Patient Thoughts on Medical Tests for Research

Here is a guest post I wrote earlier this year for METAvivor called “How Do People Feel about Bone Marrow Exams?” It was based on a study with Bonnie King from Stanford. While this may be one of the more gruesome-sounding medical procedures that some patients go through, it is not necessarily unique.

Too often, researchers think about all of the cool data they can collect during a clinical trial or research study, without thinking about what is would be like to experience all of those test procedures. Well, patients think about it, and often wonder what is wrong with the researchers!

I’m always perplexed when I hear about “adherence” issues in clinical trials. It used to be called “compliance” but that wasn’t as accurate, and brought up more negative connotations for the research community.

The fact is, for patients, endurance is the best term.

– Deborah Collyar

Staying in a clinical trial, or on any prolonged treatment plan, is an endurance test. There are many unpleasant, and sometimes risky, things that you have to do but hopefully you will get something out of it at the end. Patients hope for positive responses to treatment, or even remission, but that is not always possible.

x-ray film of the brain computed tomography

This is one of many reasons why it is important for those of us who represent patients to be involved in research discussions, from conceptual design to trial completion. We ask questions, such as, “Why do you need x number of these tests? Are they absolutely necessary to answer the questions in this study? What else could be done? Have you thought about asking the patient?”

Let’s work together to make the experience of participating in clinical trials as smooth as possible. Trial participants contribute so much already – they deserve to respect and consideration when we ask them to do things for research.

 

Patients Want Their Data Shared

Patients also want to be:

  • Respected for their contributions to science and medical advances. Those contributions include samples from their bodies (biospecimens), information (data), experiences (input), and sometimes their very lives.
  • Protected from harm and misuse of sensitive data about themselves, their family, and/or their culture or ethnicity.

As long as we are respected and protected, we will participate in clinical research and learning healthcare systems so researchers can find better ways to treat and prevent diseases and medical conditions.

What & Who

This post focuses on data from past “legacy” cancer clinical trials. The Patient AdvocateProtectionCommittee in the Alliance for Clinical Trials in Oncology drafted a resolution to add the patient voice to the groundswell of support for data sharing.

Why?

  1. People who join cancer clinical trials are often asked to donate their tissue and data for future research.
  2. When patients give consent for research, they expect their information to be shared.
  3. Unfortunately, many hospitals, clinics, academic centers, or research groups will not release information to other researchers, even though patients gave their consent.
  4. This does not honor or respect patients who want to contribute to research.

Your Call to Action

  • Please read the resolution, sign on, and share it with your networks and organizations. While this resolution deals with cancer, its intent is to help all medical conditions.
  • Include patients in data sharing activities.

“Today, oncologists and cancer researchers realize that they can’t [advance cancer progress] alone… What’s required today extends beyond any individual or any individual discipline, beyond medicine itself… It requires somewhat of a change in mindset. It requires a lot more openness – open data, open collaboration and above all, open minds.

– Vice President Joe Biden, ASCO Speech

Thank you!

How to Solve Diseases with Existing Drugs

My blog guests this week are Amy Conn, Bruce Bloom, and Clare Thibodeaux from Cures Within Reach. Disclosure: I am a member of their Advisory Board, and think that testing older drugs for rare diseases is brilliant!

The power of repurposing

What if the latest treatment for cancer, diabetes or thousands of other unsolved diseases was already available?

Although this might sound too good to be true, “repurposing” existing drugs could make this a reality. Repurposing takes any drug, device or nutriceutical, already approved in one disease, and tests it in a different disease to quickly and affordably improve more patients’ lives.

cures within reach infographicCurrently more than 500 million people worldwide suffer from diseases that lack effective treatments. As a result, global health care costs are growing and patients are suffering. Yet new drug discovery can take 10-15 years and can cost over $2 billion. This process is too long and costly for many patients who needs a solution today.

Instead, repurposing leverages prior investments by finding new uses for “old” drugs. This means repurposed treatments can reach patients in about 3 years and for less than $500,000.

Testing for repurposing sometimes means they are first tested in tissue samples or animal models in laboratories to find accurate dosing. If this pre-clinical research is done, the drug can quickly move into human trials. In some cases, it moves into human trials immediately.

How do repurposing ideas happen?

Repurposing is not a new concept. Doctors often prescribe medications ‘off-label’ when they think the patient may benefit. This happens when there is no approved treatment for a specific medical condition.

  • In fact, 1 in 5 prescriptions (21.3%) written in the United States are for off-label use. The range varies widely, based on the medical condition or patient group. For instance off-label use can be as high as 7 in 10 children for pediatric illnesses, because sponsors don’t typically focus on them. Here is a table with common off-label prescriptions.

Repurposing research can take off-label prescribing a step further by confirming clinical observations through a clinical trial. The data from these trials can then be published in scientific journals, and shared with other doctors to become part of standard treatment.

