For quite a few years, I have been strongly advocating the use of Provigil the brand name for modafinil for helping with sleep related issues n Myotonic Dystrophy. Many patients with myotonic dystrophy have tremendous issues with excessive sleep and sleep disruptions.  A study from France in 2009 concluded that a controlled study showed “no significant effects on daytime sleepiness” This study puzzled me as many patients reported very good results from this drug.

A survey conducted by the Myotonic Dystrophy Support Group,  MDSG in England has concluded that over 80% of survey respondents found that the drug had a marked benefit or a dramatic benefit. This concurs with the informal feedback that I have had here in the USA. This also focuses attention on patient centered approaches versus medical centered approaches. Patient centered approaches are very important to give direct patient feedback rather than just visibility to medical professionals opinions based on limited patient facts.

The drug provigil is not approved in the USA for Myotonic Dystrophy. That means that patients need to pay for the drug themselves as insurance companies increasingly only pay for drugs that have approved uses. Again the insurance companies are taking the medical perspective not the patient perspective. The insurance companies are also overriding the doctors prerogative in prescribing off label drug use.

Provigil is approved for narcolepsy, shift work disorder and obstructive sleep apnea. So your doctor in the USA will have to diagnosis you with these diseases if you wish to get a Rx that the insurance will pay for.

Of the 145 people who returned the survey 27 had stopped using the drug 20 did so because of side effects and 7 because it did not seem to work for them. There were some side effects. Insomnia (can get to sleep) was reported in eight people and Headaches in 4 people 2 people reported heart palpitations.

The heart palpitations are important was cardiac issues and atrial flutter the heart going to fast and not pumping well are very significant issues in myotonic dystrophy. If you are using this drug you need to inform your cardiologist and make sure your doctor knows about the heart issues. The study recommends careful monitoring of the heart function and recording of adverse events such as heart or skin issues.

On the relatives side 146 returned a questionnaire and 85% said that the drug had a marked or dramatic benefit. Side effects were:

8  headaches
9  insomnia
2  skin problems
2  Heart palpitations

The patients were clear in the benefits of this drug with the disease. Over 80% said they would be very disappointed or devastated if the drug were no longer available. IT would also vastly affect their lives. The drug supports a more active social life (See blog on Social disruptions)  A number of comments also offer up that the drug is important to keep them working “I feel without this drug my husband would not have a job, or much of a life”

Whats clear is the Medical Professionals blew it,  with the studies that have been published. They did not include patients preferences and outcomes in the studies that published. Patient outcomes such as employment and increased social interactions are key metrics to look at from a national perspective of the effectiveness of a drug. They looked at medical issues from a medical perspective and not on how they affect patients. Hopefully the medical community can learn from this.