"Why is a drug license necessary? I posted on this topic last week, under the title off-label prescribing. Each country or region has an agency that licenses drugs; in Europe it is the European Medicines Agency (EMA), in the US the Food and Drug Administration (FDA), in Canada, Health Canada, in the UK, the Medicines and Healthcare products Regulatory Agency (MHRA), etc. These agencies make sure the drug has an appropriate risk:benefit ratio for the target population of patients with a specific disease or subtype of disease. In MS this could be a clinically-isolated syndrome, relapsing-remitting, secondary progressive or primary progressive disease.
If a license is turned down by the regulators or licensing agencies it generally means they are unconvinced by the risk:benefit ratio of the drug! In other words the drug has too many side effects or undefined risks to warrant its use given it effectiveness, or the drug is not effective.
A license by an agency is essentially a seal of approval and it then allows the drug to be marketed for its specific indication by the Pharma company concerned. Marketing is important as this allows neurologists to be approached and given information about the drug and its benefits. Without a license you can't inform anybody about the benefits and payers don't have to pay for the drug either. In the UK getting payment for an off-license drug, no matter what it costs, is getting increasingly difficult. In austerity Britain where the NHS has to save £20 billion over 5 years pennies count. Who are the payers? These are the people who decide on whether or not a drug or service is worth paying for; in the UK these are the so called Primary Care Trusts (PCTs), soon to be Commissioners. In other parts of the world payers are Insurance companies or Health Maintenance Organisations (HMOs).
What happens if you market a drug off-license? If the authorities find out the Pharma companies get huge fines. Recently some of these fines have come close to $3 billion. What happens if a neurologist promotes the use of a drug off license? Not much at present, but I if the drug is risky he or she will be putting themselves at risk of a medicolegal challenge. For example, if Dr Chataway started promoting the off-license use of Simvastatin at a dose of 80mg per day for progressive MS on the back of the results of one phase 2 study and an MSer happens to die from developing a rare complication of the treatment. Would Dr Chataway, the MSer or the neurologist who prescribed the drug be responsible? The courts will decide, but Dr Chataway would definitely have to accept some responsibility. If found guilty it could result in him being sanctioned by the General Medical Council. The point I am making is that we need to have the same level of evidence applied for all drugs for treating MS, we can't simply lower our scientific standards for off patent, cheaper drugs, hoping the community will take up prescribing with a lower evidence base.
As I get older I realise that scientific research is also a social science; i.e. how do you get the community to adopt you ideas and translate the evidence into clinical practice. Part of getting the neurologists of the world to use drug X for progressive MS is having a good enough evidence-base to convince them. An important part of the evidence base is the seal of approval of regulators. In other words we need to get drugs licensed. You would have more confidence in a licensed drug compared to an unlicensed drug. Or may be I am wrong?"
Labels: BPA, off-license drugs