Wednesday, August 4, 2010

Thanks! PharmaVOICE 100 Most Inspiring in the Life Sciences

Very appreciative to be listed among the 100 Most Inspiring in the Life Sciences at PharmaVOICE. They have a pretty diverse line-up, from CEOs to R&D to marketing and more.

If you have known me for years but are still not exactly sure what I do (or why I do it), it's a nice write-up available online here (the kind that would make my grandmother proud!).

Thank you PharmaVOICE!

Friday, July 23, 2010

ePatients and Clinical Trials

Cross-posted from Pfizer's Think Science Now

The rise of the e-patient brings great opportunities — and some unique challenges — to clinical research and the conduct of clinical trials.

Rise of the e-Patient

Much has been written elsewhere about the rise of the e-patient. The white paper “e-Patients: How They Can Help Us Heal Healthcare” defines the e-patient as “a new breed of informed health consumer, using the Internet to gather information about a medical condition of particular interest to them.” Tom Ferguson MD, lead author of the e-patient white paper, described the e-patient as “empowered, engaged, equipped and enabled.”

Some key points about the e-patient:

e-Patients Are Everywhere

According to Pew Research Center’s Internet & American Life Project:

  • 61% of American adults look online for health information (the “e-patient”)
  • 60% of e-patients say they or someone they know has been helped by following medical advice or health information found on the internet (just 3% say they or someone they know was harmed)

e-Patients Are Organized

Last year The Society for Participatory Medicine was formed to organize a field defined as “a movement in which networked patients shift from being mere passengers to responsible drivers of their health, and in which providers encourage and value them as full partners.”

e-Patients Are Not a Fad

Earlier this year, the National Library of Medicine hosted their 2010 conference “The ePatient: Digital and Genomic Technologies for Personalized Health Care”. Leaders from academia and government agencies gathered at the National Institutes of Health (NIH) to discuss the implications of genomic, digital and wireless technologies – increasingly patient-facing tools – in the context of comparative effectiveness, innovation and personalized medicine.

In many ways, the e-patient is the future. Their rise brings disruptive change in the delivery of healthcare, and brings similar implications for clinical trials.

e-Patients Are Organizing Around Clinical Research

Today, many patients participate in a variety of online communities. As the New York Times noted earlier this year (“When Patients Meet Online, Are There Side Effects?”), in some of these communities patients have moved beyond chatting or posting free text. Patients are increasingly sharing structured, longitudinal health information with one another. This structured information is databased and used for searching, but is also aggregated and available for research.

In these communities patients are essentially initiating their own research (prompting buzzwords such as Research 2.0 and Crowdsourced Research). They are filling out forms that look very much like the case report forms (CRFs) used in academic clinical research studies. But the patients are not waiting for academics or industry to take interest in their disease – they are self-organizing, collecting data, and analyzing the resulting database. In some cases, these communities have even published results in peer-reviewed journals.

We may be seeing the tip of the iceberg in patient communities publishing scientific findings. Earlier this month, one organization published a paper entitled “Web-Based, Participant-Driven Studies Yield Novel Genetic Associations for Common Traits” in PLoS Genetics. The study was described as a novel participant-driven research program, building a community of patients sharing both self-reported clinical data and genetic information.

Patients sharing data in a community are conducting a form of clinical research, but this should not be confused with a clinical trial. Clinical trials involve prospectively defined protocols (including eligibility criteria and other rules) and participants are often randomly assigned to one of several treatment options. Data generated by patient communities are an important step in the continuum of evidence and knowledge, but may be difficult to compare to a randomized clinical trial.

e-Patients: Opportunities (and Challenges) for Clinical Trials

While e-patients themselves are gathering in communities and may be generating their own research findings, they also bring potential opportunities and challenges for traditional clinical trials.

Patient community initiated research is different from a clinical trial (as noted above), but the roles and tools may be very similar:

  1. Both rely on an engaged patient as a participant
  2. Both rely on the participant being interested in self-tracking and reporting
  3. Both rely on the participant being willing to share data

#1. The engaged e-patient as a clinical trial participant

The most often-cited opportunity with e-patients in clinical trials is around engagement – reaching these patients, informing them about clinical trials, recruiting them to participate. There are many companies offering services in this space, and a seemingly endless number of conferences on the topic. As an example, Pfizer has a website to help patients match for trials, is using YouTube to share information with potential participants, and is building an online community to increase awareness and connect patients to investigators while ensuring privacy. Certainly online patient engagement is one tool to reach patients in support of trial recruitment. But this will not be a silver bullet, and should be considered one of many tools used to execute a recruitment strategy.

#2. The participant self-tracking (quantified-self)

There is an exciting trend in healthcare described as ‘quantified self’ – well described in Wired magazine and online at characterized by individuals who self-track a range of information about themselves (often in the context of measuring their health and wellness). Many of the tools these self-trackers are using begin to resemble the electronic patient reported outcomes (ePRO) or electronic diaries we look to use in clinical trials, just requiring validation.

