The roles of patents and research and development incentives in biopharmaceutical innovation.

Patents and other forms of intellectual property protection play essential roles in encouraging innovation in biopharmaceuticals. As part of the "21st Century Cures" initiative, Congress is reviewing the policy mechanisms designed to accelerate the discovery, development, and delivery of new treatments. Debate continues about how best to balance patent and intellectual property incentives to encourage innovation, on the one hand, and generic utilization and price competition, on the other hand.

The evolution of biotechnology and its impact on health care.

For more than three decades the field of biotechnology has had an extraordinary impact on science, health care, law, the regulatory environment, and business. During this time more than 260 novel biotechnology products were approved for over 230 indications. Global sales of these products exceeded $175 billion in 2013 and have helped sustain a vibrant life sciences sector that includes more than 4,600 biotech companies worldwide.

Complementary versus companion diagnostics: apples and oranges?

There have been several major problems that have plagued biopharmaceutical development since the end of the 1990s, but two in particular have reached the point where they are impacting the economic viability of the industry: the lack of efficacy of new drugs and increasing competition among therapeutics that broadly attack certain common diseases and disease areas. The US FDA has noted that the era of one-size-fits-all treatment may well be reaching its end days as companies increasingly adopt approaches that involve biomarkers (there are now commercial databases that purport to track over 11,000 of them). Pharmacogenomic biomarkers in particular are used to create diagnostics that help to differentiate or stratify the likely outcomes a patient will experience with a drug, which can now be said to be targeted or tailored to patients with particular traits (i.

Personalized Medicine's Bottleneck: Diagnostic Test Evidence and Reimbursement.

Background: Personalized medicine is gradually emerging as a transformative field. Thus far, seven co-developed drug-diagnostic combinations have been approved and several dozen post-hoc drug-diagnostic combinations (diagnostic approved after the drug). However, barriers remain, particularly with respect to reimbursement.

Taking the pulse of strategic outsourcing relationships.

Purpose: Articles in peer-reviewed journals and the trade press presuppose that strategic outsourcing relationships have been formed to replace preexisting collaborative approaches with contract research organizations. They do not consider that large, fragmented pharmaceutical and biotechnology companies may be supporting competing and conflicting relationship models simultaneously. A recent Tufts Center for the Study of Drug Development study quantifies actual strategic outsourcing practices among drug development companies and sheds new light on why these relationships may be failing.

Site Characteristics Influencing the Translation of Clinical Research Into Clinical Practice.

Investigative sites participating in clinical trials play an instrumental role in aiding market adoption. Site experiences in clinical research help physician investigators and research professionals gain familiarity with and exposure to investigational treatments. This knowledge may be passed on to sponsor companies and may ultimately assist in positioning new products and driving commercialization success.

The Impact of Collaborative and Risk-Sharing Innovation Approaches on Clinical and Regulatory Cycle Times.

During the past decade, high risk, cost, and inefficiency have driven pharmaceutical and biotechnology companies to enter into collaborative and shared innovation approaches, including mergers and acquisitions, joint development, and in-licensing. These approaches can interrupt the drug development process and affect program-level clinical and regulatory cycle times. To examine these potential impacts, detailed development histories were obtained for 289 new molecular and biologics entities that received FDA approval between 2000 and 2011.

Measuring progress in neglected disease drug development.

Background: Since the late 1990s, funding for development of neglected disease drugs has increased with an influx of resources from product development partnerships (PDPs). Previous research showed modest gains in drug approvals and products in Phase III of clinical development in the period 2000-2008.

Objective: We assessed the 2009-2013 period in terms of numbers of products in Phase III development and numbers of approvals.

The pediatric studies initiative: after 15 years have we reached the limits of the law?

Background: Despite considerable disincentives for conducting drug studies in children, 15 years ago the Food and Drug Administration, pediatric health advocates and congressional sponsors created a carrot-and-stick policy approach of voluntary and mandatory programs to encourage the pharmaceutical industry to include children in the drug development process. After several rounds of reauthorization of the laws on a temporary basis, the enabling statutes have been made permanent.

Objective: The purpose of this analysis is to review the advances that resulted from the law and the areas where further progress is needed.

The biotechnology innovation machine: a source of intelligent biopharmaceuticals for the pharma industry--mapping biotechnology's success.

The marriage of biotechnology and the pharmaceutical industry (pharma) is predicated on an evolution in technology and product innovation. It has come as a result of advances in both the science and the business practices of the biotechnology sector in the past 30 years. Biotechnology products can be thought of as "intelligent pharmaceuticals," in that they often provide novel mechanisms of action, new approaches to disease control, higher clinical success rates, improved patient care, extended patent protection, and a significant likelihood of reimbursement.

Are payers treating orphan drugs differently?

Background: Some orphan drugs can cost hundreds of thousands of dollars annually per patient. As a result, payer sensitivity to the cost of orphan drugs is rising, particularly in light of increased numbers of new launches in recent years. In this article, we examine payer coverage in the United States, England and Wales, and the Netherlands of outpatient orphan drugs approved between 1983 and 2012, as well as the 11 most expensive orphan drugs.

Economic impact of a triptan Rx-to-OTC switch in six EU countries.

Introduction: Triptans have been safely and effectively used in the management of migraine for more than fifteen years, and it seems reasonable to wonder what would be the economic impact of moving a specific triptan to OTC availability. The objective of this study was then to examine the economic impact of payer policies of a triptan Rx-to-OTC switch in six EU countries (France, UK, Spain, Italy, Germany and Poland).

