NEWS & EVENTS
WFH: Enhancing Affordability and Global Supply
May 24 2011, Tue
Taken from the World Federation of Hemophilia
Biosimilars, also known as follow-on biologics, are widely touted as a key ingredient to improving affordable access to biologic products, such as clotting factor concentrates. Historically, generic versions of chemical drugs, identical in safety, purity and effectiveness, have improved affordability compared to their brand-name drug counterparts. Manufacturers of generic drugs can market these products once the period of patent protection for the brand-name drug ends. A hot public policy topic is whether and how best comparable results can be achieved for biologics derived from living organisms as has been achieved through generic versions of chemical drugs.
In an era of government budget deficits and escalating healthcare costs, lower prices for biologics could greatly benefit both patients and those who are responsible for paying for them. With 75 per cent of the worlds population having limited or no access to treatment, pursuing strategies to improve access is an important objective.
The cost savings resulting from the use of generic drugs is due to many factors, including the utilization of streamlined product development and regulatory requirements, such as reducing or eliminating requirements for clinical trials. Whether a streamlined regulatory approval process is appropriate for complex biologics is a central issue facing regulators today. While we would all welcome the arrival of new lower cost treatment, we should not be willing to accept an expedited regulatory approval process that places patient health and safety at risk.
Today, the risk of inhibitor development has replaced pathogen risk as the most significant adverse event facing patients with severe hemophilia A.
Perhaps one answer to the debate is in the name used to describe the new product itself. The term biosimilars is used because the final product is fundamentally similar, but not identical, to the original. The complexity of large-molecule biological therapies and the intricacies of the manufacturing process make it impossible to exactly replicate a product. Unfortunately, even minute changes could have dramatic consequences for patients.
Today, the risk of inhibitor development has replaced pathogen risk as the most significant adverse event facing patients with severe hemophilia A. Quality of life for someone with an inhibitor is greatly reduced; treatment involves significantly higher costs and more intensive treatment regimens. It is vitally important to include an assessment of immunogenicity for biosimilar products, which is best achieved through appropriately designed
clinical trials.
Clinical trials are essential in the regulatory approval process to ensure that biosimilars are safe and effective, including meeting an appropriate standard of immunogenicity. Skipping this important step could result in patients receiving treatment without adequate understanding of a products effect and potential adverse events. Rightly, in their guideline for biosimilars, the European Medicines Agency has insisted that plasma protein products and their recombinant alternatives must have the equivalent clinical dossier as the original innovator products.
Allowing a biosimilar to forgo human clinical trials makes it impossible to accurately claim that the follow-on product will have the same effectiveness and absence of immunogenic response as the reference product. This shortcut could result in therapies that promise to be effective, but are actually harmful to the patient.
Ensuring the development of safe, effective, and therapeutically equivalent biological products at a lower cost is a laudable goal, but it should not be achieved in a manner that risks the health and safety of patients. Biosimilars should be required to meet current safety and efficacy requirements with appropriate clinical studies that will ensure that the similarity of the biosimilar product in the laboratory with the innovator product is matched with acceptable responses in patients receiving the product. Until assurances can be made that patients will not respond adversely, any abbreviated regulatory approval process should take these issues into consideration and require follow-on biologics to demonstrate equivalent safety, quality, and efficacy
Mark W. Skinner
WFH President
Hemophilia World, April 2011
