Do We Need Phase III Randomized Controlled Trials for EVERYTHING?

Posted by Judi Space on Thu,Aug 29, 2013 @ 09:00 PM

Targeted therapy agents

Phase 3 randomized controlled trials - time to drop in some cases?In clinical trials of targeted therapy agents, sometimes the target is such a crucial driver of the neoplastic process, that its inhibition is able to stop the growing tumor in its tracks. This appears to be the case for some recently approved agents such as vemurafenib for BRAF V600E melanoma and crizotinib for ALK+ NSCLC. Careful screening of patients for these mutations ensures the likelihood of improved progression free survival (PFS) and longer survival times. Unfortunately, because patients are switched from the control regimen in the Phase III trials, the chances of proving better survival are greatly diminished, if not completely eliminated.

In a recent opinion piece in Nature Reviews: Clinical Oncology, Robert Doebele argues that when patients are screened for the relevant oncogene "the burden of proof for approval should be adjusted for oncogene-targeted therapy in biomarker-selected patients....There might be oncogenes that will not follow this paradigm, but that should not deter us from trying to extend the benefit of this precision medicine approach to patients that may fall into one of these uncommon oncogene categories." 

Crizotinib for ALK+ NSCLC

One example is crizotinib, a tyrosine kinase inhibitor (TKI) for the treatment of ALK gene rearrangement-positive (ALK+) non-small-cell lung cancer (NSCLC), which was approved by the FDA in 2011 based on the results of phase I and phase II clinical trials. Phase I observed an objective response rate (ORR) of 61% and a PFS of 9.7 months.  Phase II observed an ORR of 53% and a PFS of 9.1 months.

Considering the estimated ORR and PFS for most single-agent chemotherapy drugs in pretreated patients with lung cancer is 10% and 3-4 months respectively, was there really a need for a Phase III RCT in this case? 

The outcome of a phase III randomized trial, PROFILE 1007, of crizotinib in patients with ALK+ lung cancer merely provided further proof that cizotinbin is superior to standard chemotherapy for these patients that included a better quality-of-life and physical and emotional function.  Based on the results of this study, it seems reasonable to argue that the burden of proof for approval should be adjusted for oncogene-targeted therapy in biomarker-selected patients, especially in lung cancer. 

Not limited to TKI

Calls for the elimination of Phase III trials are not limited to TKI. During the presentation of a melanoma Phase II trial of a combination of two immunomodulators, ipilimumab and nivolumab at ASCO this year, one of the questions from the audience was, "Do we really need a phase III trial?" The results of the trial being presented were certainly impressive, with one year survival in the 80% range.

Time to shift the burden of proof?

Based on these examples, some feel that it may be time to eliminate the Phase III trial in targeted therapy agents when such overwhelming proof is given that a therapy induces remarkable tumor responses in Phase I and II.  This will allow studies to move along more quickly, minimize the need to switch patients from the control regimen, and provide more rapid approval for certain therapies.


Pennside Partners is a leading pharmaceutical and biotech consulting firm with over 25 years experience. They are headquartered in the United States, the United Kingdom and Switzerland.

Tags: Oncology, Targeted Therapy, Cancer Drug Development, Drug Development

Defining Success in Clinical Trials: Is Learning Something Enough?

Posted by Judi Space on Thu,Jul 25, 2013 @ 06:00 PM

Avastin discussed at ASCOAt last month’s ASCO conference, rapt attention was given to a presentation on the clinical trial results for the drug Avastin for patients recently diagnosed with an aggressive form of brain cancer, glioblastoma multiforme (GBM), according to an article by Clinton Leaf appearing in the New York Times, which is summarized below, with our comments added. The presentation by Dr. Mark R. Gilbert, professor of neuro-oncology at the University of Texas M. D. Anderson Cancer Center, revealed earlier clinical trial results that were encouraging:  tumors shrank and the disease appeared to stall for months in two smaller single-arm studies.

No Difference

However, Dr. Gilbert’s larger double blind study of over 600 brain cancer patients found no difference in the survival rate for patients given a placebo with standard treatment vs. those given Avastin with standard treatment.  In effect, the larger, more reliable double blind study eliminated doubt as to the general effectiveness of the drug.   In other words, the trial was “a success” because it had a clear result, but we learned that the drug has no statistically significant impact on the disease as a whole. But is that enough for the trial to truly be deemed a success?

