A 2007 PriceWaterHouseCooper study found that pharmaceutical companies needed to transform the processes it uses to conduct R&D. Too many companies were investigating new molecules before they had created a clear picture of the pathology of the diseases and that they were trying to address and the physiological responses those diseases cause. This was too narrow a focus at such an early stage in the research process, and helped explain why attrition rates in development are so high. They stated that, by 2020, ?the most successful companies would be those that focus on building a much better understanding of the pathophysiology of disease. They will study the disease variability arising from multifactorial aetiology, the underlying disease mechanisms, targets that are amenable to therapeutic intervention and what markers could be used to distinguish between patients with similar clinical symptoms but distinct biological conditions.?
Similarly, Dr. Scott Gottlieb, previously FDA?s Deputy Commissioner for Medical and Scientific Affairs, noted, the highly empirical, statistical method that currently predominates is inflexible; it restricts innovation and results in ?overly large? trials that yield information ?about how large populations with the same or similar conditions are likely to respond to a treatment. But doctors don?t treat populations, they treat individual patients.?
Pharma is delivering on this challenge.? Today, maintaining a robust pipeline of products is far from turn-crank. As scientists go deeper into disease pathways during drug development, pharma companies are innovating and combining multiple therapeutic modalities.? The business process transformation is delivering results.
A July 2017 report entitled The Biopharmaceutical Pipeline: Innovative Therapies in Clinical Development by Genia Long of the Analysis Group provided stunning insights into the advances in medical science and pharmaceuticals.
Here are some excerpts:
- Today?s pipeline of new medicines is vibrant, diverse and incredibly promising for patients. The analysis found that there are more than 4,000 projects ? defined as unique molecule-indication combinations ? in clinical development for oncology, 450 for cardiovascular disease and 700 for neurology, including 143 in Alzheimer?s disease, 67 in Parkinson?s disease and 29 in amyotrophic lateral sclerosis (ALS).
- 74 percent of medicines currently in clinical development are potentially first-in-class, meaning that they represent a completely new approach to fighting disease.
- The report also found that 822 projects currently in development have been designated as orphan drugs by the U.S. Food and Drug Administration (FDA). This is critically important as there are about 7,000 rare diseases and only 5 percent have an FDA approved medicine.
- A range of novel scientific approaches to address various diseases and conditions were being pursued in clinical development, including: - 731 projects using gene therapy, in which a patient?s genes are modified to treat or prevent a disease, or cell therapy, in which healthy, functioning cells are introduced to treat a disease or condition in which the patient?s cells are damaged or diseased. - 173 projects developing DNA or RNA therapeutics (which target DNA and RNA, which carry and transmit the genetic information that creates proteins, such as antisense drugs that block messenger RNA translation, thereby preventing the synthesis of certain disease-associated proteins). - 188 projects using conjugated monoclonal antibodies, which use monoclonal antibodies joined to other agents such as chemotherapy drugs to target them to specific cells such as tumors, while sparing nearby healthy cells.
- A range of novel scientific approaches are being pursued, including cell and gene therapies, DNA and RNA therapeutics and conjugated monoclonal antibodies.
- In addition to the medicines and treatment methods, clinical trial designs are also changing as genetics and biomarkers are being used to pinpoint select populations of patients with a higher probability of successful response. Patients are also becoming more directly involved in actual development of clinical study protocol by providing more insights to their caregivers through digital communications.
Read more about these incredible developments at Innovation.org
Digitally Redesigning the Business of Pharmaceutical Industry
These phenomenal scientific and medical advancements are ushering in entirely new pharmaceutical and clinical business models empowered by digital technologies.
Here are just a few of those changes:
The discovery process and scientists are now supplementing their work through the use of artificial intelligence.? Big Pharma Turns to AI to Speed Discovery
Digitally Connecting with Patients in Clinical Trials
Novel clinical process flows based on adaptive trial designs are being enabled by dynamic monitoring of patients with wearable devices. Wearable-derived data in clinical trials-The Biggest Obstacles are Crumbling
This wearable sensor measures skin temperature in addition to glucose, lactate, sodium and potassium in sweat. Integrated circuits analyze the data and transmit the information wirelessly to a smartphone or other device.? Der-Hsien Lien and Hiroki Ota,/UC-Berkeley
Digitally Connecting the Pharma Value Chain End-to-End
A new level of digital connectivity is creating new business process design possibilities up and down the entire pharmaceutical end-to-end value chain. Cognitive and predictive analytics can vastly change practices across the entire pharma value chain.? Source:? How Pharma Can Accelerate Business Impact from Advanced Analytics
Fundamentally Different Business Models in the Future Require Digital Rethinking
More sophisticated research into underlying disease and vastly greater genomic and epidemiological data have pushed patient-centricity and tailored therapeutics to forefront. ?The complexity of data managed by pharma companies puts them squarely in the domain of big big data.? Leading pharma companies are poised to create breakout performance as they transform their business and become part of the broader digital healthcare platforms where every patient possesses their own genetic and health history ?data-ome? through which they can communicate with a sophisticated network of care providers.? Every provider of a product or service will need to reinvent their business using this a lens for identifying themselves and their place within the provider ecosystem for the patients, the other providers of the ecosystem, and more broadly for the wider benefit to society.
When the human body is the biggest data platform who will capture the value?
Still Not Impressed?
It has been an incredible year for healthcare technologies so far. Check out this list of? The 16 Most Remarkable Healthcare Innovations, Events, and Discoveries of 2018 from World Health Day 2018.
At BP3 Global, our healthcare/pharma team is committed to helping healthcare providers and pharmaceutical companies move faster to this future state.? In addition to our pharmaceutical industry expertise, we bring full suite of digital operations services and technology to the table, including: Digital automation, robotic process automation, process transformation, decision management and rules services, and much more!
For more on how the pharmaceutical industry is advancing with technology register for our Next Generation Pharmacovogilance webinar happening on Wednesday, October 3rd at 10 A.M. (CST). Click here for more information.