drugs. If millions of people are already using treatments such as herbal remedies and acupuncture, then it seems most sensible to assess the safety of these treatments based on the experiences of patients. For instance, practitioners could be asked to note the details of any adverse reactions and post them to a central database. The next priority would be to submit alternative therapies to clinical trials in order to find out for what (if any) conditions they are effective. Finally, if these tests were positive for a particular alternative therapy, scientists might investigate the mechanism behind the therapy and conduct the preclinical research.
This reversed approach puts patient safety first, because the consumer is already being exposed to alternative medicine, but it ultimately demands the same level of scientific rigour. Conducting such rigorous testing would be expensive, but remember that alternative medicine is a billion‑dollar global industry, so it would not be unfair for it to devote part of its vast profits towards properly testing the products that it sells to the public. Moreover, governments already have funds for medical research, and they could act in a coordinated manner to devote a fraction of this money towards high quality clinical trials focused on the biggest selling products and the most popular therapies.
The reversed testing procedure, like the normal procedure, would take several years to complete. In the meantime, while awaiting the results, governments could require alternative remedies to carry labels and compel therapists to make disclosures that accurately reflect the existing evidence. Dylan Evans suggested exactly this idea in his book Placebo. For homeopathic remedies, he suggested the following labeclass="underline"
Homeopathy
Warning: this product is a placebo. It will work only if you believe in homeopathy, and only for certain conditions such as pain and depression. Even then, it is not likely to be as powerful as orthodox drugs. You may get fewer side‑effects from this treatment than from a drug, but you will probably also get less benefit.
We believe that Evans’s idea has some merit, because such open, honest and accurate summaries would certainly help patients. For alternative treatments supported by evidence, the summaries would contain the sort of helpful advice that we see on conventional pharmaceuticals. For other alternative treatments, the summaries would read more like a statutory health warning, akin to those found on cigarette packets.
As we approach the final few pages of the final chapter, it is interesting to see what happens if we apply Evans’s idea to a selection of alternative therapies discussed elsewhere in this book. In some cases, the Evans‑style summaries would appear on boxes of tablets, while in other cases they might appear on websites or in leaflets distributed in a clinic. And in every case, the summaries would help patients to be more aware of the evidence relating to any particular therapy.
Acupuncture
Warning: this treatment has shown only very limited evidence that it can treat some types of pain and nausea. If it is effective for these conditions, then its benefits appear to be short‑lived and minor. It is more expensive than conventional treatments, and very likely to be less effective. It is likely that its major impact is as a placebo in treating pain and nausea. In the treatment of all other conditions, acupuncture either has no effect other than a placebo effect. It is a largely safe treatment when practised by a trained acupuncturist.
Chiropractic
Warning: this treatment carries the risk of stroke and death if spinal manipulation is applied to the neck. Elsewhere on the spine, chiropractic therapy is relatively safe. It has shown some evidence of benefit in the treatment of back pain, but conventional treatments are usually equally effective and much cheaper. In the treatment of all other conditions, chiropractic therapy is ineffective except that it might act as a placebo.