#3. Participants sharing their health data

Similar to patients sharing data in online communities, clinical trials have always been dependent upon the participant being willing to share data to improve scientific knowledge and public health, often without any direct benefit conferred back to the participant. Today’s e-patient, however, is highly engaged and informed in their health, appreciating their role as owner of their own health data.

Understand the Risks

The e-patient who advocates being an informed participant in their healthcare, may not be comfortable with many traits of a randomized and blinded clinical trial. This raises unique challenges:

  • How can we keep the e-patient engaged in a clinical trial if we are unable to share traditional “participatory” information (or even their treatment assignment)?
  • What happens when the e-patient is engaged with communities of patients online? Are they sharing information that may provide clues to treatment assignment? Are they sharing suspected adverse events (that may create a cascade of other participants reporting symptoms)? How does the pharmaceutical sponsor educate the study participants, or monitor or engage in this conversation?
  • Is the e-patient more likely to show a response to placebo (because they are more highly engaged in their health)?

I have seen online patient communities with threads dedicated to discussing participating in specific studies. Some of these discussions have included:

  • Coaching on how to adjust certain subjective tests to meet eligibility criteria
  • Sharing potential symptoms that may indicate who is on active drug (vs. placebo)

The e-patients in these instances are doing what they do best – sharing information with one another. But the scientific implications for the study can include enrolling ineligible patients, unblinding, and influencing adverse event reporting rates. Disrupting the ability to answer important questions is not in the interests of the study sponsor, investigator or the participant.

New Opportunities, New Horizons

Moving forward we cannot ignore the role of the e-patient in a clinical trial.

Some questions to consider:

  • How can we participate in the online discussions and communities to answer questions about studies? Can we educate participants about online conversations that may impact the scientific integrity of the study?
  • Are there new opportunities for engagement with the e-patient that can help to mitigate some of the sacrifice they are making serving as a blinded participant? Specifically:
    • Can we bridge the gap between the tools a self-tracking e-patient (“quantified self”) is using and the validated tools we are using in clinical trials?
    • How can we best share the learnings from a clinical trial back with the participant?

There are clearly more questions than answers. But there are incremental solutions underway. One example is Pfizer’s work with the Center for Information & Study on Clinical Research Participation (CISCRP). Working together, Pfizer and CISCRP recently demonstrated a new way to share clinical research learnings and results with participants. This should be just the beginning of a new relationship between participants and clinical trials.

Wednesday, June 30, 2010

Keeping Pace with Electronic Health Records to Improve Clinical Research

Cross-posted from Pfizer's Think Science Now

Electronic health records (EHRs) have gained a great deal of attention in the United States, with financial incentives under the American Recovery and Reinvestment Act and expectations for quality and cost factored into healthcare reform.

But what about the potential for electronic health records to impact clinical research? How may EHRs impact our ability to match patients for trials, select optimal investigator sites, or even test the feasibility of our study protocols?

Many have seen great opportunity to improve clinical research by accessing these emerging databases. But they have been challenged by large amounts of data coming from disconnected systems each with limited access. Concerns have also surrounded how to ensure privacy.

Now Pfizer has joined with other leading research centers, pharmaceutical companies, and health information technology organizations to announce the formation of the Partnership to Advance Clinical electronic Research (PACeR).

This is another in a growing list of examples of pharmaceutical companies working together to address common challenges – PACeR includes Pfizer along with Johnson & Johnson and Merck. They are also joined by Quintiles, a contract research organization.

PACeR is about health data improving research, and so the partnership includes the Healthcare Assocation of New York State (HANYS) along with a dozen leading New York hospitals and medical centers. To ensure patient benefit while maintaining patient protection (including privacy), the partnership includes the Hastings Center and the Legal Action Committee. Additional PACeR partners include FDA along with CDISC and HL-7, the former to provide regulatory guidance and the latter two focused on sharing data and interoperability.

One of my colleagues at Pfizer – David Leventhal (Director of Health Informatics) has been instrumental in executing this vision and assembling this consortium. From David:

“Clinical research is one of the most critical aspects of getting innovative medicines to patients. It has been extremely challenging to conduct high quality clinical research on a timely basis. We see the PACeR initiative as a unique collaborative opportunity to move beyond talking about the challenge, and to fundamentally change the way clinical research is conducted. This is an effort and outcome where everyone wins.”

PACeR is a great example of working across organizations and institutions to create sustainable new models to improve clinical trial efficiency.

Emphasizing the potential impact across clinical research, Leonard Sacks, MD (Acting Director for the FDA Office of Critical Path) commented:

“In tackling issues such as the role of electronic health records in clinical research, the potential to personalize medicine using bioinformatics, and the safeguarding of medical privacy, HANYS’ Partnership to Accelerate Clinical Electronic Research has embraced the task and we enthusiastically support their effort.”

