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Faster FDA approval pathway ‘exciting’

Bioscience executives are eager to see how a potentially major change in how regulators approve new drugs plays out.

The Food and Drug Administration is looking into ways to approve what it calls “breakthrough” therapies, which could be OK’d after only a single round of studies. Typically, products are tested in several phases of clinical trials that involve progressively more exacting criteria.

Such a new framework would require companies to work even more closely with FDA staff. The designation was established under the Food and Drug Administration Safety and Innovation Act that took effect July 9.

“It’s exciting that the FDA is looking at ways of getting pathways to drugs faster,” said Rachel King, CEO of GlycoMimetics in Gaithersburg.

Taryn Joswick, vice president of clinical development for Sucampo Pharmaceuticals in Bethesda, said she also is interested in this possibility — but it could all hinge on just what “breakthrough” means. Drug-makers need to understand just how the new designation differs from current labels applied to some products to expedite their pathway to patients, such as the FDA’s orphan drug designation for rare diseases and fast track development program for treatments for life-threatening medical conditions.

The law defines a “breakthrough” therapy as one that is intended, alone or in combination with one or more other drugs, to treat a serious or life-threatening disease or condition and for which preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, FDA spokeswoman Sandy Walsh wrote in an email to The Gazette.

She said the FDA had received 22 requests for breakthrough therapy designation as of Feb. 12, with five granted and 12 pending.

Quicker approval could reduce the development costs, said Ginette Serrero of A&G Pharmaceuticals in Columbia.

“The more you get the FDA involved early on and buying into the thought process of your drug, that’s going to be a good thing,” Serrero said.

King said the designation could hold a lot of promise for Maryland biotechs.

“Any way of collapsing policy into one or two rounds will be very advantageous,” said Judith Britz, executive director of the Maryland Biotechnology Center.

lrobbins@gazette.net

Psyadon Pharmaceuticals has spent nine months working to enroll the 18 patients it needs for its phase 3 clinical trial involving its treatment for Tourette syndrome.

The Germantown company’s candidate, ecopipam, also targets Lesch-Nyhan disease, a genetic disorder that affects as many as 1,000 Americans, disrupting their ability to walk and causing self-mutilation.

Although Psyadon usually is not directly involved in its clinical trials — it usually uses companies called contract research organizations, which conduct trials for drug makers — it often tracks disease-related patient advocacy groups and sometimes uses this information to raise awareness of the trial, said CEO Richard Chipkin.

Psyadon’s experiences may be different from those of other Maryland bioscience companies that focus on larger patient populations, but the challenge of finding subjects for clinical trials remains.

Almost half — 48 percent — of clinical trial sites miss their enrollment goals. The result is often longer study time lines and higher costs for companies, according to data from the Tufts Center for the Study of Drug Development. The report was based on more than 150 clinical studies at 16,000 sites in different countries.

‘The human factor’

Enrollment procedures and challenges differ from site to site, depending on the companies involved and the nature of the trial, said Judith Britz, executive director of the Maryland Biotechnology Center in Baltimore and an industry veteran. She said in cases of rare diseases, such as Lesch-Nyhan, the company performing the trial might need to use more sites than companies studying more common diseases.

“There’s always the human factor,” Britz said, adding that sometimes subjects need to understand that even if a particular treatment may not benefit them, it may help others in the future.

People managing trial sites might overestimate the number of patients or have patients on medication who cannot participate, said Taryn Joswick, vice president of clinical development for Sucampo Pharmaceuticals in Bethesda. She said some companies prepare for this by having more sites ready than they actually need.

Sucampo has been involved in clinical trials in various phases, with most focusing on its treatments for gastrointestinal disorders. The company has sold its constipation drug Amitiza for several years in the U.S. and just this month launched sales of its glaucoma treatment Rescula.

Joswick said patient enrollment also depends on whether a trial is targeting patients who are being treated by a physician for a disease such as cystic fibrosis — such patients could learn about trials from their provider — or self-treating patients, whom the company would need to reach through advertising and other ways.

Companies performing trials also need to decide whether they need to prepare a patient recruitment plan in advance or wait until they hit a roadblock, she said.

“It’s all about finding the right site with the direct access to the patient being sought,” Joswick said. “It becomes an art at some point.”

‘Creating a culture’

When it encounters enrollment problems through its contract research organizations, MedImmune, the biotech giant in Gaithersburg, reviews its trial requirements to ensure they’re not too difficult to satisfy, Ed Bradley, senior vice president of oncology innovative medicines, said in an email to The Gazette. The company also uses newsletters to keep involved medical professionals posted about trial progress.

Companies also can work with groups that recruit volunteers to suit different subject populations for trials, said Ginette Serrero, CEO of A&G Pharmaceuticals in Columbia. A&G focuses on breast cancer treatments and has a clinical trial that will follow up to 250 women during five years of their treatment.

Serrero recommended groups such as the Love/Avon Army of Women, a partnership between the Dr. Susan Love Research Foundation and the Avon Foundation. But she also cautioned that such groups come with price tags, so companies must weigh the benefit of using them versus the cost.

Regulatory demands have increased the size of clinical trials, said Salvatore Alesci, vice president of scientific affairs for Pharmaceutical Research and Manufacturers of America in Washington, D.C.

He said clinical trials often suffer from a lack of awareness and connections among non-major medical centers. Alesci’s trade group works on these problems through its Research in Your Backyard campaign, which speaks to the economic impact of clinical trials in each state, and participation in the National Clinical Trial Network, which builds databases among medical centers.

“We can’t do this by ourselves,” he said. “It’s about creating a culture.”

The University of Maryland, College Park, has its Center for Excellence in Regulatory Science and Innovation, funded by the Food and Drug Administration, to help develop curricula for training advanced methods on running clinical trials and serving as a neutral party to help sort out issues where sites only need to tweak aspects of a trial, rather than go back to the start, Britz said.

Social media as an avenue

One of the reasons for low enrollment could be a reliance on conventional recruitment methods instead of adopting modern techniques such as social media, the Tufts Center report suggested.

Britz described social media as a creative solution.

Joswick said social media has potential in aiding recruitment, but companies are still unclear on whether it returns its costs.

“I haven’t seen many great success stories,” she said.

While Rachel King, CEO of GlycoMimetics in Gaithersburg, sees the potential for social media, she said companies also must be wary of FDA regulations that control what can be said about a product. GlycoMimetics is developing a treatment for vaso-occlusive crisis in sickle cell disease.

Psyadon uses the same ethical committee that reviews its clinical trials when reviewing its trial advertisements, Chipkin said.

There are no regulations specific to the use of social media for trial recruitment, but the FDA requires that an institutional board review and have the authority to approve all research activities, including direct advertising for study subjects, FDA spokeswoman Michelle Bolek wrote in an email to The Gazette.

“FDA expects [review boards] to review the advertising to assure that it is not unduly coercive and does not promise a certainty of a cure beyond what is outlined in the consent and protocol,” she said.

While MedImmune does not use social media in recruiting, it monitors patient advocacy groups or specific disease interest groups that sometimes post information about its trials on their websites, Bradley said.

lrobbins@gazette.net