Healthy Perspectives—

Preserving Next-Generation Biomedicines

For The Next Generation of Patients

By Dr Ken Miller

Vice President of the

Connecticut Rheumatology Society

Among the health care sectors that Congress is currently reengineering, none has higher stakes for patients than the future of biotechnology. While the drivers behind the debate are familiar — containing costs, increasing patient access, ensuring safety and efficacy — the rewards and risks to patients are entirely novel. That is because biotech drugs, powerful therapeutic agents that are little understood outside of physician specialties, are unique in the history of medicine.

While ordinary pharmaceuticals treat the symptoms of a disease, biotech drugs — called “biologics” — can be manipulated to target the underlying mechanisms and pathways of a disease. Armed with the newly-mined wealth of genomic and immunologic research, scientists and physicians have begun to harvest the extraordinary power of biology to advance breakthrough medical discoveries.

Over the past two decades, biotechnology has produced the most dramatic developments in medical science. Thanks to biotech drugs, doctors and their patients are finally gaining the upper hand in the war against life-threatening diseases such as cancer, diabetes and AIDS, as well as debilitating diseases such as rheumatoid and psoriatic arthritis.

Additionally, biologics currently in clinical trials promise to prevent the onset of deadly and debilitating diseases such as Alzheimer’s disease, heart disease, Parkinson’s disease, multiple sclerosis and systemic lupus erythematosus.

That is the good news — and for patients facing life-threatening disease, biologics have been very good news indeed. But, because they are complex and novel medications created from living protein chains, biologics are expensive to develop.

Also, owing to their complexity, biologics cannot be precisely duplicated in the manner of traditional generic drugs, raising significant safety and efficacy concerns about lower-cost “biosimilar” versions. The challenge facing Congress is how to make these breakthrough medicines less costly and more accessible while maintaining important safety standards and while continuing to nurture the research and development of improved treatments and cures.

From the perspective of medical practitioners and the patients we treat, ensuring safety and innovation in the next generation of biologics is paramount. Good public policy should aim to foster both development of better medicines and their availability to patients at lower costs.

Additionally, sufficient mechanisms should be in place to monitor the pre- and postmarketing safety profiles of these new agents.

Biologics have rightfully been hailed as “miracle medicines,” the leading edge of the new frontier of targeted, individualized medicine, but it is important to keep in mind that this “new frontier” is still just that — very new. While millions of patients have benefited from the first generation of biologic medicines, there remains a pressing need for improvement of these breakthrough medicines, including more efficient modes of administration and more favorable safety and efficacy profiles.

The research and development of improved next-generation therapies is vital to the step-by-step advancement of medicine — particularly in biologics. For instance, most biologics are injections that require doctor office or hospital visits.

When an improved biologic allows patients to have a single weekly injection rather than three a week — as is the case with Pegintron, a next-generation biologic for the treatment of hepatitis C — it has a dramatic and tangible positive impact on the quality of life for patients.

Another compelling example of the importance of incrementally improved next-generation biologics is insulin. Originally derived from pig pancreases, insulin has gradually evolved, from generation to generation of development, into a fully human-derived medication that has significantly higher purity and lower risk profile for millions of diabetics who depend on it daily for survival.

As a rheumatologist who treats arthritis and other crippling joint diseases, I have witnessed firsthand just how dramatically a next-generation biologic can improve the quality of life for patients. New self-injectable biologics are now given only once every two weeks or even once a month, improving quality of life in the treatment programs for certain patients.

Unfortunately, some on Capitol Hill have advocated sharp limits on the incentives that fuel research and development of next-generation improved biologics, even to the extent of requesting that these new and better biologics be denied access to intellectual property protections.

This would be disastrous for many patients facing life-threatening diseases for which next-generation biologics offer the best hope for better treatments and cures. Additionally, there are those who do not advocate for sufficient funding for monitoring the safety of these biosimilar agents which can cause significant reactions because of their immunogenicity and can be associated with life-threatening infections if not carefully monitored.

The biotech revolution has been a boon to doctors and the patients under our care, leveling the playing field in the fight against dread diseases. But if we hope to continue to advance patient care in the future and protect our current patients, we must foster the continued development of novel next-generation therapies and cures and maintain adequate vigilance regarding the safety of the drugs that we use.

The following medical opinion column was submitted by the author and does not necessarily represent the opinion of The Newtown Bee or its publisher.