Phillip Ward is the European editor of Applied Clinical Trails.

Phillip Ward

	The temptation to use uncontrolled, early, small studies to support further development of products may prove problematic for emerging biopharmaceutical companies, and these firms require both innovative approaches and rigor for success, according to an article posted today (February 2nd) by the 

	“Development of drugs for rare diseases may be used as a strategy, but the small size of the populations with such diseases and the small samples available for trials require approaches that can maximize the power to detect efficacy, which can include the use of historical controls, new surrogate end points, or enrichment for participants who are likely to have a response,” noted lead author Richard A. Moscicki, M.D., Deputy Director for Science Operations at the Center for Drug Evaluation and Research, Food and Drug Administration, Silver Spring, Maryland (

 2017; 376:469-474).
	
	Small biopharmaceutical companies range in size from virtual companies with no commercial products and no revenue to those with only a few commercial programs, and they are becoming increasingly important as drivers of innovation in drug development, he explained. These firms often encounter important challenges in designing and implementing clinical development programs.
	
	Small companies use a variety of approaches to address these challenges, including the use of new technical platforms, the use of new formulations or technologies that enhance the actions of known drugs, and the use of trial designs that take advantage of the specific market they hope to enter. Other businesses develop products that are spun off from or licensed from large companies, according to Moscicki.
	
	“Many small companies also repurpose drugs or pursue narrow niche markets - such as rare inherited diseases, uncommon cancers, or specific infectious diseases - to remain viable,” he wrote. “Furthermore, many companies turn to rare diseases for an opportunity to successfully negotiate many of the aforementioned issues, though such diseases present their own challenges - in particular, the small number of patients available for clinical trials.”
	
	The article focuses on strategies used by these companies, with an emphasis on drugs developed for rare diseases. For further details, 

Articles

The temptation to use uncontrolled, early, small studies to support further development of products may prove problematic for emerging biopharma companies.

Small Biopharm Firms Struggle to Meet Development Challenges

	Synexus has announced plans to open three new research centers in Bulgaria, Poland and Romania, bringing the company’s total to nine centers in Central & Eastern Europe and a total network of 25 across Europe and Africa.
	
	The population of Central and Eastern Europe exceeds that of either the US or the five largest Western European markets combined, and it also has excellent healthcare professionals, according to the U.K.-headquartered recruitment specialist.
	
	“This will help us to provide much greater support to the global pharmaceutical industry,” noted CEO Christophe Berthoux. “Since we opened our first centre in Poland in 2006 we have had an ongoing programme of investment in the region.”
	
	The new Polish facility is the company’s largest research center in the world. It occupies 1000 m

 at Beniowskiego Street in Gdansk. Magdalena Przekwas-Jaruchowska, M.D., the facility’s director, oversaw the opening ceremony, which included representatives from the local authority, the British Embassy, the medical university and patients’ associations.
	
	Romania’s new 820 m

 center is based in Bucharest, which has a population of two million. The country has been part of Synexus’ development plans since early 2000, as the total population is over 20 million and the number of new clinical trials granted approval during the last five years is at least 200, according to the company.
	
	In Sofia, Bulgaria, the total area of the new center is 650 m

, and it has eight medical rooms, two administration rooms, monitoring and meeting rooms and other areas. This facility aims to offer its patients access to new drugs in the fields of pulmonology, rheumatology, neurology, endocrinology, cardiology, gastroenterology and dermatology.
	
	 

Synexus has announced plans to open three new research centers in Bulgaria, Poland and Romania.

Synexus Pushes Ahead with Central & Eastern Europe Expansion

	Significant differences exist in the characteristics of the priority review drugs approved by the FDA and the European Medicines Agency (EMA), but more harmonization of the U.S. and European regulatory frameworks may facilitate timely approval of pharmaceutical products, according to new research published online on May 27 by the journal 

	The FDA priority review process applies to a drug that is considered to be a significant improvement over the available alternatives, while the EMA’s accelerated approval applies to a product that is of major public health interest, explains lead author Saad Alqahtani and colleagues.

	Their study assessed differences in the characteristics of priority review new molecular entities and new therapeutic biologic products approved by the FDA and EMA, including regulatory information on drug applications, approvals, indications, and orphan designations of all priority review drugs approved by the FDA and the EMA in the period 1999-2011.

	Overall, 100 FDA priority review new molecular entities and new therapeutic biologics were approved by both agencies, and 87% of the products were first approved by the FDA. The average FDA review time (9.2 ± 8.4 months) was significantly lower than the EMA average review time (14.6 ± 4 months) (p < 0.0001). The FDA and the EMA granted orphan designation to 43% and 33%, respectively, of the applications. There were differences in the administration route (1% of all products), dosage (8%), strength (23%), posology (51%), indications (30%), restrictions of use (52%), limitations of use (19.0%), and outcomes limitations (28.0%) approved by both regulatory agencies.

Significant differences exist in the characteristics of the priority review drugs approved by the FDA and the European Medicines Agency.

Phillip Ward

Articles

Small Biopharm Firms Struggle to Meet Development Challenges

Synexus Pushes Ahead with Central & Eastern Europe Expansion

Harmonization Can Improve Priority Reviews, Finds New Study