Pharmaceuticals Policy and Law

A healthy life sciences eco-system^1 is an environment where all stakeholders, patients, researchers, governments, the civil society and manufacturers, work together to support the sustainable development and provision of innovative solutions that address unmet health needs. To build a stronger eco-system, governments could prioritise policy initiatives such as: facilitating the commercialisation of academic research; encouraging clinical research; accelerating the adoption and diffusion of new innovative medicines; and promoting the local market as a place to invest and deliver life sciences innovation [1].

Historically, the development of medicines has been primarily undertaken in, and for the benefit of, high income countries. Over the last 20 years, this has started to change, partly as a result of the market opportunity increasing in middle-income countries (MICs) and low-income countries (LICs) and partly because governments in these countries have recognised the importance of encouraging innovative industries, resulting in a greater priority given to addressing diseases highly prevalent in MICs and LICs.

In this article, we draw on our experience to develop lessons on how to establish a sustainable life sciences eco-system in MICs and LICs. We highlight the importance of different types of government policy (industrial policy, the regulatory framework, intellectual property, and improving access to medicines) and how success requires different stakeholders (public and private, international and national) to work together.

Health is a global concern. The need for a globalized response is evident in the pharmaceutical industry. Although pharmaceutical products are developed and marketed internationally, they are currently regulated only at national level. The pharmaceutical agencies regulate medical products in a globalized environment. However, national regulations can create significant barriers to pharmaceutical availability. We must formulate our laws with a global focus. The globalization of regulation weakens national sovereignty but empowers transnational epistemic networks. For this reasons the pharmaceutical agencies are involved in several bilateral and multilateral cooperation activities with international partners. International cooperation is a key area of work for the agencies. This process will benefit of advancement in global governance and progress toward supranationalism. The internationalization of the pharmaceutical industry, highly globalized, involves changes in policies, lifestyle and culture, and has altered drug research, production, and regulation.

Compulsory licenses of patents under the WTO Agreement on Trade-Related Aspects of Intellectual Property Rights (TRIPS) are often mistakenly viewed as a solution to problems relating to access to medicines in developing countries. Access requires a strong political commitment, a health system that contains multi-disciplined health professionals, and an adequate infrastructure to enable transportation of patients and equipment. The use of compulsory licenses should be a rare event considered only under extremely limited circumstances and not an instrument of industrial policy. If a government decides to issue a compulsory license, there are several technical and procedural requirements that must be satisfied under TRIPS. This paper explores those requirements and examines instances where courts have issued decisions relating to compulsory license requests or grants. It analyses the key provisions of TRIPS that are relevant to a government grant of a compulsory license without the authorization of the right holder. It also provides examples and analyses of previous grants of compulsory licenses that have been deficient in meeting on more more procedural requirements under TRIPS.

A robust, time-limited system of patent protection is proven to facilitate development of, and access to, innovative pharmaceutical products and processes. In particular, a well-functioning patent protection system is a prerequisite for attracting finance for costly pharmaceutical research, given its high failure rates, by ensuring that successful innovation is rewarded. This article offers a short scientific introduction to current research and development (R{&}D) in the biopharmaceutical industry before going on to consider the economic role of patent protection, in enabling research through access to capital markets and in accelerating access to new, innovative medicines. In this regard, the article discusses the beneficial economic effects of well-functioning patent systems in regions with restricted access to investors and capital markets. It concludes by underlining the importance of a robust and finely-tuned patent system in contributing to scientific progress and development of new life-saving drugs.

Understanding the pharmaceutical value chain requires the identification of each component from manufacturer to end consumer of medicines - and to understand their interaction. In most cases, the manufacturer's selling price represents only a fraction of the retail price of a drug. More than half of the end user price results from insurance, freight charges (CIF), import tariffs and charges, importer margin, distributor margin, retailer margin and taxes.

The article describes the elements of the medicine value chain, outlines factors and costs that contribute to the difference between the net price a pharmaceutical manufacturer receives for a drug and the final amount paid for the drug by the end user. It quantifies the price build-up for specific therapy areas and countries and illustrates the diversity of approaches and costs associated with the value chain through case studies.

Recently adopted, the WTO Agreement on Trade Facilitation (TFA) adds fresh momentum to worldwide efforts to speed up the movement, release and clearance of goods across borders. With all required decisions having now been taken in Geneva, preparations are under way to ensure the Agreement's expeditious entry into force. Once in operation, this ground-breaking treaty will significantly accelerate cross-border trade and reduce related costs.

This article analyses the TFA from a pharmaceutical angle, highlighting provisions of particular interest to the industry. It will look at how the new Agreement is likely to impact trade in medical goods and where business stands to benefit. A final segment will review governments' implementation plans and discuss the road ahead.