It depends on how you define success. On one hand, doctors have no additional information on how to successfully treat brain cancer as a result of the study. On the other, one possible solution has been eliminated, so doctors and pharmaceutical companies can continue their quest for an effective drug armed with the knowledge from the study.

Not so Fast

The problem is that it is not quite as cut and dried as all that. Some patients who take Avastin do significantly better than those who do not. However, the trial failed to reveal the responders that make this result possible. Despite 400 clinical trials over 16 years, no correlation has been made as to why Avastin works or fails to work in certain patients. Again, given that we don’t know, can we deem the trial a success?  

Individualized Factors May Hold the Key

Researchers are learning that individualized pathology and physiology may be significant factors in developing effective drugs for treating certain diseases.  The variability of tumors and cancers at the genetic level can be great, as can the way our genes affect our response to drugs. So the solution to some diseases may lie in identifying the drugs that best align with the individualized factors associated with a given disease.

Failure rates in clinical trials tend to increase significantly as the drug moves from phase I to phase II. In the final phase it is not unusual for trials to wholly contradict earlier trials, or for the benefits to be far less than what was first reported, according to a 2005 paper published in the Journal of the American Medical Association by Dr. Ioannidis, an authority on statistical analysis.

The Difficulty of Matching Clinical Trial Populations to Patients

Rules governing statistical trial enrollment typically produce trial populations of younger and healthier members than those likely to use the drug. In addition, trial populations typically have had far less medical treatment. These factors combine to form somewhat of a mismatch between the trial population and patients. Given that the vast majority of drugs that enter clinical testing fail to get approved (95%), finding ways to match clinical trial participants with the appropriate genetic or molecular signature may be paramount to finding drugs that work.  

That’s the approach taken by Genentech in developing Herceptin, a breast cancer drug that targets tumor cells with an abundance of the HER2 protein. Small clinical trials were designed that enrolled only those with the required molecular or genetic signature. 60% of the new drugs in development at Genentech/Roche are being developed in concert with a diagnostic test to determine patients most likely to benefit. They did not go that route when they developed Avastin and tested it in patients with GBM.

Back to the Question at Hand

So, is it enough to simply learn something from a clinical trial, even if we don’t find a solution to treat a disease? Given that there is so much riding on finding drugs that improve and extend the lives of those suffering, anything that moves us forward, even if only eliminating what was thought to be a potential solution, is a step in the right direction.


Pennside Partners is a leading pharmaceutical and biotech consulting firm with over 25 years experience. They are headquartered in the United States, the United Kingdom and Switzerland.

Tags: FDA, Oncology, Targeted Therapy, Cancer Drug Development, Oncology Clinical Trial Design, Breast Cancer, Breast Cancer Drug Development, Breast Cancer Oncology, Glioblastoma, ASCO

Molecular Testing in Trouble from Medicare. Who’s on First?

Posted by Judi Space on Tue,Apr 02, 2013 @ 06:00 PM

Only a Bureaucracy as Inefficient and Unwieldy as Medicare Could Create a Mess This Bad and Then Walk Away Whistling, Hands in Pockets

Medicare's decision to stop reimbursing labs for molecular testing threatens both the labs and the patients who badly need these potentially life-saving testsI’m referring to Medicare’s decision to stop paying bills for molecular testing and other diagnostic tests that are transforming the way many life-threatening diseases are being treated.  This is a decision affecting hundreds of labs, including labs performing proprietary tests that target treatments for cancer patients and other serious conditions. To be clear, this is not a decision to permanently stop paying, but to withhold payments until a favorable alternative has been found to the current system of reimbursement.

Could this have come at a worse time, a time at which significant medical and diagnostic gains are being made by molecular testing?

What began as an honest effort to reduce costs has devolved into a quagmire of indecision, with no one taking charge of resolving the mess.  In the meantime, bills from hundreds of labs and health care companies are going unpaid, with no clear resolution in sight. Just how long labs and health care companies will continue to perform these expensive services without being reimbursed is anyone’s guess, but the situation should be causing alarm bell to go off inside Medicare and the Obama administration. 

Here’s How the Medicare Reimbursement System for Molecular Testing Broke Down

First, Medicare, in a much-needed effort to address an abuse-prone billing and reimbursement system, decided to seek an alternative to the billing process known as “code stacking”, a system whereby the costs and reimbursements for diagnostic tests are determined by identifying the codes and associated costs for each step in performing a particular molecular test.  Rather than paying based on the true value of the test, the code stacking system paid for the steps, I.e., the complexity of a test. This of course opened the door to abuse and inconsistency, with some labs identifying and charging for additional steps.

