As originally reported in EurekAlert,
creating effective rare disease pharmaceuticals is difficult. Yes, because it is expensive, there’s a small population to use in trials, and there’s limited research. Sometimes though, there are drugs and trials for therapies, as is the case for multiple sclerosis, but patients aren’t getting the best drug for them.
One problem in drug creation can be a misalignment between what aspects of the disease the patients want addressed and what the drug manufacturers focus on tackling, a new study says this is because trials are not asking for patient feedback. Patient feedback on drugs for multiple sclerosis (MS), says they wanted changes to their chronic fatigue and quality of life, while researchers were not taking these symptoms into consideration. How can we incorporate the patient perspective into the drug creation process so the end result is truly tailored to their needs?
(MS) is a neurological disorder in which the immune system damage mistakenly attacks the bodies nerve cells, damaging the communication between the brain and body. The disease effects individuals quite differently, making it both hard to diagnosis and hard to treat in a systematic way. Common symptoms include damage to the eyes, muscles, speech, bladder, coordination, inflammation, or numbness and weakness. The exact cause or cure are not known, though some treatment options exist. There are various subtypes of MS.
Patient-Feedback in Trials
Now, researchers are asking how they can reform the treatment options to make the drugs functions more reflective of the wishes of those using the drug.
Since 1995 for the first time, the FDA has approved access to a host of treatment options that aim to prevent the disease’s characteristic relapses. However, the conversation and objectives in the methodology of their drug creation are shifting, increasingly they prioritize individualized treatment. Paul Friedemann, Scientific Director of the Experimental and Clinical Research Center (ECRC), gives his vision of the new approach, explaining “If in future we design the studies in such a way that they are more closely orientated to the needs of patients, we will obtain study results that are more likely toenable us to provide patients with more targeted and individualizedmedical care.”
They found that while most patients responded that symptoms effecting their daily wellbeing were of utmost importance: the symptomatic chronic fatigue, pain, depression, loss of vision, or behaviour and cognitive impairments; instead drug researchers did not ask for patient feedback. Resultantly, the analysis shows they failed to take these needs into consideration.
Another issue was the short duration of the trials such that they do not pick up on side effects resulting from longterm use. This blindspot is uncomfortable for patients who will eventually find out.
The possibility of treatment options has bloomed in the last decades, today, individualized medical care and immunotherapies are getting its day. Because the disease is so patient specific, matching appropriate health care is vital for these patients. The studies say: listen to the patient’s experience and responses. Many advocates agree: patients have insights that those without their condition do not, and communicate is key.