The International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use, also known as “ICH”, is a key international harmonisation initiative founded by the drug regulatory authorities and industry associations from the European Union, Japan and the United States of America. The main objective of ICH is to promote public health globally through the development and implementation of harmonised guidelines and standards. With its recent reform, ICH became an Association under Swiss law, and set the stage to broaden its membership to regulatory authorities and international pharmaceutical industry associations beyond the three founding regions. Building on greater than 25 years of harmonisation work, ICH is now well-positioned to grow into a truly global venue for the development of guidelines and standards to facilitate the registration of human medicines across the world.

The role played by properly functioning regulatory systems towards enhancing access to essential medicines for patients is crucial. This is especially the case in Africa which has seen progressive growth in the regulatory environment. At the center of this growth has been the African Medicines Regulatory Harmonization (AMRH) initiative. This initiative seeks to strengthen regulatory capacity and encourage harmonization of regulatory requirements - with the ultimate aim of expanding access to quality, safe, and effective medicines for patients in need in Africa. A lot of progress has been made during the last years, with initial focus on the East African Community, where harmonization related regulations have already been implemented. The same is now being rolled out in other regions such as West Africa and the Southern African Development Community.

Removing bottlenecks and reducing redundancies in regulatory processes that slow access to medicines for patients in need today is critical. In this sense, collaboration between the World Health Organization and relevant stakeholders, including the research-based pharmaceutical industry, on collaborative registration procedures that support fast and efficient review and approval of essential medicines in Africa is essential.

African regulatory harmonization offers many benefits to regulatory authorities, patients in Africa and industry alike - and most critically for the protection of public health.

The role played by properly functioning regulatory systems towards enhancing access to essential medicines for patients is crucial. This is especially the case in Africa which has seen progressive growth in the regulatory environment. At the center of this growth has been the African Medicines Regulatory Harmonization (AMRH) initiative. This initiative seeks to strengthen regulatory capacity and encourage harmonization of regulatory requirements - with the ultimate aim of expanding access to quality, safe, and effective medicines for patients in need in Africa. A lot of progress has been made during the last years, with initial focus on the East African Community, where harmonization related regulations have already been implemented. The same is now being rolled out in other regions such as West Africa and the Southern African Development Community.

Removing bottlenecks and reducing redundancies in regulatory processes that slow access to medicines for patients in need today is critical. In this sense, collaboration between the World Health Organization and relevant stakeholders, including the research-based pharmaceutical industry, on collaborative registration procedures that support fast and efficient review and approval of essential medicines in Africa is essential.

African regulatory harmonization offers many benefits to regulatory authorities, patients in Africa and industry alike - and most critically for the protection of public health.

Towards African Regulatory harmonization processes - Accelerating patient access to medicines.

The role played by properly functioning regulatory systems towards enhancing access to essential medicines for patients is crucial. This is especially the case in Africa which has seen progressive growth in the regulatory environment. At the center of this growth has been the African Medicines Regulatory Harmonization (AMRH) initiative. This initiative seeks to strengthen regulatory capacity and encourage harmonization of regulatory requirements - with the ultimate aim of expanding access to quality, safe, and effective medicines for patients in need in Africa. A lot of progress has been made during the last years, with initial focus on the East African Community, where harmonization related regulations have already been implemented. The same is now being rolled out in other regions such as West Africa and the Southern African Development Community.

Removing bottlenecks and reducing redundancies in regulatory processes that slow access to medicines for patients in need today is critical. In this sense, collaboration between the World Health Organization and relevant stakeholders, including the research-based pharmaceutical industry, on collaborative registration procedures that support fast and efficient review and approval of essential medicines in Africa is essential.

African regulatory harmonization offers many benefits to regulatory authorities, patients in Africa and industry alike - and most critically for the protection of public health.

Biotherapeutic products (BTPs), also known as biotherapeutic medicines, contain structurally complex active substances produced by living organisms. Due to their complexity and method of manufacture BTPs require distinct regulatory approval standards relative to chemically-synthesized small molecule medicines. This is also relevant for licensing copied versions of a BTP, or similar biotherapeutic products (SBPs) made by a different manufacturer where regulatory concepts developed for generics should not have been applied. In all these licensing scenarios regulators need to evaluate the results of comparability exercises, including sensitive head-to-head analytical, pre-clinical and clinical comparisons with the original product as a basis for approval.