116 New Codes Went Into Effect in 2011, but Medicare Sat Them Out

In order to replace this with a system of paying based on what was being tested form, in 2010 Medicare asked the American Medical Association to create codes for the most critical and common molecular tests. 116 new codes went into effect in 2011, but Medicare decided to wait until 2012 to use the new system, and until then to continue using the old code stacking method until it could set pricing for the new codes.  In the year since, Medicare has done virtually nothing, and (stunningly) has instead decided to have local Medicare carriers assign prices to each of the diagnostic codes! Now, at the last minute, instead of a unified pricing and reimbursement standard, the local Medicare carriers are forced to determine pricing.

Tests Going Unpaid, at Least for Now

As a result, many prices have not been established, and many tests are going unpaid. The market for the development of new tests is being hammered with uncertainty, with many investors sidelined until the pricing issues are resolved. To get by, some contractors are beginning to mirror the pricing charged by the Medicare contractor for California, the only carrier who has managed to set rates. One can question whether a state as large and unique as California should be driving the rates for the US as a whole.  That said, other contractors are not following the California model, and are not getting paid.

On April 30, Medicare contractors are scheduled to submit their 2013 pricing, including their prices for molecular testing. Once this occurs, it is possible that the labs will get paid after the fact. However, this is not guaranteed, and the deadline is not firm.

Is it Fair for Medicare to Delay Payments for Molecular Testing?

One must ask, is it fair for Medicare to refuse to pay for molecular testing simply because they bungled their own deadline for producing a viable reimbursement system? Should the agency be permitted to walk away from the problem by throwing it into the hands of the Medicare contractors, while in the meantime testing labs go unpaid and patients run a growing risk of having to do without potentially life-saving diagnostic testing?

For more information on this timely issue, see the full article that inspired this post on Forbes.


Pennside Partners is a leading pharmaceutical and biotech consulting firm with over 25 years experience. They are headquartered in the United States, the United Kingdom and Switzerland.

Tags: Oncology, Targeted Therapy, Molecular Testing

Will Oncology and Pathology Unite on Molecular Testing?

Posted by Judi Space on Wed,Mar 27, 2013 @ 07:00 PM

For more than a decade, we have been listening to the leaders in cancer research tell us that in order for targeted therapy to work, we must be able to identify who will benefit from it through the use of predictive biomarkers. This is the essence of personalized medicine.

Is molecular testing the key to targeted therapies and a solution to a one-size-fits-all approach?Molecular testing (MT) is becoming the new paradigm for determining an individual cancer patient's course of treatment. Although it is fairly widespread in large academic cancer centers, community hospitals and cancer clinics, where most cancer patients are treated, are often completely out of the loop.

Recently the Association of Community Cancer Centers has sought to better understand the scope of this problem through a survey of its members (1). This survey polled the member institutions' administrators, oncologists, pathologists nurses, and pharmacists (N=52 institutions) about policy, practices, and proceedure related to MT as well as reimbursement issues.

Implementing Molecular Testing: Education of Stakeholders is Key

There are many challenges to the implimentation of MT in the community oncology setting(1).  Chief among them is unwillingness of various stakeholders to work together to make MT a routine proceedure for all newly diagnosed cancer patients. The reality is that testing is already being done on a very limited basis for breast cancer patients (HER2, and ER/PR), but the cancer centers appear to balk at the expansion of similar testing in other cancer indications.(2)

It also represents a great new opportunity to improve the coordination between pathologists and oncologists.

In order to implement MT, The ACCC survey indicated that there should be "champions" that drive the institution toward a coordinated testing program. Most often these champions come from the ranks of the oncologists. They must educate their peers and the institution's administration about the necessity for having a MT policy/proceedure to decide how and when specific tests will be implemented.

Will Oncology & Pathology Come Together Over MT?

Can oncology and pathology unite over molecular testing?The lack of collaboration between pathologists and oncologists is one of the things that's holding molecular testing programs back. Many physicians can't seem to agree on whether MT is necessary, and when. Some don't see the value in personalized medicine, while cancer center administrators see it as simply a line in the budget and are unwilling to spend the time and money in an environment of competing interests.

However, there is safety in numbers. By uniting on the molecular testing issue, oncologists and pathologists will have a greater chance of convincing administrators its necessity.

Is Molecular Testing Costly or Cost Effective?

