New models support comparative research, more nonprescription drugs


The drive for personalized medicine and greater patient involvement in treatment decisions demands more informative data on the effects and risks associated with drugs and medical products.

The drive for personalized medicine and greater patient involvement in treatment decisions demands more informative data on the effects and risks associated with drugs and medical products. FDA seeks to facilitate informed self-treatment with a proposal for expanding access to more over-the-counter (OTC) drugs with additional "conditions for safe use." Government support for comparative effectiveness research (CER) is attracting great interest from researchers, payers, and pharmaceutical companies looking to identify optimal treatment for a broad range of medical conditions. The clamor for more information on drug use and effects in such "real-world" situations is shaping clinical research and coverage decisions.


FDA officials propose to expand patient access to more nonprescription drugs, including treatments for chronic conditions such as high blood pressure and cholesterol, based on strategies that can help consumers select and use medicines appropriately without a doctor visit. This "new paradigm," as described by agency officials at a 2-day public hearing in March, aims to improve public health by making it easier for patients to obtain medicines with the assistance of innovative health information systems and pharmacy services that support self-treatment.

Pharmacists are enthusiastic about this more flexible regulatory approach as a way to expand their role in improving public health. Pharmacists working for health systems, moreover, consider themselves well-positioned to manage special safe-use programs. Under FDA's new paradigm, manufacturers would ensure "safe use" of a medication by utilizing new information technology (IT) and more home-use diagnostics to enhance and document individual understanding of appropriate treatment. Information kiosks and other IT aides in pharmacies could direct consumers through self-diagnosis information programs that check for risk factors contraindicating treatment and for tests needed to monitor continued safe use. At a minimum, "rescue medicines" such as asthma inhalers and epinephrine to block allergic reactions would be candidates for enhanced pharmacy access.

Vocal opposition to this approach comes from the American Medical Association (AMA), which insists that physician involvement in patient care and treatment is vital, especially for patients with chronic diseases. Health plans and payers, however, would save money because most insurers, employers, and government health programs do not reimburse for nonprescription medicines. Individuals with drug coverage would have higher out-of-pocket costs, a development that the AMA claims would deter adherence to therapy.

A policy that permits FDA to approve OTC products with added safe use requirements would require a lengthy FDA rule-making process and most likely authorizing legislation. It also would involve further development of label comprehension studies and actual use trials able to document that the medicine can be used safely and appropriately based on the approved "drugs facts box" label, plus supporting information and services. IT and research firms are ready with a range of technology to support these efforts, including new ways to evaluate and ensure compliance with self-diagnostic tools, to devise collaborative programs that enhance care management, and to coordinate follow-up and oversight in collaboration with physicians, payers, and patients.

An interesting side issue is whether enhanced access to nonprescription medicines could defuse the current hostilities over contraceptive coverage. If consumers pay for OTC oral contraceptives out-of-pocket, the debate over whether religious-affiliated organizations should cover birth control pills is no longer an issue. Of course, any move by FDA to authorize nonprescription contraceptives would launch a wave of objections from physicians and family planning opponents-similar to what happened last December when FDA proposed to drop OTC limitations for the Plan B morning-after pill.


Another way to help patients and providers make more informed decisions about treatment options – and to save money in the process by curbing inappropriate care-is by funding studies to evaluate effective patient treatment strategies and comparative study methods. The Patient Centered Outcomes Research Institute (PCORI) is poised to distribute approximately $120 million in coming months -and nearly $400 million in 2013-to launch a federal CER initiative, as authorized by the Accountable Care Act. It builds on other policies that already provide formulary committees, insurers, and payers with more comparative and outcomes analyses designed to assess the value, as well as benefits and risks, of a range of healthcare treatments, including prescription drugs.

PCORI has spent the last year getting organized, hammering out definitions for PCOR and CER, and seeking advice from all corners of the healthcare and research community. In April it finalized priorities for its research agenda with an eye to awarding initial grants this fall. About $35 million will support studies that identify medical products and practices with superior patient outcomes for prevention, diagnosis, and treatment. Additional funds will support evaluations for improving healthcare systems and for standards for patient-centered research.

The importance to payers and researchers of capturing real-world data in CER studies has generated considerable discussion about the design of studies to demonstrate comparability and added value of pharmaceuticals. Payers and patient advocates emphasized at a PCORI stakeholder "dialogue" in February that CER must use rigorous science and research methods. Employers highlighted the importance of assessing common chronic conditions that consume a sizeable portion of healthcare outlays, and patient advocates stressed the need to consider how treatments for less common critical diseases may affect individuals differently. Advocates for patients with specific diseases regard subgroup analysis as key to avoiding one-size-fits-all coverage decisions that often reject new technology.

These developments are prompting pharmaceutical companies to address CER earlier in the drug development process, to engage stakeholders in clinical trial design activities, and to explore innovative approaches for generating evidence, said Eleanor Perfetto, senior director for reimbursement and regulatory affairs at Pfizer, at an April CER summit sponsored by ExL Pharma. Similarly, experts at another CER conference sponsored by the Drug Information Association (DIA) and the National Pharmaceutical Council (NPC) in March discussed the need to assess comorbidities, patient biology, and other characteristics likely to affect response. Peter Neumann, professor of medicine at Tufts Medical Center, advised researchers and pharmaceutical companies to carefully choose comparators, outcomes, and subgroups to study, while also analyzing which health and payment systems support innovative approaches.

Naomi Aronson, executive director of the BlueCross BlueShield Technology Evaluation Center, was skeptical that CER will greatly improve the evidence base for health plan decision-making because most outcomes studies fail to measure overall survival. And even if a pharma company can document that some patients do better on a new drug, she said at the April CER summit, if the therapy is too expensive, the healthcare system still won't be able to afford it.

These issues are increasingly important for insurers and health plans looking to incorporate CER into new reimbursement models. In addition to drug price, plans now are considering whether a new therapy demonstrates improvements over existing treatment and if clinicians will follow guidelines for appropriate use. Payers are exploring how well case management, bundled payments, and risk-sharing models can inform coverage decisions.

A broader concern is whether larger and more costly CER studies will curb biopharmaceutical innovation. While more efficient study designs could reduce research costs, clinical trials with multiple subgroups, active comparators, and long-term end points can make drug development more expensive, pointed out NPC research director Jennifer Graff at the April CER summit. Innovative payment policies are equally important for encouraging early adoption of new products and medical procedures that can spur innovation.

To reduce costs and risks, pharma companies are forming partnerships with health plans and pharmacy benefit managers able to supply patient medical record information. A Pfizer-Medco partnership, Perfetto noted, aims to utilize genomic and phenotypic data to design studies that match patients to treatments most likely to provide benefit. Similar collaborations are evaluating a range of real-world treatment strategies for analyzing patient subgroups and assessing specific diseases.

These and other research initiatives highlight PCORI's important role in setting standards for CER analysis and in coordinating CER activities to avoid redundancies. Dissemination of CER results also is critical to ensure that clinicians incorporate research findings into practice. PCORI will support communications activities by the Agency for Healthcare Research and Quality (AHRQ), which has become an important part of the process in recent years. An AHRQ symposium this month will discuss further how research methods can shape the results of randomized trials and observational studies.

Ms Wechsler is a Washington-based reporter specializing in federal and state healthcare issues.

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