Methods: A decision model was used to model the budgetary impact of a triptan Rx-to-OTC switch from the third-party payer (TPP) and the societal perspectives, using a one-year timeframe.

New Governance Mechanisms to Optimize Protocol Design.

Pharmaceutical and biotechnology companies are actively seeking ways to optimize protocol design. An emerging approach has been the creation of a new governance mechanism designed to evaluate protocol feasibility and simplify design before final protocol approval. In late 2012, the Tufts Center for the Study of Drug Development conducted a qualitative in-depth assessment of 10 major pharmaceutical companies that have implemented these new governance mechanisms since 2009.

A proposal for integrated efficacy-to-effectiveness (E2E) clinical trials.

We propose an "efficacy-to-effectiveness" (E2E) clinical trial design, in which an effectiveness trial would commence seamlessly upon completion of the efficacy trial. Efficacy trials use inclusion/exclusion criteria to produce relatively homogeneous samples of participants with the target condition, conducted in settings that foster adherence to rigorous clinical protocols. Effectiveness trials use inclusion/exclusion criteria that generate heterogeneous samples that are more similar to the general patient spectrum, conducted in more varied settings, with protocols that approximate typical clinical care.

Prospects for rapid advances in the development of new medicines for special medical needs.

Broadly speaking, the goals of the US Food and Drug Administration (FDA) special-designation programs--orphan, priority review, accelerated approval, and fast track--have been to expedite and sustain development and facilitate authorization of new medicines for unmet medical needs through so-called "push-pull" incentives. Although generally successful over time, their success has been confined to certain therapeutic areas and, within those areas, certain diseases. Times have changed.

Clinical approval success rates for investigational cancer drugs.

We examined development risks for new cancer drugs. For the full study period, the estimated clinical approval success rate for cancer compounds was 13.4% (9.

Innovating by developing new uses of already-approved drugs: trends in the marketing approval of supplemental indications.

Background: Much of the literature on trends and factors affecting biopharmaceutical innovation has focused overwhelmingly on the development and approval of never-before approved drugs and biologics. Little attention has been paid to new uses for already-approved compounds, which can be an important form of innovation.

Objective: This paper aimed to determine and analyze recent trends in the number and type of new or modified US indication approvals for drugs and biologics.

Impact of In-Pharmacy Education on Patients' Knowledge and Attitudes About Clinical Trials.

The lack of public awareness and understanding about clinical research has long hindered the efficiency and speed in recruiting patients to participate in clinical trials. Earlier research conducted by the Center for Information and Study on Clinical Research Participation (CISCRP) examined whether pharmacists might be a viable channel to educate and engage the public about clinical research, and the results suggested that pharmacy-directed outreach and education are feasible. This study measured the impact of in-pharmacy education on patient comprehension and willingness to participate in clinical research.

Assessing the economic impact of Rx-to-OTC switches: systematic review and guidelines for future development.

Introduction: Switching drugs from prescription to non-prescription status (Rx-to-OTC) presents a unique set of challenges and opportunities to policy-makers and the industry in terms of managing health outcomes, pharmaceutical spending, and steering of consumer choices of therapy. Decision-analytic models are used to address uncertainty and produce reasonable estimates of the economic impact of switches for payers. This article presents a critical literature review of existing models which assess the economic impact of Rx-to-OTC switches, and provides guidelines in which future economic evaluations of Rx-to-OTC switches could be improved.

Pharmaceutical R&D performance by firm size: approval success rates and economic returns.

The R&D productivity of pharmaceutical firms has become an increasingly significant concern of industry, regulators, and policymakers. To address an important aspect of R&D performance, public and private data sources were used to estimate clinical phase transition and clinical approval probabilities for the pipelines of the 50 largest pharmaceutical firms (by sales) by 3 firms size groups (top 10 firms, top 11-20 firms, and top 21-50 firms). For self-originated compounds, the clinical approval success rates were 14.

Benchmarking the Study Initiation Process.

The study start-up phase of a trial is an area that pharmaceutical and biotechnology companies are focusing on in order to reduce delays and improve efficiency. To better understand and examine metrics within study start-up, the Tufts Center for the Study of Drug Development, in collaboration with 11 pharmaceutical and biotechnology companies, examined a comprehensive set of metrics and analyzed study data from 105 global clinical trials. The results indicate that the early stages of the site initiation process are areas that accounted for the majority of cycle time.

Translational research and the evolving landscape for biomedical innovation.

This article addresses current challenges facing pharmaceutical and biopharmaceutical developers, including the expiration of patents on many high-revenue-generating products, increasing competition in the marketplace, low public support, high regulatory hurdles, and the increasing time, cost, and risk of new product development. To meet these challenges, drug developers are looking to new models of innovation to improve efficiency, lower risk, and increase output. These new models include codevelopment agreements with small companies, multicompany consortia, and strategic partnerships with academic research centers.

Evaluating the impact of Medicare Part D on quality metrics.

Subsidized access to medical and pharmacy benefits is not a goal in and of itself; it is a means toward an end (improved health outcomes). Accordingly, the addition of an outpatient prescription drug benefit (Part D) to Medicare in 2006 will be deemed a success if it provides better, more affordable access to outpatient prescription drugs for Medicare beneficiaries and, more importantly, improves drug adherence and health outcomes, together with reducing or at least bending the cost curve by offsetting certain healthcare costs such as hospitalizations and emergency room visits. Priest and colleagues examine these claims and find suboptimal outcomes despite improved access.