Welcome to BioMarketing Insight's monthly newsletter.

This industry has gone through a lot of changes with many negative effects including Obamacare, as noted in a previous newsletter. This month and next I'll discuss some positive changes to the industry.  

This month, I will cover the new regulatory path for "Breakthrough Therapies" for Pharma/Biotech and next month I will talk about positive changes in the medical device industry.

Read on to learn more about this topic and other current news. On the right are quick links to the topics covered in this month's newsletter. The next newsletter will be published on April 15th.

We encourage you to share this newsletter with your colleagues by using the social media icons at the top left, or by simply forwarding the newsletter via email.

Please email me, Regina Au, if you have any questions, comments, or suggestions.

Sincerely,

Regina Au

Principal, Strategic Marketing Consultant

BioMarketing Insight  

What is the big trend in medical devices? It is wireless applications in medical devices and diagnostics. I will give a presentation at the New England Chinese Information and Networking Association (NECINA) on this subject Saturday, April 6th, at KPMG in Boston. 

Agenda

9:00 - 9:10 Opening remark
9:10 - 9:40 Emergence of Wireless Technologies in Medical Devices and Diagnostics, Regina Au
9:40 - 10:10 Emerging Opportunities in Healthcare Informatics, Terence Russell
10:10 - 10:20 coffee break
10:20 - 10:50 Better Health Through Mobile Devices, Lisa Gualtieri
10:50 - 11:20 TBD, Victor Wang, Gerijoy
11:20 - 11:30 coffee break
11:30 - 12:00 Panel Discussion
12:00 - 1:00 pm Lunch and Networking   

For more information and to register, click here.  

The FDA has been under the microscope these days, being held accountable not only for the slow process in getting drugs, biologics, and devices approved by the manufacturers, but also for approving products that are not safe in the eyes of the public, and not approving products for life threatening diseases faster. Since the change in regime with Jeffrey Shuren, Margaret Hamburg, and Janet Woodcock, there have been some positive changes in the regulatory process.

If a drug is designated as a breakthrough product, actions to expedite its development and application review of an application will be implemented and drugs could be approved based on data from an expanded Phase I clinical trial, according to Woodcock.  For more information on applying for Breakthrough Therapies, click here. 

Upon receipt of the submission, a determination will be made within 60 days to either grant or deny the request for Breakthrough Therapy designation, in the form of a designation letter (for requests granted) and or a non-designation letter (for requests denied).

The FDA has initially assigned this status to three drugs. On January 31st, 2013, two of Vertex Pharmaceutical's drugs won this designation for cystic fibrosis, the approved drug Kalydeco and the experimental VX-809, now being studied as a combination therapy. Eighteen other drugs, mostly for cancer, have been submitted for review by other drug developers. 

Woodcock is also trying to change the drug development process paradigm by creating standing trial networks of expert doctors and community physicians, rather than having each company seek its own network of clinical trial sites and key opinion leaders. In these trial networks, Woodcock said, therapies coming out of dose-finding Phase I studies can quickly be tested for efficacy in patients.  

"To me, this sounds so simple," Woodcock said. "There are thousands of experts in diseases, but they are not engaged in trials much of the time."

Woodcock also advocated that more precompetitive collaboration between academia, industry and patient groups is needed.

If personalized medicine is to succeed, doctors must be allowed to match what works for a specific patient with a specific condition at a specific time. "We need new clinical development approaches," Woodcock said. Right now, about 95 percent of adult cancer patients are not offered entry into clinical trials.

The "stately progression" of an experimental drug working through a Phase I safety trial, Phase II effectiveness study and all-out Phase III can costs billions of dollars, Woodcock noted. This is a dramatic contrast to the get-it-today focus of personalized medicine.

Companion Diagnostics

In mid- February, J&J won breakthrough designation for its experimental drug ibrutinib, a small molecule inhibitor of the Bruton tyrosine kinase enzyme, designed to kill B-cell malignancies like leukemia and lymphoma.  Now, Abbott Lab is teaming up with J&J to develop a companion diagnostic.

"Abbott's role is to develop a test that can identify ideal patients with a genetic subtype of chronic lymphocytic leukemia (CLL), using the company's fluorescence in situ hybridization technology, or FISH," as reported by FierceMedicalDevice

Abbott's test will identify CCL patients who have a specific abnormal chromosome, and are likely to respond to ibrutinib but a poor responder to chemoimmunotherapy the company said, potentially improving outcomes and speeding up treatment.

