Report Highlights

The global orphan drugs market should reach $315.6 billion by 2024 from $127.5 billion in 2017 at a compound annual growth rate (CAGR) of 13.8% for the forecast period of 2017 to 2024.

Report Scope:

The scope of this study begins with the challenges facing biotech and pharmaceutical companies today. It goes on to discuss regulatory approval processes in various countries, how these are changing, and the knock-on effects which the pharma/biotech companies will have to address. The report then reviews several key financial ratios, then discusses how to maximize clinical development success rate. We then go into how machine learning (ML) and artificial intelligence (AI), along with Big Data, are disrupting the pharma/biotech industry, in a positive way. The report then reviews the global markets for treatments for selected cancers, chronic diseases, cardiovascular disease, neurological diseases, infectious diseases and rare diseases. Chapter 15 discusses how companies need to deeply integrate ML, AI and Big Data into drug discovery and clinical testing—or risk falling behind the competition. The report wraps up with summaries of selected companies active in these spaces and beginning to utilize digital tools in R&D.

Report Includes:

- 49 data tables and 30 additional tables
- An assessment of return on investment (ROI) in R&D by top pharmaceutical companies within the industry
- Analyses of the global market trends for various chronic disease areas, with data from 2016 to 2017, and projections of compound annual growth rates (CAGRs) through 2024
- Pipeline analysis of various therapeutic drugs with oncology being the largest, and coverage of ongoing clinical trials and promising forthcoming therapies in late stage etc.
- Outlining details of many factors involved in calculating ROI in R&D in pharma and how some of those factors are adjusted/developed to improve R&D ROI
- Discussion of the competitive landscape and key mergers and acquisition deals, partnerships, collaborations during the timeframe, 2014-2024
- Profile descriptions of top 15 pharmaceutical companies, their sales data, market capitalization and areas of R&D etc., including 23andMe, AstraZeneca PLC, Celgene Corp., Eli Lilly and Co., GlaxoSmithKline PLC, Merck & Co. Inc., Pfizer Inc. and Sanofi

Summary

This report investigates recent years of performance of pharmaceutical and biopharmaceutical companies with respect to return on investment (ROI) in research and development (R&D), and to discuss ways that companies are trying to improve ROI through 2024.

ROI and R&D Outlays

Many factors enter into the calculation of ROI; this is not a straightforward calculation, especially if one does not have access to the company’s internal accounting books. However, the fact that many factors enter into the calculation, also may be taken to mean that a number of factors might be adjusted or developed to improve ROI. Laying off employees is one way, but this is not effective longer-term unless the company is at the same time reorganizing for greater efficiency. Sanofi, for example, hit hard by pricing pressure in diabetes, started laying off 400 US sales employees in June 2019 but states that it is also undergoing a reorganization to its salesforce.

Global research and development spending is forecast to grow by REDACTED in 2019 to a total of REDACTED in purchasing parity values for the more than 110 countries that have significant R&D investments of over more than $100 million, according to the 2019 Global R&D Funding Forecast, an annual compilation produced by R&D Magazine. The amount of investment in R&D is heavily influenced by a country’s economic power, plus the importance that the country places on research. China continues to increaseits global share of R&D spending with an increase of REDACTED, as other countries lose share in the R&D pie. The US will increase its R&D spending by about REDACTED in 2019 over 2018.

Using Technology to Weed Out Losers, Focus on Potential Winners

The most significant route to decreasing costs and improving failure rates is to cut off the development of a failing drug as early as possible in order to focus on potential winners. Pretty obvious—but how best to do this?

Many pharma and biotech companies are exploring the use of technology to make the most of clinical development and clinical trials. In the next three to five years:
- Robotic automation will streamline resources across the clinical trial value chain.
- Use of AI, ML and IT with Big Data will enable researchers to see larger trends and patterns in data, and to act on those trends earlier in the process.
- Clinical trials will become open, and trials will be better understood by the public.
- Clinical trials will become patient-centric and better integrated into medical systems, thereby becoming an integral part of health care.