Table 2 Results of voting on ‘selecting 10 criteria considered to be particularly important’

Research topics related to various QOL aspects such as psychological and lifestyle aspects

18

11

5

2

Research topics related to life and death

17

9

6

2

Research topics related to expected findings meant to alleviate patients’ pain and burden and lead them to gain ‘independence’

16

11

4

1

Research topics that cannot be solved without involving researchers or policymakers

13

9

4

0

Research topics that patients readily experience as issues and would directly feel the benefits of, if properly addressed

13

7

5

1

Research topics related to physical function

11

6

3

2

Research topics that directly affect the diseases or the patients themselves

11

8

3

0

Research topics that impact society as a whole (social systems or individuals’ behaviours)

11

4

6

1

Research topics on the difficulties that cause other problems, and their resolution which is expected to simultaneously solve many other problems

10

4

5

1

Research topics related to children

9

6

2

1

Research topics related to many rare diseases

8

4

3

1

Research topics specific to rare diseases

8

4

3

1

Research topics whose findings could have a ripple effect beyond rare intractable diseases

8

4

2

2

Research topics that have big disadvantages before and big advantages after finding a solution

8

5

2

1

Research topics related to expected research findings that lead to reducing the burden imposed on families and healthcare providers

8

6

1

1

Topics of research in which resources and knowledge are already available, and their application remains a bottleneck

8

5

3

0

Research topics with few studies addressing the issue

8

4

4

0

Research topics where Internet use is expected to be an effective problem-solving tool

7

4

2

1

Research topics that affect the surrounding environment, such as families and healthcare providers

6

5

1

0

Research topics expected to have a high cost-effectiveness

5

1

2

2

Research topics on problems that cannot be resolved even though patients have been making their own efforts

5

4

1

0

Topics of research that can be carried out with limited time and resources

4

1

3

0

Topics on which research has been set aside or delayed because the number of the patients with the rare disease is so small, or the diseases were not life-threatening

N/A

N/A

N/A

N/A

Research topics that require legal knowledge and that aim to bridge gaps between real life and related laws

N/A

N/A

N/A

N/A

Research topics that aim to develop new applications and methods for newly discovered or invented technologies (e.g. iPS cells)

N/A

N/A

N/A

N/A