One of the drawbacks to molecular testing programs is the cost of testing. The decision for doing MT may lie with the payer, rather than the patient's specific needs.  Each assay can run as much as $7,000, which can make insurance companies unwilling to cover the cost in many cases.

However, most oncologists and pathologists agree that molecular testing can actually save money in the long run. The detailed, personal information that it provides can guide an otherwise uncertain treatment plan. A negative test result can save a lot of money on expensive, targeted therapies.

In order for progress to take place, continuing education is essential. This is true of both the medical oncologists and the pathologists. In the rapidly evolving world of cancer drug development, new molecular tests will emerge on a regular basis which will help choose new options for treatment where there were none before.  Pathologists and oncologists must work together to use the test results to guide treatment by identifying who will benefit from specific targeted therapy regimens. 

References

(1)http://accc-cancer.org/education/molecularTesting-Overview.asp

(2) http://obroncology.com/obrgreen/article/Molecular-Testing-Preparing-for-the-New-Normal-in-Community-Oncology


Pennside Partners is a leading pharmaceutical and biotech consulting firm with over 25 years experience. They are headquartered in the United States, the United Kingdom and Switzerland.

Tags: Oncology, Predictive Biomarkers, Targeted Therapy, Drug Development, Molecular Testing, Personalized Medicine

NCI Improvements: Drug Development Opportunity for Pharma and Biotech

Posted by Judi Space on Tue,Mar 12, 2013 @ 06:00 PM

Three years ago the Institute of medicine declared that the nation’s cooperative group system for conducting clinical trials was so moribund as to not be an effective vehicle for the generation of scientific data in a timely fashion. In a report generated in 2010, the IOM recommended that the number of cooperative oncology groups be reduced from 10 down to 5[1], improve speed and efficacy of the trial processes as well as incorporate new scientific discovery into clinical protocols. Along with this, the report said it was critical to create a data platform that would assist in the administration of trials across all of the remaining cooperative groups. This would be a system to which both large academic centers and community cancer centers could adhere.

NCI Less of a Barrier; More of a Facilitator

To a large extent, the NCI was  a barrier to the conduct of clinical trials due to their role of providing oversight. The IOM report urged that NCI become part of the solution by helping to prioritize and design trials as well as directing patients to relevant studies so that enrolment could proceed more rapidly. In addition, CMS was tasked with removing barriers to enrollment by supporting appropriate costs associated with patient treatment during the trials.

Efforts Bearing Fruit

Now, nearly three years later, we can see the effects of this report. In a two day workshop[2] that took place in Washington last month, improvements in the national cooperative group trial system were documented[3]. Among the improvements noted:

  • The time between concept and activation of phase II trials sponsored by NCI has shrunk by more than a third. Trials not meeting timelines will be terminated!
  • Time to activation for phase III trials has been halved since the overhaul process began
  • The number of cooperative groups has actually been cut in half – now there are only 5
  • A common enrollment and data management system has been put into place
  • Patients can enroll 24/7 and they may participate in ANY Ph III trial in the system regardless of origin
  • There are now  standardized agreements for industry collaborations
  • A new  review process has been launched for grading the quality of trials
  • Integrated biospecimen banks are being created

Still to come are more innovative Ph II trials based on molecular biology of the tumors and the extensive use of biomarkers and genomic profiling.

Ecology of Clinical Trials

Community Cancer Centers are Key

The key to moving clinical research forward more rapidly will be enhanced participation of community cancer centers. Investigators at these sites are often hesitant to take part as many of the costs associated with the trials are not covered.  After more provisions of the Affordable Care Act go into effect, next January, this may no longer be a barrier. Federal law will require insurers cover the cost of routine patient care in clinical trials.

Design and Conduct of Trials Improving in UK As Well 

Interestingly, the US is not the only place that has had a sea change in the way it conducts clinical research for cancer. Over the same period, the UK’s National Health Service has recognized that clinical research is an important part of its mission, and therefore has provided incentives for patients to participate in trials. The key here is that cancer patients do not get access to novel agents unless they participate in the trials. The result is a trials network that rivals anything in the US but existing in a much smaller country.

 

Implications for Drug Development:

Drug approval process improving in the UK as well as the US

Pharma  and biotech companies can harness the power of the NCI to get things done faster than they have become accustomed to in the past, and the improvements will continue into the foreseeable future. This is also good news for cancer patients who will be able to access more cutting edge clinical trials no matter what part of the country they are from.

