Category Archives: health care
Johns Hopkins is launching a new Center for Drug Safety and Effectiveness. Via G. Caleb
On Wednesday, October 24, we will launch the Johns Hopkins Center for Drug Safety and Effectiveness, a collaborative effort of the Bloomberg School of Public Health and Johns Hopkins Medicine. The Center will fulfill its mission by supporting individuals engaged in research, training, clinical programs and public service to optimize the safe and effective use of prescription medicines in the United States and around the world.
We are delighted that Dr. Mark McClellan will deliver the inaugural lecture for the Center on October 24 at 4:00 PM, with a reception to follow.
The Patient-Centered Outcomes Research Institute’s mission is to fund health research that offers patients and caregivers the information they need to make medical decisions. The PCORI Board of Governors has adopted the following working definition of “patient-centered outcomes research.”
Patient-Centered Outcomes Research (PCOR) helps people and their caregivers communicate and make informed health care decisions, allowing their voices to be heard in assessing the value of health care options. This research answers patient-centered questions such as:
- “Given my personal characteristics, conditions and preferences, what should I expect will happen to me?”
- “What are my options and what are the potential benefits and harms of those options?”
- “What can I do to improve the outcomes that are most important to me?”
- “How can clinicians and the care delivery systems they work in help me make the best decisions about my health and healthcare?”
To answer these questions, PCOR:
- Assesses the benefits and harms of preventive, diagnostic, therapeutic, palliative, or health delivery system interventions to inform decision making, highlighting comparisons and outcomes that matter to people;
- Is inclusive of an individual’s preferences, autonomy and needs, focusing on outcomes that people notice and care about such as survival, function, symptoms, and health related quality of life;
- Incorporates a wide variety of settings and diversity of participants to address individual differences and barriers to implementation and dissemination; and
- Investigates (or may investigate) optimizing outcomes while addressing burden to individuals, availability of services, technology, and personnel, and other stakeholder perspectives.
Listen to this inspirational speech by PCORI Board of Governors member Harlan Krumholz, at the PCORI National Patient and Stakeholder Dialogue, February 27, 2012:
In the USA at least, the data legally belong to trialists on the grounds that it requires work to create knowledge from data. But science, particularly medical science, is essentially an enterprise conducted for moral reasons. We need to do not just what is legal but what is right. As such, we must take into account the probable wishes of the patients who give us their blood, fill in our questionnaires and die on our trials. It is difficult to believe that any patient on my trial, who completed complex questionnaires so diligently over such a long period of time, would really have wanted me to keep the data for myself rather than share it with others for the benefit of medical science in general. Vickers AJ. Whose data set is it anyway? Sharing raw data from randomized trials. Trials. 2006;7:15.
Every day, patients and their caregivers are faced with difficult decisions about treatment. They turn to physicians and other healthcare professionals to interpret the medical evidence and assist them in making individualized decisions. Unfortunately, we are learning that what is published in the medical literature represents only a portion of the evidence that is relevant to the risks and benefits of available treatments. In a profession that seeks to rely on evidence, it is ironic that we tolerate a system that enables evidence to be outside of public view. Krumholz HM. Open Science and Data Sharing in Clinical Research: Basing Informed Decisions on the Totality of the Evidence. Circulation: Cardiovascular Quality and Outcomes. 2012;5: 141-142
We are all patients, and will all face questions about what medical treatments to pursue. Some questions are trivial and unimportant, others can mean the difference between life and death. We rely on evidence-based medicine to give us reliable information about the risks and benefits associated with medical interventions, but a disturbing amount of evidence indicates that the medical literature is not always reliable. Many clinical trials are not published within a reasonable time after completion or are never published at all. Missing data leads to systematic reviews that are based on only a portion of the trials that were conducted, which can affect the results in unknown and unpredictable ways. Missing data may in some cases hold important information about risk, as in the case of Vioxx (rofecoxib). Merck had data several years before Vioxx was withdrawn from the market that showed the drug increased the risk of heart attacks, but most of the data was unpublished and out of public view. In other cases, clinical trials are published but the data are reported in a misleading and biased way, as when a negative trial is presented so as to appear positive, or analyses showing harm are omitted.
What is to be done? What can we do to make evidence-based medicine more evidence-based? Four commentaries in the March 2012 issue of Circulation: Cardiovascular Quality and Outcomes discuss how making clinical research data available outside individual drug and device companies or research groups could greatly add to the depth and reliability of our knowledge. Currently, with certain exceptions, access to most clinical trial data is restricted to the investigators or the funders. Harlan Krumholz, in an editor’s perspective, outlines the key concepts:
Now is the time to bring data sharing and open science into the mainstream of clinical research, particularly with respect to trials that contain information about the risks and benefits of treatments in current use. This could be accomplished through the following steps:
Post, in the public domain, the study protocol for each published trial. The protocol should be comprehensive and include policies and procedures relevant to actions taken in the trial.
Develop mechanisms for those who own trial data to share their raw data and individual patient data.
Encourage industry to commit to place all its clinical research data relevant to approved products in the public domain. This action would acknowledge that the privilege of selling products is accompanied by a responsibility to share all the clinical research data relevant to the products’ benefits and harms.
Develop a culture within academics that values data sharing and open science. After a period in which the original investigators can complete their funded studies, the data should be de-identified and made available for investigators globally.
Identify, within all systematic reviews, trials that are not published, using sources such as clinicaltrials.gov and regulatory postings to determine what is missing.
It must be acknowledged that there are many obstacles — political, cultural, financial — to accomplishing these goals. Some of these obstacles are discussed in the other three commentaries, which are open access and which I urge you to read:
Spertus, JA. The Double-Edged Sword of Open Access to Research Data. Circulation: Cardiovascular Quality and Outcomes. 2012;5;143-144.
Ross JS, Lehman R, Gross CP. The Importance of Clinical Trial Data Sharing: Toward More Open Science. Circulation: Cardiovascular Quality and Outcomes. 2012;5;238-240.
Gotzsche PC. Strengthening and Opening Up Health Research by Sharing Our Raw Data. Circulation: Cardiovascular Quality and Outcomes. 2012;5;236-237.
Whatever the difficulties, the current situation is clearly intolerable. Patients deserve reliable information on the risks and benefits of medical treatments and the subjects of clinical trials deserve that their contributions be fully used to benefit other patients.