Orphan Diseases(关注罕见病)

Essay

Name: Shen Hao

Student #: 14S153035

Tue 10:30

Orphan Diseases

Orphan

diseases,diseases

that

affect

a

small

percentage

of

the

population, have aroused great public concern worldwide in the past three

months.

With

the

growing

popularity

of

the

Ice

Bucket

Challenge,an

activity held by an American charity organization three months ago which

aims

to

raise

money

and

awareness

for

ALS,

people

from

all

over

the

world

begins

to

pay

attention

to

a

special

group

of

people

——

people

with

orphan

diseases.

Despite

the

significant

breakthroughs

on

medical

science we have made in the past few years, the cure of orphan diseases

has

not

made

any

substantial

progresses.

The

basic

causes

of

the

slow

progress

in

treating

orphan

diseases

can

be

traced

to

three

factors:

the

difficulties of scientific research into orphan diseases,the lack of available

effective drugs, and the lack of knowledge of orphan diseases.

The first cause of the slow development of treating orphan diseases

is

the

difficulties

of

scientific

research

into

orphan

diseases.

First

of

all,the finite research resources and cases make it difficult for scientists to

develop

relative

research.

Generally,

the

rate

of

people

with

orphan

diseases

among

the

whole

population

is

so

low

that

scientists

may

be

hard-pressed

to

get

enough

cases

to

carry

out

their

research.

Moreover,

patients with the same kind of orphan diseases are dispersed throughout

the

country,

which

adds

to

the

difficulties.

Second,

science

projects

on

orphan

diseases

are

inadequate.

There

are

no

effective

laws

and

regulations

that

encourage

scientific

research

into

orphan

diseases;

furthermore,scientists have not paid enough attention to orphan diseases

compared

with

other

common

diseases

due

to

the

small

number

of

patients.

As

a

result,only

a

small

group

of

scientists

and

research

institutions are engaged in research into orphan diseases. Indeed,these are

only

two

of

the

many

difficulties

we

meet

when

carrying

out

scientific

research into orphan diseases.

Another

cause

is

the

lack

of

available

effective

drugs.

There

are

two reasons for this. First of all,most drug companies have no inclination

to develop new drugs treating orphan diseases due to the expensive cost

of

drug

research,

the

high

risk

of

failure,

and

the

weak

demand

in

the

orphan drugs market. Second, there are few relevant laws and regulations

which

protect

the

economic

benefits

of

orphan

drug

companies,

which

leads to the dependence on imported orphan drugs.

The

final

cause

that

puts

sand

in

the

wheels

of

the

treatment

of

orphan

diseases

is

the

lack

of

knowledge

of

orphan

diseases.

First,

popularization of orphan disease knowledge is far from satisfying. Since

most

medical

professionals

lack

experience

in

orphan

diseases,misdiagnosis of orphan diseases is inevitable, which would delay