Law Seeks to Combat Drug Shortages, Bolster Rare Disease Efforts

Patients with rare diseases may have expanded access to medications – and more affordable ones, at that – under a new piece of legislation signed into law this week by President Barack Obama.

The legislation, which passed both the House and Senate with bipartisan majorities, is aimed at speeding up the process of getting safe and effective medical products to patients.

The passage of the law comes at a crucial time, as the number of drug shortages in the United States hit an all-time high in 2011, posing a risk to patients who rely on life-saving drugs for medical procedures and treatment.

The Food and Drug Administration Safety and Innovation Act, or S. 3187, enhances the tools available to the FDA to combat drug shortages by requiring manufacturers of certain drugs to notify the FDA when they experience circumstances that could lead to a potential drug shortage. This provision is meant to complement the actions directed by the 2011 Executive Order to address this worrisome public health issue.

S. 3187 will provide the FDA with the tools needed to continue to bring drugs and devices to market safely and quickly as well as promote innovation in the biomedical industry.

Specifically, the law allows the FDA to provide vouchers to drug manufacturers so they can obtain quicker approval for profit-making medications in return for developing drugs in smaller, less profitable markets, like rare childhood diseases.

Under the new law, expedited FDA review can cut the approval process from an average of 10 months to six months for rare conditions that primarily affect individuals from birth to 18 years of age.

The law also gives the FDA the power to accelerate the approval of a drug for a serious or life-threatening disease or condition after determining whether the drug is reasonably likely to have a clinical benefit, taking into account the severity, rarity or prevalence of the condition and the availability or lack of alternative treatments.

In addition, the law mandates that the FDA develop a list of external experts who are qualified to provide advice on rare diseases if the agency needs consultation on these types of diseases and conditions for drug development purposes.

The new law will also implement a program proposed in the president’s 2013 budget to accelerate approval of lower-cost generic drugs as well as fund the new approval pathway for biosimilar biologics – products that are marketed after expiration of patents and have similar properties to existing products – created by the Patient Protection and Affordable Care Act. These new programs are important to increasing patient access to affordable medicines, according the U.S. Department of Health and Human Services.