Of the

nearly 7,000
Many Medicaid programs make coverage rare
decisions for therapies without input from diseases,
only 5%
either the public or experts. These have
processes often result in inappropriate treatments
restrictions for vulnerable patients or
even a denial of needed medications.

Processes vary by state, but recommendations are generally made by Pharmacy

& Therapeutics (P&T) Committees or Drug Utilization Review (DUR) Boards.
These Committees and Boards often do not provide an opportunity for the public
or clinical experts to provide input. Many also lack clearly defined processes for
coverage decisions, which can lead to policies that are inconsistent with real-world
clinical practice and harmful to patients.

Average wait before

30 Million a proper diagnosis
Proper diagnosis and treatment of rare
diseases is often time-consuming and
Americans suffer 7 Years difficult. Treatment regimens are often
from a rare disease complex and require supervision by
specially-trained, expert physicians.

Following the example set by several states, based on a similar program at the FDA
(EXPERRT Act), and endorsed by the Consumer Representatives to the National
Association of Insurance Commissioners (NAIC), state Medicaid agencies should
incorporate experts into the coverage process:

Input from Public

Coverage Policy Draft Policy Testimony/ DUR Review
Clinical
Initiated Made Public Notice & & Final Vote
Community
Comment

Clinical Expert Input

Sources: Medicines in Development for Rare Diseases. PhRMA. Published 2016 | Rare Disease Impact Report. Shire. Published April 2013
 FDA Approval

Many states have a complete Policies should be made

lack of information as to how public & the process should
the process works be open
Coverage Process

Outside experts are rarely, if Outside specialists should be

ever, called upon to offer called upon to provide
input into complex therapies expertise in the therapy area

P&T/DUR Review

There is no draft policy or the A draft policy should be made

policy is not made public available to the public & allow
for adequate time to comment

Draft Policy

Stakeholder input is minimal Physicians, patients & caregivers

or non-existent, leaving should be given adequate
patients out of the process opportunity to comment
Public Comment

The final policy has restrictions The final policy should be

inconsistent with clinical consistent with the FDA label &
guidelines & denies access real world clinical practice

P&T/DUR Vote

Coverage Initiated