Other sources

  • Patients also contribute insights when they describe how a drug or supplement that they are taking for one issue impacts a different disease or diagnosis (i.e. “my asthma drug seems to be helping my eczema”).
  • Ideas also come from laboratory scientists, who connect the dots between newly discovered disease information and existing compounds.
  • And in our emerging world of big data, informatics engines can comb through scientific literature to find existing ideas and generate new ideas suitable for immediate testing.

Who pays for repurposing research?

Sometimes, pharmaceutical companies finance repurposing projects.

A case in point: ViagraViagra_in_Pack

Originally tested as a drug for angina, sildenafil failed. Instead, it was repositioned into the Pfizer blockbuster Viagra for erectile dysfunction after patients mentioned this side effect. Sildenafil was still under patent protection and the market was huge, making it a lucrative endeavor. This drug was later repurposed by Pfizer a second time in a different dosage under the name Revatio for pulmonary arterial hypertension.

Too many are orphans

Many repurposed opportunities are based on generic drugs that are often inexpensive and widely available. These characteristics make them ideal candidates for repurposing for patients, but industry doesn’t often pursue them, since there is no clear path to profit. This is where philanthropy plays an important role.

Cures Within Reach (CWR) is a global non-profit dedicated to repurposing research

CWR focuses on improving patient outcomes in any disease with repurposed treatments, while addressing larger needs around global collaboration, and the creation of alternative financial incentives for repurposing.

Successful repurposing

Key In Lock Showing Forbidden Information And Privacy

Since 2010, 13 repurposing projects that Cures Within Reach supported have made a clinical impact. One such project involved repurposing sirolimus, a generic drug originally used to reduce organ transplant rejection, to treat a rare and deadly childhood disease called autoimmune lymphoproliferative syndrome (ALPS).

Children with ALPS carry a genetic mutation that causes some of their white blood cells to multiply and crowd out other types of blood cells. They suffer from enlarged lymph nodes and spleens, increased infections and anemia. Some patients can spend up to 10 days a month in the hospital receiving treatment, placing physical, emotional and financial burdens on patients and their family.

CWR funded research that helped prove that sirolimus produced a lasting positive response in patients, leading to fewer hospitalizations, greatly lowered medical costs and improved quality of life. This result happened in 3 years for about $250,000. Sirolimus has since been repurposed in 5 more childhood autoimmune diseases with similar results.

Ready for more successes with CureAcceleratorTM

Philanthropic funding was critical to this high-impact repurposing research. There are many more opportunities like this one, in which a small “investment” can create a life-saving repurposed treatment.

To find important repurposing opportunities CWR asks:cure accelerator logo

  • How can we help repurposing researchers connect with strong funding streams?
  • Are there financial models other than traditional philanthropy that can help scale this research?

To answer these questions, CWR created an online crowd-sourced platform in 2015 called CureAccelerator.™ Check us out, and watch for another post soon about CureAccelerator.

Repurposing is an important healthcare strategy, both in terms of patient impact and cost savings. As scientists and clinicians learn more about the mechanisms and molecular targets of diseases, repurposing will play an even larger role. The key stakeholders in repurposing, from funders to researchers to patient advocates to industry, all need to work together to drive new “old” treatments to patients.

Learn more about repurposing drugs here.

Getting Real about Patients & Clinical Trials

I’ve been asked countless times to write about patients and clinical trials. Guess it’s time to share what I’ve learned, along with other patient advocates, as we work beside researchers in the proverbial trenches. Just so happens that this post coincides with April Fool’s Day – hope that’s not an omen!

Why this series?

This is the first in a series on Patients & Clinical Trials (CT). Why? Because clinical trials are really the only tried & true way we get new diagnostics, treatments, and preventive approaches to people. For all diseases and health conditions.

But the research system isn’t very good at it, even after 70+ years. It’s not for lack of trying – BILLIONs have been spent on tweaking existing structures, but perplexing problems persist.

Heads up on CT activities

Other themes will also emerge, based on over 20 years of changing research and medicine culture (or at least trying to). In the meantime, here are some of my activities in the CT world:

Event Inscription on Sign

Other activities:information-orange-circle_fkMzuUIu_L

  • The California Technology Assessment Forum (CTAF), part of ICER, released their recommendations from the February meeting for new drugs in diabetes and asthma.
  • CTTI just released materials from an expert meeting about the issues surrounding FDA streamlined approvals for new antibiotic drug development. A summary will be posted soon. You can also read a past post called Antibiotic Resistance – It’s Complicated!
  • Cures Within Reach focuses on ways that existing drugs can be used for other purposes. More from this group in a guest blog post here soon!
  • Two DCIS studies are being funded by PCORI to help answer treatment questions for this pre-cancer, and learn how women feel about them. You can also read a past post called When is ‘Carcinoma’ Not Cancer?
  • The Center for Medical Technology Policy (CMTP) is working on recommendations to allow patients to switch treatments in a clinical trial if their cancers keep growing.
  • The Metastatic Breast Cancer Alliance (MBCA) is creating a summary of their February meeting from the Research Task Force to Advance Progress.
  • The NC ProCESS project on prostate cancer has convened a patient advisory board to help communicate effectively with prostate survivors in their study.
  • Two groups I’m working with (MRCT and the NCI NCTN Biospecimen Banks) are discussing how to handle the challenges of giving individual research results to patients.

