2015 was a record year for biopharma in every sense of the word. There were a total of 45 FDA approvals this year, starting with Savaysa (endoxaban), a novel oral anticoagulant (NOAC) from Daiichi Sankyo, and ending (to date) with the approval of Zurampic (lesurinad), a medication for the treatment of gout-related uricemia marketed by AstraZeneca.
2015 was a record-breaking year in terms of M&A activity, too, with more than 1,000 transactions throughout the year. Total health sector M&A volume, in dollar terms, blew past the $600 billion mark, helped in large measure by the largest-ever healthcare merger—a $160 billion-plus deal between Pfizer and Allergan set to close next year. Overall corporate deal-making also reached staggering new heights, with $4.5 trillion in M&As this year, according to Thomson Reuters.
2015 was also the year when the conversation around drug pricing picked up enough momentum to actually force drug makers to publicly address the controversial issue. Lawmakers, payers, independent analysts such as the Institute for Clinical and Economic Review (ICER), disease-advocacy groups, physicians, and patients themselves started to weigh in on the debate. As the year ends, the conversation will continue—into 2016 and beyond.
This amalgam of forces provided context for what could possibly be considered one of the most innovative years ever for the biopharma industry—and marked a year when the FDA tried to usher as many treatment options to market as possible.
The year saw the approval of breakthrough immunotherapies for cancer, in addition to treatments for hypertension, diabetes, hypokalemia, schizophrenia, heart failure, cystic fibrosis, hypoactive sexual desire disorder in women, plaque psoriasis, and many other areas where there have long been unmet medical needs.
With everything that's happened in the past year, it’s hard to pin down exactly what was most significant. But BioPharma Dive asked top thought leaders in the industry to share what they considered to be the top biopharma "game-changers" of 2015.
Also be sure to check out our own analysis of the 10 top trends that drove biopharma in 2015.
Dr. Jay Edelberg, VP & Head of PCSK9 Development & Launch Unit, Sanofi
"2015 was a game-changing year in cardiology with the launch of Praluent (alirocumab), a PCSK9 inhibitor for cholesterol. Praluent was the first PCSK9 inhibitor available in the U.S., and it’s the only PCSK9 inhibitor currently available in two doses, which allows patients to personalize their treatment.
Praluent’s efficacy was displayed in clinical trials of patients with high cholesterol and/or high cardiovascular risk where most patients using Praluent achieved their cholesterol goals within weeks of commencing therapy. This same group of patients had previously struggled to control their cholesterol and lower their risk– often for decades."
Bert Liang, Bert Liang, MD, PhD, MBA, Chair, Biosimilars Council & CEO, Pfenex
"For the biosimilars world, the obvious game changer in 2015 was the approval of the first U.S. biosimilar, Zarxio, which transformed the theoretical into reality by marking a clear arrival of these disruptive technologies to the marketplace. Moreover, this development has initiated the process of statutory interpretation, which will have a significant impact on the industry as it develops and matures."
Heather Gartman, Regional Managing Director, inVentiv Health Public Relations Group
"In 2015, pharmaceutical companies evolved practices to focus more on incorporating the voice of the patient into the clinical development process, some as early as pre-clinical, to inform trial design and end-points.
Companies are increasingly recognizing the value of partnerships with patient groups so they can better understand the patients they are trying to serve. Earlier patient engagement allows companies to obtain meaningful insights that will better inform the strategies and activities that support clinical and product excellence."
Brian Overstreet, CEO, Advera Health Analytics
"One of the areas in 2015 where innovation started to take hold was in the area of improved data analysis – or 'big data.' While many have come to recognize the value of improved data collection and dissemination in healthcare, the real advances are being made in how best to analyze those disparate data and transform them into actionable intelligence.
Data don’t lie. Data tell stories that can’t be ignored. Data is factual evidence. And when presented via analytics it becomes clear that this knowledge truly is power for all stakeholders – payers, physicians and patients. Broad datasets have enabled MCOs to better monitor, control, and mitigate patient risks and system costs.
And the advent of these analytics that review and dissect data has enabled proactive and not just reactive action to be taken. Managed care health professionals are modifying the way they determine formularies and are altering their daily workflow, which substantially reduces the decision making cycle."
James Crowley, Accenture Strategy Life Sciences, Senior Managing Director, Accenture
"2015 was exciting year, with record-setting deal activity including the 3rd largest M&A deal ever announced (the Pfizer-Allergan merger). These were driven by the need for pipeline/innovation, scale and efficiency, and diversification.
The biopharma industry also had a record number of new drug approvals (45) by the FDA, which continues the trend of increasingly effective drug development in our industry. Additionally, this year was game-changing because many of the components that will enable a future of outcomes-based collaborations have come to fruition. The industry saw consolidation of major payers, meaning that the five largest for-profit health insurers are now down to three.
In parallel, integrated systems, which are also going through consolidation, are offering new opportunities to partner with providers to improve outcomes. As this occurs, many of Accenture’s biopharma clients have stated their intent to change their revenue streams from volume to value through outcomes-based arrangements in the near future.
Finally, the improvements in technologies (e.g., EMRs, wearables, mobile, etc.) are rapidly enabling the analytics needed to track patient outcomes to support these arrangements. At Accenture, we believe that 2016 is the year when all of these components will converge and biopharma leaders in outcomes-based partnerships will emerge, creating competitive advantage, new business models, and immense commercial value for first movers."
