A New Working Group
A team of healthcare professionals from the Institute for Quality and Efficacy in Health Care, the Drug Commission of the German Medical Association, and the Charité University Hospital Berlin in Germany, set about to reform clinical trials for multiple sclerosis (MS). The ultimate goal was to improve patient centrality by better taking into account patient preferences and ensuring personalized care.
The full set of their recommendations has been published in the EPMA Journal.
This team of researchers investigated 29 Phase 3 MS trials. All of the therapies these trials were investigating have received approval. These treatments are for clinically isolated syndrome, primary progressive MS, secondary progressive MS, and relapsing-remitting MS. The team analyzed the following for each trial:
- Trial duration
- Placebo/other comparator treatments
- Sample size
- Outcomes of magnetic resonance imaging
- Patient-reported outcomes
Ultimately, they found that patient perspectives are largely left out of the picture. Outcomes which are noted as “highly relevant” for MS patients were either not measured or were used as secondary/explorative objectives. These include depression, pain, cognitive impairment, fatigue, loss of vision, spasticity, and sleep disorders. Simultaneously, biomarkers of “dubious clinical importance” were studied frequently.
This group explains that by increasing our focus on variables most important to patients, we can better personalize care.
The team has offered 4 primary recommendations going forward.
- First and foremost, future trials need to focus more on patient experiences to ensure that individualized care is provided. That means that in addition to clinical findings, patient reported outcomes need to be measured. If this data isn’t collected, we will never have the full picture regarding the possibilities of the drug. They explain that it can be as simple as the addition of a patient questionnaire which asks about symptoms. This survey, they argue, should be established and validated internationally.
- Secondly, studies should include longer follow up on participants. Immunomodulating therapies often necessitate long term treatment. However, studies are normally short with minimal follow-up. This means they are missing potential side effects, complications, or other issues that could show up later after the treatment is administered for a longer period of time.
- Third, clinical trials should reform their enrollment criteria to ensure that the MS patients who are the most expected to benefit from the therapy are able to participate in the trial. This will also help to minimize safety concerns for participants.
- Fourth and finally, the team suggests that increased emphasis should focus on accuracy of diagnosis prior to trial enrollment. Misdiagnosis could mean the administering of harmful therapies. For instance, MS treatments may harm patients who have similar conditions to MS like neuromyelitis optica spectrum disorder.
Hopefully, these recommendations will be implemented quickly, improving outcomes and minimizing risks for current and future multiple sclerosis patients.
You can read more about this investigation here.