Why do we Need Transparency in Clinical Trials?

Recently, there has been a lot of debate surrounding the subject of publishing all clinical trials results. One petition has been making a lot of noise: the All Trials Registered petition, which the AKU Society has signed.

We support this initiative because we believe it is time for all clinical trial results to be reported. Around half of all clinical trial results have not been published, and some trials have not even been registered.

But why does this matter?

Not reporting or publishing all clinical trials results leads to a lack of information. Doctors and patients cannot make informed decisions about the treatments they choose without all of the facts. Currently, companies and researchers can choose to withhold the results of clinical trials even when they are asked for them.

They may only report the good outcomes of the drug, but not the bad.

Imagine if this was you: there is a new treatment that could possibly ease some of your symptoms. You research it thoroughly, finding only good results. The recent clinical trial on the drug reported a good outcome. You start taking the medicine, but then find out that these good results only accounted for 10% of the actual findings. Effectively, this drug only worked for 10% of people within the clinical trial. You have been wasting your time all along. As a worst case scenario, your condition has significantly deteriorated, you have endangered your health and you end up suffering from unreported side effects.

Now imagine if you were the doctor who prescribed this drug. You meant to help your patient, but instead you only caused them harm.

This is exactly what happened to Ben Goldacre, previously a GP and now the driving force behind the All Trials Registered petition. In clinical trials, the drug he prescribed had shown no improvement over the placebo and it had caused more side effects. This data was withheld.

Writing about the experience in his recent book, Bad Pharma, he said: “I did everything a doctor is supposed to do. I read all the papers, I critically appraised them, I understood them, I discussed them with the patient, and we made a decision together, based on the evidence… As a doctor, I did something which, on the balance of all the evidence, harmed my patient, simply because unflattering data was left unpublished.”

“As a doctor, I did something which […] harmed my patient, simply because unflattering data was left unpublished.”

This is a problem which may be even worse for those affected by rare or orphan diseases.

As Ben Goldacre says in Bad Pharma: “People working on orphan diseases will often research existing drugs that have been tried and failed in other conditions, but that have theoretical potential for the orphan disease. If the data from earlier work on these drugs in other diseases is missing, then the job of researching them for the orphan disease is both harder and more dangerous.”

Flavia Diniz Mayrink, a rare disease patient with alkaptonuria (AKU), agrees that all clinical trial results must be published.

“It is really important to know ALL the clinical trials results, in order to know the impact of the trials on our lives. It is even more important to know the reports of clinical trials for rare diseases, as they are even scarcer than clinical trials for chronic diseases or the ones that often give more profit to the industries involved. All the information is needed for lives to get better.”

“All the information is needed for lives to get better.”

The rare disease sector is becoming an extremely profitable one for companies and researchers. Under EU law, pharmaceutical companies in Europe can sell an orphan drug for ten years with no competition from other companies. This means they have exclusivity. Many orphan drugs are extremely expensive. An example of this is Soliris, used for the treatment of the rare bone marrow disease Paroxysymal Nocturnal Hemoglobinuria, costing a massive £250,000 a year.

With this in mind, the potential for companies to withhold orphan drug clinical trial results is huge. Rare diseases are often debilitating, disabling and can even be fatal. But most have no effective treatment or cure, meaning patients are desperate for a drug or treatment to help them. If results are unpublished, rare disease patients could find themselves wasting their time and money with drugs that won’t help them – and may even complicate their symptoms with nasty side effects.

Clinical trials are undeniably necessary: they’re the only way we have of finding out what works and what doesn’t. It’s also important to note that generally, clinical trials are safe. Drug companies have improved, extended and saved lives by developing some extraordinary drugs and treatments. But for this to continue, and for us to be able to trust new treatments, all results must be published. We’re taking part in a clinical trial this year, to find out if the drug nitisinone can treat AKU patients. All of the results of our trial will be published, no matter the outcome.

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