Monday, June 28, 2010

The Path to Personalized Medicine [NEJM]

Cross-posted from Pfizer's Think Science Now

NEJM: The Path to Personalized Medicine

My colleague in Regulatory Affairs, Dr. Sharada Truter, was good enough to share the following regarding a Perspective published last week in the New England Journal of Medicine:

FDA Commissioner Margaret Hamburg and NIH Director Francis Collins write that the initiatives will support the advancement of personalized medicine through rapid development, review and approval of safe and effective genetics-based disease therapies.

More specifically, the NIH initiative calls for the agency to invest in research that will support scientists’ efforts to translate genetic-marker discoveries into effective diagnostic tests and disease therapies, while the FDA’s program will work to develop an effective and timely pathway for the review and approval of new gene-therapy drugs.

The full-text of the NEJM article is available at this link.

Friday, June 18, 2010

FDA Commissioner's Keynote at DIA Annual Meeting

Cross-posted from Pfizer's Think Science Now

Earlier this week FDA Commissioner Dr. Margaret Hamburg delivered the keynote address at the 46th Annual Meeting of the Drug Information Association (DIA) in Washington DC.

Dr. Hamburg focused on two areas -- the need to strengthen FDA's regulatory sciences and the need for FDA to manage safety on a global basis.

Hamburg described regulatory sciences as "the critical bridge between biomedical research and the people who need those products." She noted how science and R&D are rapidly evolving with innovative products, citing examples such as stem cells, an artificial pancreas, and combination therapies. But she raised concerns about the agency's ability to keep pace in evaluating the safety and efficacy of these innovations. Hamburg called for academia, industry, and government to embrace the field of regulatory sciences noting that "We need new approaches. The truth is, this is not a task we can approach on our own."

She then expanded on the need to operate globally, noting the volume of food, drugs and devices being imported from abroad. Solutions from FDA include collaborations among the global regulatory community, harmonizing global regulatory requirements, and more frequent foreign inspections.

While on the topic of DIA, I would like to thank the organization for awarding me this year's Outstanding Service Award. DIA is an organization of over 18,000 drug development professionals from 80 countries, providing a neutral forum for sharing knowledge. I have supported DIA in a number of ways over the past decade, and have appreciated the opportunity to network with the global drug development community to test and share new ideas, improve how we do our work and ultimately impact human health.

(And while moving into tangents -- For those who follow me on Twitter it is true that I gave up my seat for the Chairman of the Federal Reserve on the Acela from Washington back to New York. Sitting in front of Mr. Bernanke for the 2 1/2 hour train ride, he quietly read his Kindle and failed to share any tips other than noting "There are no perks that come with my job!")

Public Release of Alzheimer’s Clinical Trial Data by Major Pharmaceutical Researchers

Cross-posted from Pfizer's Think Science Now

Today Pfizer along with other major pharmaceutical researchers announced a unique data sharing agreement for Alzheimer’s Disease (press release here, and media coverage here). This is another exciting example of collaboration among pharmaceutical companies to advance science and address unmet medical need.

Data from over 4,000 Alzheimer’s patients that participated in 11 industry-sponsored trials will be made available to researchers around the world (patient identifiers are removed to protect individual privacy). Each company will also use common data standards from CDISC to ease data sharing (a step that will also improve FDA review time).

Is this the beginning of a new era of collaboration to make progress against some of the toughest diseases?

Tuesday, June 8, 2010

As ASCO Comes To A Close, Special Thanks To The Medical Heroes

Cross-posted from Pfizer's Think Science Now

Today marks the final day of the 2010 Annual Meeting of the American Society of Clinical Oncology (ASCO). Meetings such as ASCO are an important gathering place for researchers, clinicians, and patients to share the latest data on new treatments. With high unmet medical need remaining for cancer patients, a great deal of energy and hope hovers over ASCO.

At this year’s meeting, Pfizer and other pharmaceutical companies shared data from experimental drugs and biologics for cancer of the lung, brain, and more.

This progress is a result of the dedication of researchers at Pfizer and our partners from academia and other companies, working side-by-side with physicians serving as investigators to understand how these new treatments work in their patients.

But a special thanks must also go to the patients who volunteer as participants in our cancer clinical trials.

We know that it takes partnerships to address diseases as important as cancer. Sometimes these are Partnerships of drug companies working together, such as the collaboration in cancer research between Pfizer, Merck, and Lilly announced earlier this year.

It also takes partnerships with patients. This is why we sponsor organizations such as CISCRP (The Center for Information & Study on Clinical Research Participation) a non-profit created to inform the public about clinical research. CISCRP created the Medical Heroes program to honor and thank those who have given by taking part in clinical research.

(We also have a partnership underway with Private Access to help inform patients about clinical trials; more on that later.)

Pfizer has over 100 cancer clinical trials underway. According to PhRMA, our industry has over 800 medicines and vaccines in testing against cancer. But all of this progress will stop if not for patients serving as participants – and Medical Heroes.

Information about clinical trial participation can be found at websites hosted by FDA and NIH.

To help find a trial you can contact the Pfizer Oncology Clinical Trial Information Service by phone (877-369-5793) or online ( The service is free and confidential.