SBPs do not contain chemically identical active substances, and may have slightly different benefit-risk profiles, therefore it is necessary to monitor post-approval safety on a product-specific basis. Policymakers may therefore emphasize the need for product-specific identification in patient records and safety reports using either a unique trade name or a distinguishable non-proprietary naming system. The unique nature of BTPs also informs the nature and degree of interchangeability between the originator and SBPs versions. Many policymakers also emphasize that switching between SBPs should only occur with the involvement of the prescriber. It is recommended that pharmacy substitution would only be appropriate when there is a robust framework for a competent authority to assess product-specific evidence of interchangeability. Another challenge is posed by the historical existence in some jurisdictions of copy BTPs that were not assessed according to current regulatory standards. To address this situation the World Health Organization has proposed a regulatory assessment framework wherein the status of such products can be normalized via the orderly submission and review of supplementary data.

The presented facts suggest that import testing does not protect patients. On the contrary, it introduces potential risks to access of medicines and reduces the remaining shelf life time of medicines driving possible drug shortage. In the absence of data proving the evidence that import testing is decreasing risk to patients, if manufacturers comply with the evolving Good Manufacturing Practices (GMPs) and Good Distribution Practices (GDPs) regulations, including secure supply chains with documented controls, import testing should be waived. In these cases, importing country's Health Authorities should be confident the product is safe, of high quality, and in compliance with registered specifications.

This article presents risk assessments demonstrating that product quality is continuously controlled. Moreover, import testing does not detect counterfeit or substandard products nor reduces the additional risks related to local distribution channels, as testing occurs at the point of entry into a country.

Every day Pharmacovigilance becomes increasingly important to patient health. There are some gaps and limitations in the current Latin American Pharmacovigilance framework which could be addressed to have a better system to correctly and promptly identify suspected adverse drug reactions (ADR). Considering this context, Vigilantia was born as an initiative to foster Pharmacovigilance both scientifically and educationally, and enhance all aspects of the safe and proper use of medicines, across all Latin America.

Counterfeit medicines are, first and foremost, a matter of patient health and safety. Counterfeit medicines pose a threat to patients because of the conditions under which they are manufactured, in unlicensed, unregulated, uninspected and often unsanitary sites.

The “medicines” themselves pose a threat to patient health and safety because their contents are not regulated and they may not contain the correct active pharmaceutical ingredient (API) to deliver the therapeutic benefit for which they were prescribed, or even ingredients that are themselves harmful such as heavy metals or pesticides.

To mitigate that threat, and ensure that their patients receive safe and effective medicines, pharmaceutical companies have incorporated anti-counterfeiting technologies into their packaging and implemented campaigns to detect and disrupt those counterfeiters who place greed above patient safety.

Although counterfeiting presents a global threat from which no company, therapeutic area, region or country is immune; gauging the true scope of the problem has remained a challenge. There are hopeful signs, however, as we have seen improved reporting and greater transparency by enforcement and regulatory agencies.

Antimicrobial resistance (AMR) has emerged as a significant threat to global health security and threatens the achievements of modern medicine. Research and successful development of new antibiotics, especially those with novel mechanisms of action vital to combat resistance, has slowed dramatically since the 1980s. Surveillance for AMR is highly variable globally with significant limitations in many countries impeding the ability to fully characterize the problem. Global efforts to control tuberculosis, malaria and HIV are facing increasing difficulties from the emergence of resistance. Similarly, bacteria causing some of the most common infections in communities or in hospitals such as Escherichia coli and Klebsiella pneumoniae have shown high levels of resistance to third generation cephalosporins requiring treatment with expensive carbapenems as last-resort. Additionally, Streptococcus pneumoniae has shown reduced susceptibility to penicillin in many regions, exceeding 50% in some settings. The cost in lives from AMR over the next 40 years could go as high as 10 million per year with the cost to economic development as high as $3 trillion per year if current trends continue. In addition to ensuring appropriate use of antibiotics and development of novel classes with new or enhanced mechanisms of action, many plans for the global response call for new vaccines as integral to the fight against AMR. Vaccines and antibiotics should be used together to produce synergistic gains in public health, and ultimately, vaccines will extend the clinical utility of antibiotics. The decrease in cases of invasive pneumococcal disease and decrease in prescriptions for antibiotics in some settings resulting from the introduction of broad access to, and utilization of conjugate vaccines for Streptococcus pneumoniae exemplifies the synergy that can be achieved in the fight against AMR.

Companies in many industries are engaging in a changing business environment where the community is expecting greater transparency and ethical standards than in the past. This has been for a variety of reasons associated with globalization, technological and social changes. The pharmaceutical industry is one industry where such issues are regularly under the spotlight. In this context the IFPMA works with its member companies and national associations to enhance the agenda of self-regulation and ethical behavior. The global IFPMA Code of Practice, and the many national industry association codes that implement it, have evolved over time to help the industry take the lead in driving greater ethical standards and transparency. This article will review the current international business literature on ethics generally, review the functions and evolution of the IFPMA Code of Practice and examine some of the more recent evidence and analysis of the role of pharmaceutical industry codes, ethics and reputation in the pharmaceutical industry.