Public Policy
At Alexion, we are committed to working with private healthcare organizations, policy makers, and public health authorities to provide patients with rare diseases the access to innovative therapies that they deserve.
We demonstrate our commitment to breakthrough innovation through our first product, Soliris®
(eculizumab). Soliris has received two of the industry’s highest honors: the 2008
Prix Galien USA Award for Best Biotechnology Agent and the 2009 Prix Galien France
Award in the category of medicines for ultra-rare diseases.
We are focused on our objective that every patient who can benefit from Soliris should have access to Soliris. Alexion offers and supports numerous programs designed to achieve this objective, including:
The OneSource™ Treatment Support Program. Patients with PNH who enroll in this program are assigned an Alexion Nurse Case Manager who coordinates care between the physician's office and the insurer and assists with searches for alternative payment or funding options.
The PNH Fund. This fund, established by the National Organization for Rare Disorders (NORD) with support from Alexion, helps cover treatment-related costs for underinsured patients with PNH.
The COMPLEMENT Foundation. This foundation was established by Alexion to help patients with PNH who do not have insurance, access to insurance, or any other means for obtaining Soliris.
Early Access Program. Alexion, together with the FDA, established this program to help patients with PNH obtain access to Soliris in the United States prior to its approval by the FDA in March 2007.
Learn more about our Access programs.
Supporting Innovation and Access
Healthcare authorities in many countries have recognized the need to encourage therapeutic
innovation for patients with rare and ultra-rare diseases – and, equally important, to support patient
access to innovative drug therapies once they are available.
For example, the U.S. Orphan Drug Act (ODA) provides important incentives, including
seven years of market exclusivity, to companies that succeed in bringing a treatment
for a rare disease to market. The Regulation on Orphan Medicinal Products (ROMP),
enacted by the European Parliament in 1999, creates similar incentives in the EU.
These vital pieces of legislation have led to the development of more than 300 safe
and effective therapies for patients with rare disorders, including Soliris. Similar
incentives exist in other countries, including Japan, where orphan drug designation
provides for accelerated review of New Drug Applications and 10 years of market
exclusivity upon approval.
The success of the ODA and similar legislation derives from the recognition that,
despite the small number of treatable patients, orphan drug development entails
levels of investment and scientific innovation – and risk of failure – equal to or greater than that associated with developing drugs for diseases that affect millions. In any one country,
and moreso on a global basis, the job of helping patients who suffer from rare disorders
has just begun.
Factors in Orphan Drug Innovation
Biopharmaceutical companies face unique challenges as they seek to develop innovative,
safe and effective therapies for patients with rare and ultra-rare diseases. These include
the need to identify sufficient numbers of patients to participate in drug trials,
and the necessity of including a large number of study sites, in multiple countries,
since each may be able to enroll just one or two patients. In addition, regulatory
risk may be markedly increased for development of orphan drugs since there is usually
no approved therapy for these diseases and no well-established roadmap to marketing
approval.
The relatively small number of products that eventually receive approval necessarily require the burden of the failed development to be incorporated into the costs of
the few successfully developed products. Also, most orphan drugs are complex biologics
(such as proteins) that require living cells for production, rather than chemical
drugs whose production is typically simpler and less expensive.
Once an orphan drug is approved, the innovator typically engages in extensive post-marketing
research, physician education, and patient support, as well as safety monitoring,
which may also be more expensive for orphan drugs, especially since orphan drug
companies are often required to establish expensive lifetime safety patient registries
to further monitor patients with these rare disorders and other mechanisms to assess and mitigate
risk.
These and other factors can combine to impact the costs, and thus the pricing, of
an orphan drug, which in some cases will be used to treat only a few hundred patients around the world.