FDA lays guidelines for “biosimilar” biotech drugs

By Dr. Ananya Mandal, MDFeb 12 2012

The Food and Drug Administration (FDA) has come up with guidelines for the sale of lower-cost versions of biotechnology drugs.

The proposed rules, issued on Thursday, require studies showing that the generic copies are “highly similar” to the originals, but there are several ways that might be proven. Because of their complexity, generic copies of biotech drugs - first introduced in the 1980s - are known as “biosimilars.” The worldwide market for copies of biotech medicines will grow to $3.7 billion by 2015, from just $243 million in 2010, as more than 30 branded biologics with sales of $51 billion lose patent exclusivity, according to market analysis firm Datamonitor.

Since their introduction in the 1980s, biotech drugs have never faced generic competition because the FDA did not have power to approve copies of such medications. For years the biotech industry successfully argued that their drugs, often made from living cells, were too complex to be duplicated by competitors. That finally changed with the Obama administration's 2010 health overhaul, which ordered the FDA to create a system for approving so-called “biosimilar drugs.” The industry term arose because biotech scientists insisted it would be impossible to produce exact copies of their biologically engineered drugs. They differ from traditional drugs, which are made by combining various chemicals.

“These draft documents are designed to help industry develop biosimilar versions of currently approved biological products, which can enhance competition and may lead to better patient access and lower cost to consumers,” FDA's drug division director Dr. Janet Woodcock said in a statement.

“We're trying to send the signal that it's not one-size-fits-all. It's product-by-product,” Rachel Sherman, director of the FDA's office of medical policy, said during a conference call with reporters. The FDA rules would set “an abbreviated pathway” to approval that would consider factors including a product's complexity, formulation and stability, the agency said.

“We do not want companies repeating studies that do not need to be done — that wastes precious resources, and of course, exposing humans and animals to unnecessary testing is unethical” said Sherman. She said the FDA has not yet received any applications for biosimilars drugs, though companies have submitted three dozen requests for meetings on potential products.

While no companies have filed for FDA approval yet of a biosimilar, the agency has received nine applications to conduct human clinical trials on the drugs, Sherman said. The agency also has had 21 meetings with companies seeking to develop biosimilars. “This is a completely new paradigm,” Sherman said. “I don’t know when we’re going to receive our first application. We’ve been open for business since March 2010.” The FDA will aim to decide on each application within 10 months -- as it does for most new drugs.

The FDA said it would decide on the “extent and scope of animal and clinical studies” needed for approval once it has considered other analytical data. Manufacturers will have the option of asking the FDA to deem their copies “interchangeable” with a brand-name drug, but the `agency said that would require additional clinical studies.

Makers of branded biotech drugs have argued that full-scale human trials need to be conducted before a rival version of an existing biologic drug should be allowed on the market. “While the documents provide a roadmap, they are sufficiently vague as to give FDA leeway for case by case assessments of each proposed biosimilar along their respective development paths,” said Wells Fargo analyst Brian Abrahams.

The Congressional Budget Office has estimated that the United States could save $25 billion from the use of biosimilars over 10 years. European regulators have already approved cheaper versions of some biotech drugs. Biosimilar drugs are expected to sell at discounts of 25 to 45 percent to branded rivals, compared with generic versions of traditional pills that often sell for one-tenth the price of the branded product.

The agency is accepting public comment on the draft guidance documents for the next 60 days.