Pharmaceutical Commerce - September/October 2017 - 17

Therapy Focus
market adoption of the treatment.
In lieu of a randomized controlled trial,
orphan drugs can also be approved based
on data collected through compassionate
use programs due to a clear unmet need and
the resulting generation of sufficient data
showing safety and efficacy of the drugs
in the patient populations. For example,
Cholbam for bile acid synthesis disorders
and Zellweger spectrum disorder, and
Myalept for lipodystrophy each had more
than 10 years of data in patients before Food
and Drug Administration (FDA) approval.

to evaluate long-term benefits can be
hard to definitively measure. Successful
commercialization of an orphan drug,
therefore, requires several components,
including demonstration of value during
the course of clinical development,
thoughtful trial design, and understanding
the natural history and long-term outcomes
of the disease.

Continued commitment
Patients with rare diseases are a largely
underserved population with a huge unmet
need in terms of awareness, diagnosis,
and available therapies and medical
management. Just as science has adapted
and introduced new techniques to address
the unique challenges of rare disease drug
development, all the stakeholders-policy
makers, payors, patients and pharmaceutical

companies-have to adapt and engage
with one another for continued success in
developing new drugs and ensuring market
access. Ultimately, our commitment to
rare disease research and tackling the large
unmet need in this small population is a
reflection of our values as a society. Much
progress has been made, but much more
needs to be done.

Trends in drug approval
The regulator y landscape for rare
disease development is evolving as more
drugs enter clinical trials. Currently, the
regulatory environment is favorable with
both FDA and the European Medicines
Agency (EMA), as they offer incentives
for the development of orphan drugs. As
such, the number of rare disease therapies
approved has increased exponentially in the
last two decades.
In 2015, FDA updated guidance for
rare disease drug development to help
industry sponsors create successful drug
development programs that address the
par ticular challenges posed by each
disease. The guidance recommends that
pharmaceutical companies invest in
understanding disease pathophysiology
and progression-initial symptoms,
diagnosis, surrogate markers and clinical
outcomes. This will help in designing more
efficient clinical development programs,
as well as influencing decisions regarding
reimbursement and formulary placement.
Happily ever after?
The challenges in the rare disease market
extend beyond a successful clinical trial
and approval for use. In cases of treatments
where the endpoint is based on surrogate
markers or other non-outcomes-based
measures, establishing the value of the drug
can be difficult. Many agencies have specific
decisionmaking thresholds based on criteria
of cost-effectiveness and evidence quality.
For example, the UK's National Institute
for Health and Clinical Excellence considers
patients' quality of life and the length of life
they will gain as a result of an intervention.
The health benefits are expressed as
quality-adjusted life years (QALYs). Health
technologies with an incremental ratio
of less than £20,000 ($25,600) per QALY
gained are considered cost effective and are
highly likely to be reimbursed; those with a
ratio in excess of £30,000 ($38,500) would
require other conditions to be met in order
for them to be funded.
O r p h a n d r u g s r a re l y m e e t t h e s e
criteria, so they pose a unique problem for
reimbursement decisionmaking. There is
currently a lack of specific protocols to assess
reimbursement-related determinations
for these therapies. The epidemiology and
natural history of rare diseases are not well
understood, so extrapolating from surrogate
markers or other clinical trial endpoints
September | October 2017 Visit our website at www.PharmaceuticalCommerce.com 17


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Pharmaceutical Commerce - September/October 2017 - Table of Contents
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