Dr. Eilon Kirson, Chief Science Officer and Head of Research and Development, Novocure
"2015 has been an amazing year in the field of oncology with the emergence of two new treatment modalities for the treatment of cancer. Historically cancer has been treated using surgery, radiation therapy and chemotherapy. After decades of research into possible effects of the immune system on cancerous tumor growth, the field of oncology has finally received a push forward with the approval of some of the first immunotherapies for advanced, late stage lung cancer, and melanoma.
However, even more exciting in my eyes is the promise of a completely new approach to treat cancer using tumor treating fields, or TTFields. TTFields are a physical treatment modality based on use of low intensity, intermediate frequency, alternating electric fields.
In October 2015, the FDA approved Optune (the Novo TTF-100A System), a system in which patients receive TTFields therapy via a home-use medical device. Optune was approved for treatment of patients with newly diagnosed glioblastoma---the most common and aggressive primary brain tumor.
Results of pivotal clinical trials proved unequivocally that TTFields, when added to standard- of- care chemotherapy, significantly extend both progression-free survival and overall survival in glioblastoma patients, without adding significant toxicity. In fact the proportion of patients alive at 2 years from starting treatment was 50% higher with TTFields than with chemotherapy alone.
I believe this is just the beginning of a new era in cancer treatment, and with these new approved therapies now added to our armamentarium of treatment options, we are that much closer to a cure for more and more types of cancer."
Kevin Robert Frost, CEO, amfAR, The Foundation for AIDS Research
"The Trans Pacific Partnership free trade agreement was passed, and it will change the game for the worse in terms of access to affordable medicines for people living with HIV in many parts of the developing world.
The agreement’s IP and patent provisions will undoubtedly delay the introduction of generics and will do little to facilitate the massive expansion of access to treatment recommended by the WHO in its 2015 guidelines urging treatment for all HIV-positive individuals, regardless of CD4 count. The TPP also underscores the need for an HIV cure.
My hope is that the newly established amfAR Institute for HIV Cure Research at UCSF will be a game-changer that accelerates the pace of cure research."
Ruchin Kansal, Executive Director, Business Innovation at Boehringer Ingelheim
"In 2015, the idea of moving from a pay-per-pill model to a pay-for-performance model received increased attention, with some pharma companies proposing new approaches to payers. CVS Health Corp. has said that they are evaluating several pay-for-performance drug plans.
This trend represents a shift in the structure of the industry and calls for innovative approaches to define value, including better options to track and monitor real-world data. We are seeing real examples of value-based approaches that could impact how companies talk about the value that they bring to the market and payers beyond the medicine."
Dr. Barry Mennen, Washington, D.C.-based physician
"The great breakthroughs this year, in my opinion, are the approval and/or development of small molecules indicated for the treatment of specific disease-causing genetic mutations. While the rapid translation of molecular insights into small-molecule therapy for these orphan diseases demonstrates brilliant science, it also showcases the efficient collaboration of the FDA and the pharma industry.
For example, July saw the approval of Vertex Pharmaceuticals’ Orkambi (lumacaftor 200 mg/ivacaftor 125 mg) for treatment of a specific type of mutation causing cystic fibrosis (CF). This combination addresses protein-folding micropathology in a subset of individuals with CF.
Another example is ataluren, a small molecule developed by PTC Therapeutics., which defeats the stop codon responsible for certain genetic diseases. Ataluren is part of a therapeutic category referred to as protein restoration therapy, which would have been thought of as science fiction 15 years ago.
In patients with a stop codon in diseases as diverse as cystic fibrosis and Duchenne Muscular Dystrophy (DMD), ataluren is able to allow the ribosome to read past the nonsense stop and produce viable protein. For CF, the protein is the chloride channel and in DMD the protein is called dystrophin and protects muscle cells from damage. There are other examples, but these make the point quite nicely.
Before these advances, the parents of children with these genetic/orphan diseases had little hope, but the game has changed, and the goal posts have been moved—closer!"
Dr. Hugo Stephenson, Executive Chairman, DrugDev
"The widespread approval and reimbursement of hepatitis C therapies was a major achievement for the biopharma industry. These transformational, common-curative therapies will prevent significant downstream health complications for patients.
The accomplishment is especially good for the pharma industry overall because after years without a big blockbuster approval, these therapies remind us all of the amazing results phrma can deliver to improve the health of hundreds of millions of people worldwide. The broader reimbursement means more money will flow back into the industry for continued R&D innovation. With these therapies, pharma proved to society that it is capable of doing great things, with more exciting therapies to come.
The second major disruption in 2015 was that the biopharma industry started to focus more on the importance of clinical trial sites, working to improve site satisfaction and ease the burden of those onsite. One example of this is TransCelerate’s creation of the Investigator Registry and its platform, which pools investigator data together into a centralized, cloud-based resource utilizing a universal identifier known as the DrugDev Golden Number."
Ashish Singh, Partner, Head of Bain’s Global Healthcare Practice
"A major game-changer in 2015 was increasing price pressure on prescription drugs in the U.S. market coming from a rapidly consolidating customer case (payers/PBMs, providers, and channel). This trend is now starting to endanger the economic lifeblood of the global pharmaceutical industry—the premium price reimbursement in the U.S.
Just consider 40% rebates for breakthrough products in the HCV market (Gilead and AbbVie) and the beginning battle for PCSK9 products (Sanofi and Amgen). After this year, the sustainability of business models based on raising prices for well-established legacy products, a common industry practice, is questionable. We are already seeing this play out in the cases of companies such as Valeant and Turing."