A Helping Hand For Emerging Pharma

THE USUAL REACTION to the line, "I'm from the government, and I'm here to help" is to run, fast, in the other direction.

But small- and medium-sized drug and biopharmaceutical companies in Europe are welcoming that offer from the European Medicines Agency (EMEA), the European Union's equivalent to the U.S. Food & Drug Administration. The offer comes specifically from its Small- & Medium-Sized Enterprises (SME) Office, a unit dedicated to meeting the needs of small companies.

To date, some 223 companies have applied for SME status with EMEA, and 158 have received it. This designation entitles them to a significant reduction in fees, deferral of payments, help with language translation, and administrative assistance in filing for approvals for their new drugs.

"This service has been very important for emerging biotech companies who are struggling to bring their medicines to market," says Aisling Burnand, chairman of the National Associations Council within the European Association for Bioindustries. The group, known as EuropaBio, represents 1,800 of an estimated 2,000 biotech companies in Europe.

The willingness to help testifies to the commitment of the European Commission, the EU's administrative arm, and EMEA to meet the needs of SMEs, Burnand adds.

Those are encouraging words for the staff at EMEA's SME Office, located at the agency's London headquarters. The office, part of EMEA's Preauthorization Human Medicines unit within the Sector for Scientific Advice & Orphan Drugs, is staffed by Melanie Carr and Constantinos Ziogas.

As Carr notes, small drug companies "often struggle in understanding and accessing the appropriate advice. We want to help them through the regulatory hurdles." However, she emphasizes, EMEA applies the same standards to a product being registered, no matter what size the producer. "That burden is not lower" for small companies, Carr says.

The beginnings of the office go back to a March 2004 review of EMEA's main pharmaceutical regulations. A new provision was added, Carr says, "that there should be some special help for SMEs." That provision required the commission to draft an implementing regulation that was passed in December 2005.

To qualify for SME designation, a company has to meet size, sales, and balance- sheet criteria. According to the EU, an SME can have no more than 250 employees. Annual sales can be no more than 50 million euros (roughly $65 million), and the annual balance sheet can total up to only 43 million euros.

Some of the companies are start-ups from university research, some are spin-offs from big pharma, and some are orphan-drug makers.

Some EMEA incentives are designed to help companies overcome bureaucratic hurdles. For example, the office gives help in handling procedural issues, in devising a regulatory strategy, and in providing scientific advice on drug development.

Some of the incentives pamper the wallet as well. For example, SMEs receive reductions of up to 90% in fees for scientific advice on data requirements for pharmaceutical nonclinical safety and clinical efficacy and safety, Ziogas points out.

THINGS BECOME more complicated, though, when a company is ready to prepare a marketing application. According to Carr, a scientific committee within EMEA reviews all applications from companies big and small. An approval leads to a marketing authorization valid throughout the 27 countries of the EU and in Iceland and Norway, which recognize the license.

SMEs receive no fee reduction when applying for a marketing authorization, which can run about 232,000 euros, Carr notes. However, the payment can be deferred until an authorization is granted. "That is usually when a company starts getting a revenue stream," she points out. Moreover, if an application is turned down after the company had previously sought scientific advice and taken it into account during development, the authorization fee is waived.

The SME Office also helps with the very European problem of translation. As Carr points out, the initial registration information is in English, but that information must then be translated into all the EU languages. That means 22 languages currently, and Ireland is campaigning to have Gaelic recognized as yet another. "SMEs find this an enormous burden," she says. Accordingly, the office will provide translation for the initial authorization.

The SME Office is preparing a new user guide to help companies understand EMEA regulations, Carr and Ziogas say. The first draft was posted on EMEA's website for consultation in December, and the deadline for comments was the end of March.

When the SME Office was established, Carr recalls, "we didn't know how many companies would be applying. We have been overwhelmed by the numbers registering. We are very pleased with the response the first year."

According to Ziogas, the companies hail from 17 countries. About 25% of them are "micro," with sales of less than 2 million euros per year and fewer than 10 employees. "This is quite encouraging-these are the ones we're especially eager to work with," he says. And some companies have drug candidates already in Phase II or Phase III trials, he adds, with several products getting ready for market.

After just one year, Carr concedes, "it is way too early to say what the impact of our SME Office will be." However, she points to some good news: "We've had 25 companies go through the scientific advice process, and eight have submitted marketing applications."