Improving access to quality medicines in East Africa: An independent perspective on the East African Community Medicines Regulatory Harmonization initiative.

Alexander Giaquinto and co-authors discuss the East African Community's Medicines Regulatory Harmonization initiative.

Improving Drug Development and Patient Access With the Right People, Processes, and Culture: What Needs to Happen Right Now to Bring Better Medicines to the Patients Who Need Them.

In a global environment where health care costs are soaring, R&D efforts are flatlining. Meanwhile, payers are demanding more value for their money. In this environment, the traditional siloed drug development model is not sustainable. Yet innovations such as adaptive trial designs, real-world data collection, precision medicine, and patient-centric trials that have been proven to streamline clinical trials, enrich the data they produce, and facilitate reimbursement have not been widely adopted. Why? Because the medicines ecosystem currently lacks 3 key prerequisites for innovative and sustainable change: (1) a highly trained workforce that can handle and interpret high volumes of fragmented data; (2) a dependable process for judging what constitutes value in medical innovation; and (3) corporate cultures that learn fast from failure and mine the competitive riches offered by a diverse workforce. Cultivating the right people, processes, and culture will require a concerted effort by industry, academia, governments, and nongovernmental organizations (NGOs) to devise efficient, effective solutions.

Pharma Opportunities and Risks Multiply as Regulatory Reform Remakes APAC: Expanded Accelerated Pathways Challenge Developer Value Story, Evidence Collection, and Market Access Strategies.

Sweeping reforms in the largest markets of the Asia-Pacific region are transforming the regulatory and commercial landscape for foreign pharmaceutical companies. Japan, South Korea, and China are leading the charge, establishing mechanisms and infrastructure that both reflect and help drive international regulatory convergence and accelerate delivery of needed, innovative products to patients. In this rapidly evolving regulatory and commercial environment, drug developers can benefit from reforms and proliferating accelerated pathway (AP) frameworks, but only with regulatory and evidence-generation strategies tailored to the region. Otherwise, they will confront significant pricing and reimbursement headwinds. Although APAC economies are at different stages of development, they share a common imperative: to balance pharmaceutical innovation with affordability. Despite the complexity of meeting these sometimes conflicting demands, companies that focus on demonstrating and delivering value for money, and that price new treatments reasonably and sustainably, can succeed both for their shareholders and the region's patient population.

Collaboration and Convergence: Bringing New Medicines to Global Markets in the 21st Century.

In the coming years, the drug development process is likely to change dramatically as manufacturers, regulators, and payers across the world face intense pressure to meet the growing needs of their constituents. Higher hurdles in the regulatory and pricing/reimbursement landscape will likely present new challenges to the drug development and market access process, which will in turn impact patient care. Manufacturers of medicines must focus on "intelligent innovation" in which investments are targeted to pursue true therapeutic breakthroughs, to minimize the risk of failure, and to maximize global market access and patient benefit. The future of the medicines landscape must evolve toward a more collaborative framework, where regulatory agencies will pursue a greater degree of convergence and where sponsors, regulators, and payers, as essential stakeholders, will undertake drug development with reimbursement in mind to achieve the common global goal of bringing safe, effective, and affordable medicines to the world's people.

The Clinical Trials Transformation Initiative (CTTI).

The Clinical Trials Transformation Initiative (CTTI) is a public-private partnership created in 2007 between the United States Food and Drug Administration (FDA) and Duke University for the purpose of identifying practices that will increase the quality and efficiency of clinical trials. The initiative was generated from the realization that the clinical trials system in the United States has been suffering as a result of increasingly longer study start-up times, slowing enrollment of patients into trials, increasing clinical trial costs, and declining investigator interest in participating in clinical trials. Although CTTI was created to address a crisis for US clinical research, it seeks to identify practice improvements that can be applied internationally, and is therefore engaging international collaborators with international efforts that have similar objectives. CTTI's approach is to involve all sectors in the selection, conduct, and interpretation of its projects; to keep the dialogue open across sectors; to provide evidence that can influence regulatory guidance, and to attempt to create a "level playing field" when recommending change. The hope is that a broad and diverse data-driven discussion of the important issues in clinical trials will lead to meaningful change for the benefit of all concerned, and importantly for patients.