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Amyotrophic Lateral Sclerosis Case Study Essay

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a) Amyotrophic lateral sclerosis (ALS) is a serious debilitating disease with poor life expectancy of typically only 3-4 years from onset. Patients with ALS become rapidly and severely disabled losing the strength in their arms and legs and eventually lose the ability to speak. As these patients are clearly highly vulnerable, there are a number of study design and treatment aspects that a HREC would be concerned about. These are discussed below: • Intervention arms: The trial here wishes to evaluate a new drug for ALS compared to placebo. As there is a drug available to treat ALS (Riluzole)(1), the decision to use placebo over available treatment would need to be clearly justified and evaluated in detail. • That there is sufficient justification that the drug will be as least as effective as current methods of care. • That if the treatment is to be highly effective, would the trial have stopping rules to enable the placebo group to also be treated. • Access to additional drug use/physiotherapy for ALS symptom management: There are several additional medications that can help with the symptoms (e.g. spasticity, pain) of ALS during the course of the treatment (2). Physiotherapy in ALS patients has also shown to help alleviate fatigue and pain and may help to delay muscle weakening (3). Would patients (in both …show more content…

CTNs are used to notify the TGA that a clinical phase I trial is to be undertaken and can only be submitted once a local ethics committee has evaluated all the relevant scientific data related to the drug and its use in treating ALS (e.g. in laboratory experiments and animal studies). More than 98% of Phase I trials in Australia for new drugs use a CTN. A CTX is only used when the local ethics committee is not willing to review the scientific data and the data is then submitted to the TGA. CTXs are avoided where possible as they are more expensive and have longer processing

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