Table 1 Health state descriptions for the generic states

Controlled disease

• You have an inherited condition that may lead to you developing health problems. These problems could include tiredness, issues with your bones causing pain and becoming more likely to fracture, and enlargement of your liver and spleen which can result in serious complications.

• In order to try and prevent problems developing you are required to receive treatment for the rest of your life. The treatment is effective as long as it is taken according to instructions.

• You need to take the treatment on a regular basis. You need to consider your access to treatment when travelling.

Intravenous treatment

• You have an inherited condition that may lead to you developing health problems. These problems could include tiredness, issues with your bones causing pain and becoming more likely to fracture, and enlargement of your liver and spleen which can result in serious complications.

• In order to try and prevent problems developing you are required to receive treatment for the rest of your life. The treatment is effective as long as it is taken according to instructions.

• The treatment you need to take is administered intravenously. You need to receive a 1 to 2-h infusion (directly into a vein) every 2 weeks. You also need to consider your access to treatment when travelling as the infusion must be administered by, or under, the supervision of a healthcare professional. The drug must be stored in a refrigerator when not in use.

• Following the infusion there is a small chance you may experience an infusion-related reaction (discomfort, burning, swelling) and/or a reaction to the drug resulting dizziness or a rash

Oral treatment

• You have an inherited condition that may lead to you developing health problems. These problems could include tiredness, issues with your bones causing pain and becoming more likely to fracture, and enlargement of your liver and spleen which can result in serious complications.

• In order to try and prevent problems developing you are required to receive treatment for the rest of your life. The treatment is effective as long as it is taken according to instructions.

• The treatment you need to take is administered orally and can be taken with or without food. You need to take a capsule once to three times a day every day. The treatment does not require any special storage conditions. You also need to consider having your treatment with you when travelling.

• Following the treatment you may experience a minor side-effect such as temporary diarrhea