Principles and Practice of Pharmaceutical Medicine

Patients and families

The motivation of those with the disease or those
with relatives with the disease is clear – they want
better treatments that are affordable and will
improve the quality of life for the patient.

Physicians and other healthcare
providers

The motivation of these people is also clear – they
seek to find better treatments for their patients
and are often willing to test new drugs in clinical
trials.

Academicians

Orphan drugs offer research opportunities for
scientists and clinicians. Another important moti-
vating factor are the opportunities presented for
career enhancement.

21.6 Specific sources

of information

on orphan drugs

The Food and Drug Administration (FDA) pub-
lishes an annual list of orphan drugs that have been
approved for marketing since the signing of the
Orphan Drug Act in 1983. They also publish a
monthly list of drugs that have received orphan
drug designation within the last month and an
annual cumulative summary of the current desig-
nations. There is also a home page on the Internet
and an annual publication of grants to evaluate
orphan drugs in clinical trials.
Most specific disease organizations, as well as
umbrella disease organizations, provide infor-
mation of relevant diseases for members and
sometimes for researchers, and the public.
These groups may also provide current scientific
information. For example, the NORD database
is a valuable source of information for many

groups of people interested in a particular

disease.

21.7 Discovery, development,

marketing and distribution

of orphan drugs

The process ofdiscoveringnew orphan drugs is not

different from that used to discover drugs for more

common diseases (Spilker, 1994). This is a broad

topic and will not be discussed further.

As with discovery, the methods used todevelop

drugs for rare diseases do not differ from the meth-

ods used to develop drugs for more common dis-

eases in terms of strategies created, methodologies

used and criteria for success (see Chapter 2.2).

However, certain differences in quantity of data

exist. If there are only 500 patients with a specific

disease, it is probably impossible to have two ran-

domized, well-controlled placebo trials. Nonethe-

less, the quality and amount of manufacturing data

required for regulatory submissions do not differ

for orphan drugs than for more common drugs.

The same marketing tools are available tomar-

ketorphan drugs and non-orphan drugs. Probably

the greatest difference between orphan and non-

orphan drugs from a marketing perspective is that

the amount of money spent on orphan drugs will be

significantly less than that for non-orphans.

Although the same mix of marketing tools can be

and are often used, the number of symposia to be

held and the number and size of advertisements

will usually be fewer. Another exception is that the

use of sales representatives to promote a new

orphan drug may be nonexistent. However, a

large company may in fact be developing the

orphan drug so that the sales representatives can

discuss the orphan drug (and non-orphans too),

whereas a small company may have decided to

develop an orphan because sales representatives

are not necessary to promote the product.

Distributionmethods differ more between orphans

and non-orphans more than the other categories

discussed in this section. Conventional drugs are

generally sold through wholesalers, as well as

21.7 DISCOVERY, DEVELOPMENT, MARKETING AND DISTRIBUTION OF ORPHAN DRUGS 269