Scientific advancements, more readily available medical information and emerging technologies are driving greater patient engagement, improving diagnosis and treatment, and enabling people with life-threatening diseases to live longer and healthier lives, said CSL Limited CEO and Managing Director Paul Perreault. CSL is the parent company of CSL Behring.
"It's all about diagnosing sooner, improving our ability to get the right treatment to the right patient at the right time, or even finding a cure," Perreault told global healthcare leaders at the 2015 International Plasma Product Biotechnology meeting this week. "Industry plays a critical role in this evolution. The way to succeed in the new environment is to innovate and adapt. In the end, we are about improving the well-being of patients."
As CEO of a leading global specialty biotherapeutics company, Perreault noted patients and doctors are more informed and engaged today than ever before. The vast amount of medical information available and new advances in technologies have empowered patients and helped doctors to better diagnose and treat those with life-threatening conditions. Advanced diagnostics and genomics are two of the technologies that are making a difference, Perreault said.
Advanced diagnostic technologies
"Technologies such as hand-held devices, advanced diagnostic tests and the Internet are revolutionizing the practice of medicine and the diagnosis of rare diseases, which often went undiagnosed for years," said Perreault. "This is important for physicians who now have information at their fingertips that can mean the difference between successfully diagnosing and treating a patient with a rare medical disorder or not being able to make a diagnosis, which can be frustrating and disheartening for both patient and doctor."
Genomics and personalized medicine
Genomic or precision medicine enables physicians to tailor treatment to the patient, or to better understand how a patient may respond to a particular therapy in advance. While this is well-established with certain cancers, it is less so in the rare disease space.
Gene therapy trials for patients with rare diseases are in progress to correct the gene mutations causing rare or serious conditions. Additionally, while personalized medicine is still in its infancy, advances such as the approval of new flexible dosing regimens for products used to treat immune deficiencies has improved administration convenience and the quality of life for many patients, Perreault said.
Inadequate market access
Perreault said inadequate market access affects many patient groups, but is especially critical for those with rare diseases. "We spend a great deal of time and resources understanding payer needs, the evolution of health technology assessments (HTA) and what these mean for patients, and support constructive engagement with payers," he said. "It's important that payers do not just look at cost alone. The value of therapies needs to be considered, too. Standard HTA assessments should be modified to reflect the special nature of rare diseases, the impact of these therapies on such serious conditions, the societal benefit and the small patient population size involved."
New healthcare delivery and business models
As a result of cost pressures and trying to improve efficiency and quality of care, Perreault said, "We are seeing an evolution of healthcare delivery and business models such as outpatient, home care and new dosage forms, which lead to improved quality of life, change in facility use, and broader and different make-up of healthcare teams." Today, more patients are being treated in outpatient settings, through home care, or Accountable Care Organizations, and often with a broader and different make-up of healthcare teams.
Cost of medicines
Although medicine spending is often portrayed as driving up healthcare costs, medicines are not a main driver of increased healthcare costs in the United States, Perreault noted. In fact, medicines account for a small and declining share of health spending growth. Every dollar spent on healthcare is broken down as follows, according to the Pharmaceutical Research and Manufacturers of America:
- $.07 – government administration and net cost of private health insurance
- $.08 – home health and nursing home care
- $.09 – prescription drugs
- $.21 – physician and clinical services
- $.23 – other
- $.32 – hospital care
In such a complex and evolving global healthcare environment, the engagement of stakeholders as partners in solutions is paramount, Perreault said. For instance, rare disease patient groups are increasing their global presence and capabilities in information sharing, community building and advocacy. Physicians work in tandem with those groups and governments are increasingly engaging them for input, including helping design health programs and policies. Industry has a role in supporting patient care, information sharing, stakeholder capabilities and engagement with all stakeholders. Working together provides the best opportunity for successful evolution.
"Our healthcare systems worldwide have evolved tremendously over the past decade, and they will continue to do so at exponential rates," said Perreault. "Change is constant, but our future will remain bright if we embrace the opportunities that result in better patient care, and if we work together to shape the healthcare environment to increase access and improve outcomes."
About CSL Behring
CSL Behring is a leader in the plasma protein therapeutics industry. Committed to saving lives and improving the quality of life for people with rare and serious diseases, the company manufactures and markets a range of plasma-derived and recombinant therapies worldwide.
CSL Behring therapies are used around the world to treat coagulation disorders including hemophilia and von Willebrand disease, primary immune deficiencies, hereditary angioedema and inherited respiratory disease, and neurological disorders in certain markets. The company’s products are also used in cardiac surgery, organ transplantation, burn treatment and to prevent hemolytic disease of the newborn.
CSL Behring operates one of the world’s largest plasma collection networks, CSL Plasma. CSL Behring is a global biopharmaceutical company and a member of the CSL Group of companies. The parent company, CSL Limited (ASX:CSL), is headquartered in Melbourne, Australia. For more information, visit www.cslbehring.com.
Chris Florentz, CSL Behring