When rare disease patients take part in clinical trials, they may never be able to see the results of the clinical trial that they took part in. Negative results are often not published, simply because by time the results are known, companies or investigators are too busy picking up the pieces and moving onto the next project. Sometimes the companies may not want to publish results that could help their competitors. This results in challenges for patients who took part in the clinical trials, because they feel “in the dark” about whether or not the drug worked overall, and if there were reasons why the drug was not ultimately approved. Sometimes the drug may eventually be approved, but it could take time, because the company or Sponsor may need to complete a number of additional clinical trials before they can submit or resubmit the application for approval.
How should patients and carers approach this situation? Patients can contact the companies and ask for information about the status of the drugs that were in clinical trials. Drug Information Departments many years ago, were trained to not speak to patients. Hopefully by now, there are specialists who are able to speak to patients and provide information, without breaching confidentiality, that will be helpful to patients.
Do you have any concerns about studies you took part in that have effectively been buried, because the results have never seen the light of day?
Lorna Speid, Ph.D.
Founder and President
