Imagine a world wherein certain diseases previously considered incurable are indeed treatable due to some radical science. And it involves biopharmaceuticals at the forefront of this change in medical science. But what is this thing called a biopharmaceutical, better known as a biologic? And how will these revolutionary new drugs change the paradigm of drug development?
So, let’s do a little deeper dive into what biopharmaceuticals are, why such a big deal, and how this part of biotechnology stood the traditional approach to drug development on its head. As I’ll go on further, it is going to be a little bit personal. Moreover, I have tried keeping it light with simple explanations such that it should not need you having studied any science class.
What is a Biopharmaceutical?
Biopharmaceuticals are pharmaceutical products prepared from living organisms, comprising proteins, cells, genes, and others. Biologics are produced through the use of living systems, in contrast to traditional drugs in pharmaceuticals that are synthesized chemically. Examples include but are not limited to insulin, vaccines, and monoclonal antibodies.
But most simply, it is the different nature of the complexity. In fact, the structure of aspirin is quite simple from a chemical point of view, whereas all the biologics are huge complex molecules. As these products are made out of substances already present in the body, they have indeed become particularly effective in dealing with different sorts of specific diseases.
How Biotechnology Powers Biopharmaceuticals
Biotechnology exploits biological systems for the obtaining of useful products for man. With respect to biopharmaceuticals, biotechnology avails ways through which scientists can:
1. Identification of Targets of Diseases: New tools such as CRISPR and next-generation sequencing have considerably empowered researchers to identify genetic causes of a disease.
2. Design Effective Therapies: With biotech, treatments can actually now be designed for the individual patient.
3. Manufacturing-Scale Production: Manufacturing processes using the full range of techniques from fermentation through cell culture applied to large-scale production of biologics.
Personal Story
A few years back, my friend’s father was diagnosed with rheumatoid arthritis. He did not improve on the traditional drugs and slowly worsened. Then his doctor started him on a biopharmaceutical called Humira. In a matter of months, he was drastically improved. To say the least, this was quite a marvel of how one biologic-targeted, acting at the site of inflammation changed one’s life.
The Biopharmaceutical Development Journey
The course of development that biopharmaceuticals take is tortuous. Sometimes running into tens of years of preclinical and clinical research pass many stages of trials, including:
1. Discovery and Preclinical Research
Scientists identify the disease target first something like some malfunctioning gene or protein. Thereafter, design a biological agent that acts on that particular target. Monoclonal antibodies hitch themselves to particular proteins and neutralize them.
2. Clinical Trials
Clinical trials are designed to test the safety and effectiveness of the biologic in human subjects. These studies proceed through three phases of testing:
Phase I: Safety is tested in a limited number of healthy volunteers.
Phase II: Effectiveness and side effects are assessed in a larger group of patients.
Phase III: Confirms efficacy and monitors adverse reactions in a broad population.
3. Regulatory Approval
If all clinical trials are successful, the drug is then submitted to regulatory agencies such as the FDA or EMA for approval. This assures that the biologic is safe and effective for use by the general public.
4. Manufacturing
Biomanufacturing is made through the culture of living cells under controlled environments. It is a form of farming, only that crops grown are therapeutic proteins.
5. Post-Market Surveillance
Even after hitting the market, biologics are followed for a period of time so that any further safety and efficacy can be noted.
Why Biopharmaceuticals Are Game-Changers
1. Targeted Therapies
Biopharmaceuticals work in conjunction with particular molecules of the body and are, hence, quite effective against cancers, autoimmune diseases, and genetic conditions common and rare.
2. Personalized Medicine
With recent advances in genomics, biologics can also be personalized to a patient’s unique genetic profile and offer a greater chance of success with fewer side effects.
3. Fewer Side Effects
Because biologics mimic the substances in nature inside our bodies, most of these drugs have fewer side effects compared to other kinds of medicines.
4. Breakthroughs in Diseases Considered Rare
Actually, biologics have opened up avenues for treating disorders that were beyond the reach of human beings to treat earlier. Enzyme replacement therapies for different metabolic disorders, which are extremely rare, are an outcome of this science.
I recently met a young woman at a conference who shared with me her story in fighting MS. For years she struggled under the weight of debilitating symptoms. But one single biologic, Ocrevus, changed everything. Magic, really, to hear her speak of regaining mobility and confidence.
Challenges in Biopharmaceutical Development
Despite their promise, biopharmaceuticals also have a number of challenges:
1. High Costs
Developmental aspects are very expensive and run into billions of dollars of investment. This in turn will translate into very expensive medicines for the patients and hence these may not be accessible.
2. Complex Manufacturing
Biologics require very advanced facilities and a rigorous system of quality control to produce, hence further leading to complexities in supply chains.
3. Regulatory Hurdles
Naturally, and rightly so, this translates to an extremely strict process in approving biologics, and it often severely postpones access to lifesaving therapies.
4. Biosimilar Competition
One patent expiration in a biologic opens up the market for biosimilars. Prices, subsequently, are reduced from the ensuing competition, but simultaneously raise questions on the viability of future innovation.
The Future of Biopharmaceuticals
The future of biopharmaceuticals looks brilliant, and a few of the trends to watch out for are as mentioned below:
1. Gene Therapy
Technologies like CRISPR are allowing scientists to edit genes and hence giving hope for the cure of genetic disorders.
2. Immunotherapy
Biologics that allow the body’s immune system to fight the living disease of cancer much more effectively will gain much momentum.
3. AI in Drug Development
Artificial Intelligence is also being used in Big Data to screen the possible biologics, therefore, reducing drug development times.
4. Global Access
The effort to make these biologics more available and accessible to the rest of the world will not leave anyone behind.
In Conclusion, Needless to say, biopharmaceuticals stand at the very frontier of medicines today. They can easily be mistaken to be much more than medication but sometimes as lines of life that rescue the futile hope of the millions that are suffering under debilitating life conditions. Biotechnology ushers in a beginning of times when medicines were never tailored to be so powerful and so promising.
The next time the term “biologic” is brought up, it has to be viewed not only in terms of achievement as far as science goes but as something that represents human genius and humanity.