Industry Concerns Over Six-Month Deadline Australia Insists Orphan Designations Will Last Only Six Months, Overrides Industry Objections→

Sponsors making drugs for rare diseases in Australia who fail to file for approval within six months of being granted orphan designation will see their designation disappear, the Therapeutic Goods Administration has decided. The new procedure will be problematic for orphan drug sponsors and might even make it more difficult for them to use the new expedited pathways the agency... 

A public consultation on a proposed version of the reforms in 2016 had raised concerns among drug makers who were worried about designations lapsing too soon; previously, orphan designations lasted indefinitely. The TGA’s original proposal was for designations to lapse automatically after three to six months, but in April this year it settled on a six-month deadline after considering the feedback to the consultation.

According to the responses to the consultation, drug sponsors generally agreed with the concept of orphan drug designations lapsing after a set period of time, but most of them said three to six months would be too short. Industry body Medicines Australia said: “We suggest instead that the period before which designations lapse be 12 months at a minimum.”

From the editors of Scrip Regulatory Affairs.

Drugmakers Manipulate Orphan Drug Rules To Create Prized Monopolies→

More than 30 years ago, Congress overwhelmingly passed a landmark health bill aimed at motivating pharmaceutical companies to develop new drugs for people whose rare diseases had been ignored.

By the drugmakers’ calculations, the markets for such diseases weren’t big enough to bother with.

But lucrative financial incentives created by the Orphan Drug Act signed into law by President Ronald Reagan in 1983 succeeded far beyond anyone’s expectations. More than 200 companies have brought almost 450 “orphan drugs” to market since the law took effect.

Yet a Kaiser Health News investigation shows that the system intended to help desperate patients is being manipulated by drugmakers to maximize profits and to protect niche markets for medicines already being taken by millions. The companies aren’t breaking the law but they are using the Orphan Drug Act to their advantage in ways that its architects say they didn’t foresee or intend. Today, many orphan medicines, originally developed to treat diseases affecting fewer than 200,000 people, come with astronomical price tags.

And many drugs that now have orphan status aren’t entirely new. More than 70 were drugs first approved by the Food and Drug Administration for mass market use. These medicines, some with familiar brand names, were later approved as orphans. In each case, their manufacturers received millions of dollars in government incentives plus seven years of exclusive rights to treat that rare disease, or a monopoly.

Drugmakers of popular mass market drugs later sought and received orphan status for the cholesterol blockbuster Crestor, Abilify for psychiatric conditions, cancer drug Herceptin, and rheumatoid arthritis drug Humira, the best-selling medicine in the world.

More than 80 other orphans won FDA approval for more than one rare disease, and in some cases, multiple rare diseases. For each additional approval, the drugmaker qualified for a fresh batch of incentives. Botox, stocked in most dermatologists’ offices, started out as a drug to treat painful muscle spasms of the eye and now has three orphan drug approvals. It’s also approved as a mass market drug to treat a variety of ailments, including chronic migraines and wrinkles.

Orphan Drug Utilization and Pricing Patterns (2012 - 2014)→

Orphan drugs- developed to treat rare diseases affecting patient populations of fewer than 200,000 people annually - have become a lucrative business opportunity for drug makers. These medications made up almost half of all new drugs approved by the U.S. Food and Drug Administration (FDA) in 2015. 

Read here.

Non-Profits At Odds On How To Address Third-Party Payment Concerns

Two non-profit charities that offer premium payments to help low-income patients afford insurance are at odds over how CMS should mitigate concerns that such programs are being used to steer patients away from Medicare/Medicaid and into private coverage with higher reimbursements. Patient Services, Inc. (PSI) and American Kidney Fund (AKF) both proposed solutions that would require plans to accept payments from nonprofits to adhere to certain criteria to avoid potentially fraudulent behaviors that concern CMS. The agency issued last month.

Read more.