#apaperaday: Sharing “Negative” Results in Neuromuscular Research: A Positive Experience
In today’s #apaperaday, Prof. Aartsma-Rus reads and comments on the paper titled: Sharing “Negative” Results in Neuromuscular Research: A Positive Experience
Today’s pick is this paper from Virginia Arechavala and yours truly on the positive experience of sharing “negative results” in Journal of Neuromuscular Disease to commemorate today’s session on this topic. DOI: 10.3233/JND-219007
AON Delivery is big not only on ways to improve delivery of antisense therapies by having consensus on model systems and studying safety, but also on sharing ALL results, whether expected/hoped for or not. The Action coordinated special issues on these topics in 2 journals!
- Mary Ann Liebert Pub Nucleic Acid Therapeutics had a special issue on this topic focusing on antisense oligonucleotide experiments. Then, because 1 issue was not enough..
- The Journal of Neuromuscular Disease had a special issue too. To sustain publishing unexpected results they have a section ‘the null hypothesis stands’. #apaperaday is the editorial to the inaugural issue dedicated to this section with 8 publications on unexpected results or non replicated studies
The reason for publishing unexpected results & results that are not replicable are obvious: avoids wasting money, work & effort. AON Delivery did a survey with its members, where the vast majority was in favor of publishing these results, but most did not pursue it themselves.
There is still a negative connotation with ‘negative results’. However, a scientist comes up with hypotheses and then tests them. If the hypothesis is wrong, that does not mean the scientist was not clever – often they got funding after peer review to test the hypothesis. The fact that things are not as assumed is part of science, and actually when things do not work out as anticipated that can teach us – sometimes more! When previous results cannot be replicated, especially in therapeutic research, often researchers think “I did something wrong”.
However, that is not necessarily true. Sometimes results may seem positive by accident, or are only positive in an extremely controlled environment. Validation experiments are crucial to confirm robustness, as treatment in patients is not ‘an extremely controlled environment’.
As mentioned, the inaugural Journal of Neuromuscular Disease issue on ‘the null hypothesis stands’ contains 8 publications, including a clinical trial in Duchenne on edasalonexent, where authors are commended for publishing the results quickly so the valuable data could be shared with the community.
Because even when a phase 3 clinical trial shows the therapy is not effective, the trial produces a wealth of data that is useful for other research and planning and designing of future trials. Publishing ‘negative results’ helps others to continue and build on the work.