Open source pharma: A gem for patients?

Open source pharma: A gem for patients?

Last Updated on April 21, 2017 by Joseph Gut – thasso

Overall, the scientific and medical community has discovered and developed many groundbreaking medicines that have had a major impact on public health. However, drug development is challenged by a widening gap between health needs and the pharmaceutical industry’s motives and business model, alongside a decrease in efficiency per research dollar spent in medicinal product research and development (R&D), a trend known colloquially as Eroom’s Law. Such fundamental challenges result in frequent high-level calls for new initiatives to develop therapeutics and bring them to market. These include market push and pull mechanisms such as priority review vouchers, advance market commitments, and public R&D funding. New organizational models have also emerged, including public–private partnerships (PPPs) and not-for-profit product development partnerships (PDPs) (for example, the Drugs for Neglected Diseases Initiative [DNDi], the Medicines for Malaria Venture [MMV], and the Global Alliance for Tuberculosis Drug Development [TB Alliance]) that often apply a full “de-linkage” model in which the price of medicines and the cost of R&D are uncoupled, in an attempt to make effective medicines affordable for those who need them most, but who do not have themselves the economic means to pay full prices set by companies and health care systems in economically powerful world regions.

Here comes open source pharma (OSP) into play, a suggested approach that has not yet been fully evaluated. In general, the term open source (OS) refers to radically transparent working practices particularly pioneered in software development, such as the prepublication sharing of data and ideas, the possibility of participation in a project by anyone in real time, and a form of shared ownership that ensures that the underlying methods and data are public domain. In OS pharma, such principles would be applied from the discovery of a potential medicine through to its entry into the market; the impact of this, and the ease with which such principles can be applied, will vary at each stage of the process.

OSP could bring the greatest possible community involvement, but the transparency brings economic uncertainty in how to exploit the solutions. In fact, the underlying financial and legal structures of the pharmaceutical industry are designed for use by profit-making entities in ways that rely on monopolies, usually through patents. Under the current model, it is assumed that without exclusive ownership, there can be no guarantee of profit to shareholders—a disincentive for private sector investment in certain areas of health R&D, despite significant public investments. The requirement for a financial return causes the current system to follow priorities that can be misaligned with the greatest public health needs. The requirement for secrecy inevitably reduces the efficiency of the scientific research. The transparency at the heart of OSP is thus fundamentally incompatible with traditional approaches to drug discovery and development. The involvement of the pharma industry in consortia that address neglected or tropical diseases (e.g., the Tuberculosis Drug Accelerator (TBDA), which is invitation only and has no commitment to open data) ensures that a similar walled approach is usually taken even for diseases traditionally considered to yield a low return on investment.

In their essay, the authors propose that OSP methods may be applied to (1) increase the efficiency of the research process and (2) realign R&D to address the most pressing public health problems as opposed to the most promising market opportunities. Thus, in OSP, medical R&D should respond to specific priority health needs. Opening up the R&D process to a wide range of contributors and stakeholders will allow the design of medicines that are better adapted to the needs of the end users, and will define the preferred product characteristics (PPCs) and target product profile (TPP) that will guide all the phases of product development. In OSP, there are no insiders, meaning that strategic decisions may be made through informed community debate, under inclusion of the (apomediary) patient expert, against the agreed-upon TPP. The broader community is thus involved in designing how a product will be developed, defining the studies that will be done, and establishing the criteria that will be applied for making stop/go decisions.Such an open system would also easily allow for the development of targeted therapies for enormously large global patient populations who carry identical genetic outfits for drug efficacies and drug response. In order to achieve this, investments are needed to build online communities and platforms that help groups collaborate effectively.

One of the main problems surrounding OSP is the uncertainty as to how it can be financed, due to the lack of a precedent. We foresee that approaches can be refined as more projects are developed following these principles and experience accumulates. However, If a promising drug candidate addressing a major public health burden was generated through this process, it would be expected that significant public and philanthropic funds would be available for further development. Such is already the case in the field of poverty-related infectious diseases and neglected tropical diseases, in which there is a traditional type of what is often termed “market failure,” and where other financing mechanisms, such as crowdfunding and prizes, are being piloted. There are recent precedents of drugs being taken through such stages with government or philanthropic money and in the absence of patent protection, as well as an increasing number of major new funding mechanisms designed to assist with such efforts. One could see OSP as a key means to achieve the aims of the London Declaration and OSP projects as good candidates for support from the proposed Pooled Fund.  The potential should be broader, however. The enormous toll on our society from cancer led former United States Vice President Biden to urge researchers to “break down silos and bring all the cancer fighters together—to work together, share information, and end cancer as we know it” as part of the Cancer Moonshot initiative. There are persistent calls for new approaches to the development of antibacterials to counteract the looming threat of antimicrobial resistance (AMR) [44]. Given the magnitude of the problem of dementia, it is conceivable that governments of countries in which the population is shifting progressively towards old-age citizens would be willing to invest in the development of affordable treatments (in ways that the private sector cannot), rather than being overwhelmed by the eventual costs of patient care. Consortia and funds may be established to tackle these threats without correspondingly new approaches in how the underlying research is being conducted.  On can  see  OSP as providing that genuinely new approach.

To follow up on the topic, please read the full article by Balasegaram M, et. al. here. You may also consult this underlying text containing further details of the open source pharma roadmap, and you may also want to visit




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Ph.D.; Professor in Pharmacology and Toxicology. Senior expert in theragenomic and personalized medicine and individualized drug safety. Senior expert in pharmaco- and toxicogenetics. Senior expert in human safety of drugs, chemicals, environmental pollutants, and dietary ingredients.

1 Comment on “Open source pharma: A gem for patients?

  1. In a process known as venture philanthropy, disease-specific foundations have for some time now formed partnerships with industry and federal agencies to share the financial risk of therapeutic development, shorten the early translational pipeline, and advance research with a focus on human, not financial, return. These collaborations could serve as models for open source pharma approaches discussed above.

    There is a nice article in the NEJM, which illustrates the progress being made in developing medicines in a translational and quasi open source phase setting in Type-1 Diabetes, Multiple Myeloma, and Cystic Fibrosis.

    See here the article: <> by Ramsey, coined Academic, Foundation, and Industry Collaboration in Finding New Therapies.