星期三, 4月 27, 2016


Highlights of the Day
Day Two of the World Medical Innovation Forum was brimming with fireside chats and panel discussions. The first session of the day highlighted the growing global cancer burden in which panelists from leading international companies and key government entities discussed the challenges to providing innovative technologies in diverse markets, as well as potential strategies to overcome these obstacles.

In the second panel, experts provided an in-depth discussion on the promise of epigenetics for cancer research, describing the complex science behind the regulation of how genetic information is processed and the development of therapies that leverage these alterations to target cancer.

Two panels focused on the promise of immunotherapeutic strategies for the treatment of cancer, highlighting the recent burst in the field of immuno-oncology. A set of fireside chats provided insights into drug pricing, data sharing, collaboration, and patient-driven innovation.

The final plenary session, moderated by CNBC Biotech and Pharma Reporter Meg Tirrell, saw a panel of oncology investors discuss some of the most compelling topics in the industry today, including the most promising technologies, investment trends, comparison to other periods in cancer investing and, naturally, drug pricing.

The day concluded with four concurrent focus sessions, which provided discussion around radiation therapy, oncology devices, study design for personalized cancer medicine, and the unique challenges posed by increased cancer survivorship. For more in-depth highlights from some of the most interesting panels from Day Two, see below.

Notably, panelists demonstrated surprising candor when discussing their personal struggles with cancer, and seemed optimistic about the current trajectory of science, with the word "cure" being used more freely in the context of cancer and without reservations of this being a crazy notion, emphasizing the reality of working towards cures.


2016 World Forum

Global Cancer Markets
As the global cancer burden is expected to increase from 14 million to 22 million cases by 2030, panelists from leading international companies and a key governmental entity, namely the famed National Institute for Health and Care Excellence at the UK NHS, discussed the future of cancer in a global market. Moderator Sheila Dharmarajan, Head of Business Development at Zelnick Media Capital, started out by emphasizing the term "tremendous" to describe not only the prolificacy of cancer, but also to describe the complexity of the market, breakthrough technology, and the dollars at stake in terms of both pricing and cost of innovation.

When posed with the question of whether pharmaceutical companies should fund diagnostics, panelists acknowledged that, due to the innate differences in business models, each company must decide their own path, but Bruno Strigini, President of Novartis Oncology, also noted the importance of considering the development of targeted therapies with companion diagnostics, which can be critical to providing the right products to the right patients.

Additionally, Newton Crenshaw, Vice President of North America Oncology at Eli Lilly and Company, stressed the importance of symbiosis between diagnostic and therapeutic companies, and indicated that reform is needed for regulatory bodies to accelerate diagnostics and provide access to patients. Following up on this point, Gillian Leng, MD, Deputy Chief Executive and Director of Health and Social Care at NICE, stated that this relationship could be most effective if regulatory bodies were allowed to evaluate the drug and diagnostic together.

As technology advances and we begin to consider cancer to be a chronic rather than terminal condition, Mr. Crenshaw also posed the question of how to best support patients beyond medicine, but noted that the cost burden of long-term treatment must be considered, as well.

Immunotherapy: Checkpoint Activation, Cancer Vaccines, and Cell Based Therapies
Day Two featured two panels on the promise of immunotherapy in cancer treatment in which panelists discussed various approaches to leveraging the human immune system to fight cancer, including checkpoint inhibition, anticancer vaccines, and CARTs.

In the first session, panelists were cautiously optimistic about immunotherapy as a tool for cancer treatment and agreed that this wave of enthusiasm is still in its early stages and we must proceed by paying particular attention to the determinants of tumor response and resistance. In this context, a recurring message was the importance of tissue biopsy and biomarker analysis before and during treatment, which will be critical to the understanding of what is happening to patients at a molecular level and, as Scott Rodig, MD, PhD, Associate Professor at Brigham and Women's Hospital, mentioned, will be extremely important in the determination of rational drug combinations going forward.

Similarly, panelists also stressed that we should think carefully about how we are approaching combination therapy; rather than using a shotgun approach in which we try every drug combination, we should be deliberate in understanding the underlying mechanisms to predict optimal patient populations and avoid toxicity. More specifically, Thomas Daniel, MD, Chairman of Celgene Research, stated that we should be considering the patients for which combinations are medically valuable, along with a scientific grounding for why a specific combination makes sense, which is a more tractable and financially feasible option as compared to the shotgun approach.