 


[1] http://www.iom.edu/~/media/Files/Report%20Files/2010/A-National-Cancer-Clinical-Trials-System-for-the-21st-Century-Reinvigorating-the-NCI-Cooperative/NCI%20Cancer%20Clinical%20Trials%202010%20%20Report%20Brief.pdf

[2] http://www.iom.edu/~/media/Files/Activity%20Files/Disease/NCPF/2013-FEB-11/Agenda%20-%20IOM%20ASCO%20Implementation%20Workshop.pdf

[3] http://www.nap.edu/catalog.php?record_id=18273


Pennside Partners is a leading pharmaceutical and biotech consulting firm with over 25 years experience. They are headquartered in the United States, the United Kingdom and Switzerland.

Tags: FDA, Drug Developers, Oncology, Predictive Biomarkers, Targeted Therapy, Data Standardization, NCI, Cancer Drug Development, Oncology Clinical Trial Design

ASCO GU: Heads-Up for Biotech Drug Development in Kidney Cancer

Posted by Marne Wessner on Mon,Feb 11, 2013 @ 11:50 PM

Interesting Data for Pharma and Biotech Drug Developers

ASCO GU2 2013 Genitourinary Cancers Symposium for Pharma and Biotech Drug DevelopmentASCO’s second meeting of the year, ASCO GU, is coming up next week, and here is our list of anticipated data releases for renal cell carcinoma.  (Note that full abstracts won’t be available until February 12, so it is impossible to comment on the significance of these results until then.) It looks to be a quiet meeting this year for RCC, according to key opinion leaders, but there are some presentations on the schedule with the potential for interesting data for Pharma and Biotech drug developers.

Additional Data from TIVO-1 Trial of Aveo/Astellas’ Tivozanib

There are several abstracts reporting aspects of the TIVO-1 trial data at this year’s meeting. OS, subgroup analyses, and quality of life data are of high interest, and two additional poster presentations will also address biomarker research and tivozanib efficacy in patients previously treated with Bayer’s Nexavar (sorafenib).

Emerging Immunotherapies

Immunotherapy is an exciting field in RCC. The latest generation of immunomodulating agents includes nivolumab, an anti-PD1 from BMS; and AGS-003, a dentritic cell therapy from Argos—both in Ph III for RCC. These agents each have poster presentations, which will hopefully give early indications about their potential in RCC.

Sequencing and Combinations of Approved Therapies for Kidney Cancer

The hottest issue in RCC therapeutics is the sequencing of the many approved agents on the market, and the positioning of any new ones about to be approved. This is a conundrum for oncologists as well as drug developers seeking to leverage the data to gain market share.

  • Votrient: Additional quality of data from the COMPARZ trial of GSK’s Votrient (pazopanib) vs. Pfizer’s Sutent (sunitinib) in first line RCC will report, likely an update of what was reported at ESMO 2012 last fall.
     
  • Inlyta:  Pfizer announced that the AGILE 1051 trial of Inlyta (axitinib) in first line RCC did not meet its primary endpoint in October 2012, and it looks like we will finally see data from that trial as well as the Ph II dosing trial in first line RCC.
     
  • Avastin combos: The Ph II BEST trial, which compared Roche/Genentech’s Avastin alone and combinations of Avastin, Nexavar, and Torisel, will also report data. In RCC, combinations of currently-approved targeted agents have been unsuccessful historically, so expectations are low for this trial.
     
  • mTOR inhibitors: Finally, a new safety analysis of mTOR inhibitors will be presented, which will hopefully provide oncologists with more information on dealing with the side effects of this class of drugs, which are very different from the tyrosine kinase inhibitors that are commonly used in RCC.

 

Key Presentations of Interest at ASCO GU

  1. Overall survival results from a phase III study of tivozanib hydrochloride versus sorafenib in patients with RCC (Motzer, et al, abstract 350)
     
  2. Subgroup analyses of a phase III trial comparing tivozanib hydrochloride versus sorafenib as initial targeted therapy for patients with mRCC (Hutson, et al, abstract 354)
     
  3. Treatment benefit of tivozanib hydrochloride versus sorafenib on health-related quality of life among patients with advanced/mRCC: Tivo-1 study results (Cella, et al, abstract 355)
     
  4. Axitinib vs. sorafenib as first‑line therapy in patients with mRCC (Hutson, et al, abstract LBA348)
     
  5. Quality of life among patients with RCC treated with pazopanib versus sunitinib in the COMPARZ study (Cella, et al, abstract 346)
     