"Like Abbott's other collaborations in the area of companion diagnostics, our goal is to leverage molecular technologies to help ensure that the right medicine is getting to the right person," Molecular Diagnostics Vice President John Coulter said in a statement. "Cancer is a complex disease where, historically, therapies have demonstrated only a 25% efficacy rate."

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There have been some positive changes at the FDA that will expedite the approval process for all types of products. The new "Breakthrough Therapies" designation is a step forward in getting critical drugs to market for diseases that are life threatening and have no treatment. This benefits both the patient and the drug developer. It is impressive that the FDA will approve a breakthrough therapy on an expanded Phase I trial.

However, I think the FDA will be very cautious and only approve a drug if the preclinical and clinical data are overwhelmingly effective and safe. It won't be that easy, even with this new designation in place. The FDA still has to balance the safety of the product against the needs of the patient.

As expected, many drug companies developing cancer therapies are applying for this designation. I don't believe all the cancer therapies will be classified as Breakthrough Therapies because it will depend on a number of factors, such as the indication in terms of the type of cancer (slow or aggressive), the patient survival rate, treatments currently available and the success rate of existing treatments.

Uncommon or rare diseases are another area that can greatly benefit from this designation, since the cost for development will be less and hopefully more companies will be incentivized to tackle these diseases.

Even if the FDA approves these Breakthrough Therapies, will the patient ultimately be able to get the drug? Will the insurance companies pay for these new expensive drugs? Insurers are probably more conservative than the FDA in assessing risk and the probability that the drug will work is now based on less data and less patient experience, since Phase II and Phase III may be eliminated.

Companion diagnostics will grow, as is in the case with J&J and Abbot Labs. You will see more collaborations and deals with companies for developing companion diagnostics. Researchers are very good at identifying biomarkers, but the hard part is determining which biomarkers are relevant to screen for a disease that matches up with the treatment. While companion diagnostics will grow, it won't grow at a rapid rate because we still don't understand the biology of diseases to the extent that we should in developing these diagnostics. Mastering this will bring us closer to personalized medicine.

Having a companion diagnostic is great for the patient, but again the question that must be asked is, will this be reimbursed by the insurance provider? Is it cost effective for payers? In other words, how many patients will have to be screened for a biomarker/s to say that this drug will/or will not work in that patient? Is this more cost effective than doing trial and error with the available drugs?

Janet Woodcock advocates for more pre-competitive collaboration between academia, industry, and patient groups for drug development, but unless the insurance providers are on the same page, the patient may not get the drug in the end.

In conclusion, as companies develop drugs, they must consider the reimbursement piece as part of their due diligence very early in product development. One must also be able to make a case that is relevant to what the payers consider to be cost-effective, not what we consider to be cost-effective.

Need to conduct the due diligence? Email me in discussing this further.

Back-of-the-brain view shows fMRI data superimposed on MRI scan data; level of increased activity in left and right visual cortex (blue) predicted depressed patients' responsiveness to scopolamine, an experimental antidepressant--Courtesy of Maura Furey, Ph.D., NIMH. FierceBiomarker

In treating depression, as with most medications, it's trial and error to find the right medication for the patient. Investigators at the NIH claim to have found a neuroimaging biomarker at the back of the brain, the brain's acetylcholine system, which temporarily stores information in the brain that could prove effective in personalizing drugs in the future--raising the odds of matching a patient to with the best therapy.

"We have discovered a potential neuroimaging biomarker that may eventually help to personalize treatment selection by revealing brain-based differences between patients," explained Maura Furey, Ph.D., of NIH's National Institute of Mental Health.

To read the full article in FierceBiomarker, click here .

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Twenty Medical Device and Nine Pharma/Biotech Funding Deals

To determine whether funding is picking up, I will be focusing on all types of funding that are $1 million or greater in seed investments and series A or B (or the valley of death) that are pre-IPO. Even though VCs are investing, they continue to invest in their existing portfolio companies and less in start-ups. Incubators, state funding, and business competitions are great for initial seed money but not enough to keep the company going long-term.  These are worldwide funding deals. 

Partnerships and licensing deals with upfront payments and milestones will not be included.

Funding deals are in chronological order by date.

$0 = No financial terms disclosed. For more information, read more ....

Funding deals are in chronological order by date.

$0 = No financial terms disclosed. For more information, read more...     

Mergers & Acquisitions continue to be made for both medical device (6) and pharma/biotech (7).  

Acquisitions are in chronological order by date with Medical Device/Diagnostics followed by Pharma/Biotech.

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About BioMarketing Insight

We help companies de-risk their product development process by conducting the business due diligence to ensure that it is the right product for the right market and the market opportunity for the product meets the business goals of the company. We can then develop marketing strategies to drive adoption for the product.

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