David Reese, MD, Senior Vice President of Translational Sciences at Amgen, noted that a major challenge in the field at present is a need for the development of new surrogates for traditional response criteria; as patients live longer and overall survival becomes increasingly difficult to measure, some trial designs will essentially become intractable.

The second immunotherapy panel discussed what's next for CAR T-cell therapy, with Usman (Oz) Azam, MD, Global Head of Cell and Gene Therapies Unit at Novartis, referring to the current technology to as "version 1.0." Panelists discussed the promise of CART in hematological malignancies, but as Mark Frohlich, MD, Executive Vice President of Portfolio Strategy at Juno Therapeutics, pointed out, we must still determine the right targets for solid tumors.

When quizzed about the future of the technology, Dr. Azam also mentioned that gene therapy could be utilized to take CARTs to the next level, which Chuck Wilson, PhD, CEO of Unum Therapeutics, expanded upon by suggesting that the use of gene editing to target specific genes, create allogeneic T cells, or inhibit signaling pathways, could support the work of CARTs.

Dr. Wilson also reminded the audience that the reality is, as an autologous cell therapy, the logistics of taking cells from the patient, manufacturing cells, and returning them to the patient, will be inherently more costly than other therapies, but that scalability could bring this cost down. Additionally, as Marcela Maus, MD, PhD, Director of Cellular Immunotherapy at MGH, pointed out, the development of point-of-care methods or "off-the-shelf" T cells that could treat many patients with one lot are potentially less costly.

2016 World Forum

Fireside Chat Roundup
The first two days of the Forum were filled with fireside chats, which allowed for an intimate perspective on pricing, the importance of data sharing, academic and industry collaborations, current healthcare challenges, and much more.

Kicking off the first fireside chat of the Forum on Monday evening, Richard Gonzalez, CEO of AbbVie, sat down with Nancy Snyderman, MD, Medical Advisor, GE Healthymagination, to discuss:

• The acquisition of Pharmacyclics and the "blind dating process"

• "Doubling down on R&D" to bring a consistent stream of good medicines

• Catering to patients by "bringing improvements for standard of care" that the healthcare system would reward

• The most important model today of "partnering with academic and biotech"

Tuesday's lineup of fireside chats started with Amgen's CEO Robert Bradway and Bloomberg's biotech reporter Caroline Chen on a variety of topics including:

• The need "to collaborate outside of your organization, especially with cancer"

• The costly price tag that comes with innovation but "the need to focus on the benefit that it brings for society"

• Project Data Sphere, an independent and not-for-profit initiative of the CEO Roundtable on Cancer's Life Science Consortium (learn more here).

• A call to society to invest more in cancer prevention

With nine months left on the job, Andy Slavitt, Acting Administrator for the Centers for Medicare & Medicaid Services (CMS), spoke candidly with CNBC's biotech and pharma reporter, Meg Tirrell on:

• The need for new payment models because governments don't have "magic pots of money" and "we are still in the 1st and 2nd generation of pricing models"

• Finding new ways to care for the "most difficult people with the lowest income and are disconnected from the healthcare system"

• Pilot programs for cost containment and spurring innovation focused on "doubling down on primary care and prevention"

• The qualifications for the next CMS administrator - "doesn't need the job, doesn't see it as a stepping stone, doesn't need to make friends, and has struggled to find and provide care"

Praising the industry for its strides in finding important cures for cancer, Bristol-Myers Squibb CEO, Giovanni Caforio, MD, started off his chat with Meg Tirrell by highlighting three critical points to being a leading company in oncology: keeping the patient at the center, following the science and collaborations with industry and academia. Other topics included:

• The need for "different payment models and flexibility in the healthcare system"

• The "beginning stages of understanding why patients don't respond to immunotherapies"

• The need to focus on biomarkers as "they are really important"

Rounding out today's fireside chats, "patient scientist" Kathy Giusti, Founder of the Multiple Myeloma Research Foundation and Multiple Myeloma Research Consortium, spoke with Nancy Snyderman, MD, to discuss her journey:

• Being a patient advocate, patient and business disruptor who has elevated attention and propelled advancements for multiple myeloma

• The need for data sharing for research and cures

• The "tough lifting" that comes with trying to stay on top of the cancer landscape

• Partnership with Harvard Business School to "put together a business plan by which business and science could start disrupting" and "to bring the absolute best-in-class to ask the tough questions and to share and solve the problem"

• How she made the decision to stop her own treatment