  6. Axitinib with or without dose titration for first-line mRCC: Unblinded results from a randomized phase II study (Rini et al, abstract LBA349)
     
  7. The BEST trial (E2804): A randomized Ph II study of VEGF, RAF kinase, and mTOR combination targeted therapy with bevacizumab, sorafenib, and temsirolimus in advanced RCC (McDermott et al, abstract 345)
     
  8. Incidence and risk of treatment-related mortality in patients with RCC and non-RCC treated with mTOR inhibitors (Choueiri, et al, abstract 347)
     
  9. Clinical activity and safety of antiPD-1 (BMS-936558) in patients with previously treated mRCC: An updated analysis (McDermott, et al, abstract 351)
     
  10. Prolonged survival with personalized immunotherapy (AGS-003) in combination with sunitinib in unfavorable risk mRCC (Amin, et al, abstract 357)


Pennside Partners is a leading pharmaceutical and biotech consulting firm with over 25 years experience. They are headquartered in the United States, the United Kingdom and Switzerland.

Tags: Drug Developers, Targeted Therapy, Drug Development, Kidney Cancer, Biotech Consulting, Pharma Launch Planning

Breast Cancer Drug Development - Targeting HER2 Activating Mutations

Posted by Judi Space on Thu,Jan 10, 2013 @ 09:00 AM

A Possible Development Pathway for Biotech Companies with PanHER Inhibitors?

Many Breast Cancer Patients Not Helped by Herceptin

For more than ten years oncologists have been aware of the role that Herceptin (trastuzumab, Roche/Genentech) has played in extending the lives of women with HER2 overexpressed breast cancer. Herceptin works by blocking the HER2 receptor from receiving activating stimuli from outside of the cell. In addition there is a second agent, Tykerb (lapatinib, GSK) which is able to block signaling from the tyrosine kinase domain of the receptor, inside the cell. Still, there are many patients who are not helped by these agents. These targeted therapies work when the receptor is overexpressed, but may not work when the receptors are mutated.

Breast cancer drug development - exposure to neratinibSomatic Breast Cancer Mutations Identified

At December’s San Antonio Breast Cancer Symposium, there was a talk given by Ron Bose from Washington University, about other perturbances of the HER2 receptor[1][2]. Over the last year, the Cancer Genome Atlas Breast Cancer has conducted genome sequencing of breast cancers. This endeavor has identified several somatic mutations that appear on the HER2 receptors in some breast cancers. A majority of the mutations were found in the kinase domain of the receptor. Most of the lesions analyzed were considered HER2 negative as they did not achieve the 3+ score in IHC testing.  

Ph II Trial of Neratinib in Breast Cancer Patients

Because the mutations cause constitutive activation of the receptor (it is always turned on), Herceptin cannot block its signaling from outside of the cell. The mutations are rare, and it is estimated that only about 5000 women in the US with breast cancer would have them. In cell line studies, Tykerb has some effect on cells with these mutations, but the agent that appears to have the most activity is neratinib (Puma), a “pan-HER” inhibitor. To that end, there will be a fairly large Ph II trial of neratinib in breast cancer patients who test negative for HER2 amplification but positive for these somatic mutations.

Breast Cancer Drug Development - Targeting Somatic Cancer Mutations

There is precedent for targeting somatic mutations in malignant lesions. Even in the same family of receptors, there are three agents with activity against EGFR: Iressa (gefitinib, AstraZeneca), Tarceva (erlotinib, Roche/Genentech) and afatinib (Boheringer Ingleheim). This has implications for the larger world of breast cancer biotech drug development targeted at the EGFR family of receptors as there are many “pan-HER” inhibitors in development that could potentially be used to treat tumors with these mutations.

Barriers to Breast Cancer Drug Development

The biggest barriers for development would be working up accurate testing systems to detect the mutations as predictive biomarkers of drug efficacy, and then getting these tests included in the battery of diagnostics used to analyze both primary breast tumors and recurrent disease.     


[1] Bose, R Activating HER2 mutations in HER2 gene amplification negative breast cancers, SABCS December 2012, S5-6


Pennside Partners is a leading pharmaceutical and biotech consulting firm with over 25 years experience. They are headquartered in the United States, the United Kingdom and Switzerland.

Tags: Oncology, Predictive Biomarkers, Targeted Therapy, Breast Cancer, Breast Cancer Drug Development, Breast Cancer Oncology, Breast Cancer Therapy

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