You can also read past CT posts about:

There are many other clinical trial happenings – these are just some of the ones in which I’m involved! Yes, it’s heavy on cancer, but remember that cancers cover the gamut from rare diseases to millions of people.

Updates will be posted regularly. Let me know what you’d like to hear about. TTFN.

All content © 2016 by Deborah Collyar unless otherwise specified. All rights reserved. Permission is granted to use short quotes provided a link back to this page and proper attribution is given to me as the original author.

6 Ways to Turn Data into a Good Read

Ever take a bunch of facts and turn them into something readable? For real people? From many dry scientific journals? When sources disagree? Vehemently?

That is exactly what UCSF researchers Thea Tlsty, PhD, Philippe Gascard, PhD, and I did. Here is what we learned in order to help make sense out of a major worry for women.

But First, Why?

Computer generated 3D photo rendering.

We want to help ~60,000 people (mostly women) who deal with a condition called Ductal Carcinoma In Situ (DCIS) each year. It’s often over-treated, so women want to know what to do. Many questions surround DCIS, and researchers argue at every step along the way.

“DCIS means cells inside the breast duct look and behave like cancer cells. DCIS cells, though, stay inside the ducts and do not travel anywhere else in the body.”

Excerpt From: Deborah E. Collyar. “DCIS Dilemmas

For instance, is DCIS really cancer? Some say no, and want to change its name. Others treat everyone as if they have breast cancer. Still others want to find the 1 out of 10 women (11%) who will eventually get invasive breast cancer so they can be treated, or find the next 11% who might get another DCIS that won’t affect how long they live.

“When you put a frightening term like carcinoma on a lesion that, on average, doesn’t go on to invade normal tissue, that can prompt women who are justifiably frightened of the word cancer to have therapy that’s every bit as aggressive as if they had a true invasive cancer.”

Barry Kramer, Director, Division of Cancer Prevention at the National Cancer Institute in an article from Proto

A few focus on finding almost 8 of 10 women (78%) who will only have DCIS once, and don’t need to endure the long-term problems of over-treatment. Our efforts turned into a new eBook called DCIS Dilemmas: Discussions about Ductal Carcinoma and the Research Behind It. So, how did we do it?

How to group complex data

1. Gather a Range of Data

Research is important, but don’t expect simple answers. We scoured scientific studies (in journals and conferences), and separated conclusions and opinions into different categories. We set a rule that all of the facts we used had to come from at least 2-3 different sources. Articles like this one from HealthNewsReview.org were also helpful.

“New DCIS study, news release lead to (very) mixed messages: ‘And we wonder why patients get confused’”

Dave Mosher in a HealthNewReview.org article

2. Find Out What is Important to Readers

Before starting the eBook, we surveyed 3 groups:

Important Stamp Shows Critical Information Or Documents

  • Women who were cancer-free
  • Women who had DCIS
  • Women who had invasive breast cancer (IBC)

We also held a DCIS Forum in San Francisco to present real information, dispel myths, and hear directly what women want to know.

3. Put It in Perspective

While DCIS is serious, it is not fatal. Unfortunately, many women are led to believe they have breast cancer and are treated this way even though most (over 3 out of 4) will never have another event. The important part is to figure out who is actually at risk for a future invasive breast cancer and what they can do about it.

4. Explain Why Everyone is Confused

Puzzled Confused Lost Signpost Shows Puzzling Problem

DCIS is as confusing to doctors and researchers as it is to patients. A lot of this is due to old ways of thinking, and old terms that are still used. For instance, most doctors and researchers talk about the risk of a “recurrence,” meaning another DCIS or breast cancer. This is actually impossible for DCIS! The old mindset treats DCIS as if it is cancer, not a pre-cancer. It’s time to change that.

5. Explain What Can Be Done About It

It was clear from women that they want to know what to do about DCIS. So, we shared information about:

  • How DCIS is diagnosed ebook DCIS Dilemmas - older woman w tablet
  • The different kinds of treatment used (including Active Surveillance)
  • What risks exist for all women, as well as risks after being diagnosed with DCIS

We also listed current research that will hopefully produce clearer answers in the future.

6. List Resources for All to See

We wanted full disclosure on all of our sources, so we listed everything we reviewed in categories for those who want more information. We also flagged some of the controversies so people can make up their own minds.

What Do You Do?

List your ideas in the comments, so we can learn from each other. We’d also appreciate your feedback on our approach. If you’re interested, check out DCIS Dilemmas: Discussions about Ductal Carcinoma and the Research Behind It and let us know what you think!

All content © 2016 by Deborah Collyar unless otherwise specified. All rights reserved. Permission is granted to use short quotes provided a link back to this page and proper attribution is given to me as the original author.