Dr Craig Rayner

CEO – d3 Medicine "Creative thinking must not be restricted by complexity and dogma"

http://news.yahoo.com/fda-approves-roches-tamiflu-infants-flu-symptoms-190834755–finance.html

An exciting day.

Today the FDA approved Tamiflu as the first anti-viral that may be used to treat influenza in children under the age of one year.

According to the CDC, infants are especially vulnerable to developing complications from influenza and those 6 months of age and younger have the highest rates of hospitalization.

Conducting clinical trials in such vulnerable patients is extremely complex, yielding low patient numbers, and requires novel approaches in order to identify dosing recommendations.

In this case, it was through the longstanding (since 2006) successful collaboration between NIH Collaborative Antiviral Study Group (CASG) and Roche. Each group conducted  dedicated clinical studies, yielding a total of 135 children under 1 year old with confirmed cases of the flu. These investigations provided safety information for Tamiflu in infants, and innovative clinical pharmacology strategies were applied to assist with defining dose recommendations.

 

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Thinking about the finishing line earlier! – Value Focused Development

When we are engaged in conversations about developing medicines, how often do you hear language thrown around, such as “capturing the value”, “fast to the next value inflection point” or words to this effect?

 

Drug development is a complex and risky business where remuneration for value creation occurs, by analogy, by passing the baton from one relay runner to the next.

It should come as no surprise, that all engaged in the broad church of “medicine development”, seek to create value and be paid, and that this mostly involves attention placed on needs of their adjacent customer, i.e. the next baton change.

For example, an academic innovator desires to capture the interest of an Angel investor or venture capitalist (VC) to finance their invention. A VC requires a multiplier, often over a short time, to satisfy their institutional investor customers. A biotech needs to woo the interests of a pharma transaction. A pharma needs to create a compelling case for registration, and crucially, reimbursement.

And somewhere in the distance out of sight, is the relay’s finishing line, the needs of the patient and society.

Now, the drug development process with multiple baton changes might just work seamlessly, if only everyone had a common interpretation as to who the ultimate customer was, and what the customer actually valued. It would mean every stakeholder would perform just those key development activities that focused on the value creation desired by that end customer.

However, the truth is the interpretation of what constitutes value gets ever more murky, the greater separation between stakeholders in the drug development process.

This information asymmetry creates important misalignments, where key questions are sidelined as other less critical issues are addressed resulting in financial and time wastage.

Indeed, it is not uncommon for a licensor to have to expend substantial resources and suffer serious delay repeating parts of the development programme for in-licensed drug candidates.

Additional value could have been captured by the licensee had they known what their customer actually wanted. In some cases, decisions to terminate programs due to insufficient patent life highlight systemic failures, resulting in important medicines never reaching patients in need. This is exactly analogous to dropping the baton!

It is important to ask seeminlgy innocuous questions like “what is value?” and “what does value focused development mean to you?” to the various sectors involved in developing medicines.

By doing so, one can begin to recognise both the divergent and shared perspectives on these key questions from stakeholders including the payor, pharma, biotech, life-science investors, academia, not-for-profit developers and entrepeneurs.

By increasing awareness and reducing information asymmetry, it may be possible to have stakeholders develop an shared view of each other’s requirements and importantly a more consistent view of the finishing line. Perhaps via better alignment, cross sector collaboration can be improved and waste reduced, increasing the likelihood that new medicines will reach patients.

 


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Creating value for society is not only measured in terms of publications and grants

On 28th Nov 2012, Senator Evans announced, on behalf of the Australian government, the National Research Investment Plan.

Does this represent getting the ball rolling?

The plan states a commitment to “deliver a strong, cohesive research fabric”. I sincerely hope this means there will be renewed focus on developing a stronger “research and commercialisation ecosystem” than what we have today in Australia. If so, then this provides hope that we may be able to attract back to Australia and also retain, more of the experience necessary to help the nation better captialise on our world-class science.

And then there is the Strategic Review of Health and Medical Research in Australia, with the McKeon Review being finalised this month.

Will either of these reviews improve Australia’s biotech ecosystem?

I hope so.

Is there enough recognition that a key training ground for developing and commercializing medicines is the Biotech and Pharmaceutical industry? Some skills can only be obtained by “doing”.

Encouraging cross sector collaboration is key. Breaking down silos between academia, clinical practice and industry and encouraging easier movement of professionals across the sectors.

This works at a student and also senior practitioner level. One barrier to fluid movement across the industry and academic interface is the old “publish or perish” mentality. A greater awareness that “impact” and “creating value for society” is not only measured in terms of publications and grants.

An excellent example is the Monash University and Gates Inhaled Oxytocin program.This collaboration brings in the best and brightest, irrespective of sector, to be focused on finding a solution to the critical issue of postpartum hemorrhage.

Creating a new medicine is never a slam dunk. We wish it was. This means academic investigators engaged in such programs, based on the old “publish or perish” mentality risk their publication records whilst pursuing these potentially life changing areas. Should they be trading off career versus impact to society?

Extend this argument now to innovators who have left to academic environments to work on life changing medicines in biotech and Pharma.

We need the Australian research ecosystem to encourage, not discourage cross-sector collaboration. I hope the outcome of the reviews above address this mind-set.


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Stranded Sea Turtles Could Help Australia Transform Locally Discovered Molecules into Global Medicines

How many of you had seen the articles Smart Country Sells Itself Short and Australia Blind to the Innovation Boom – Beattie published in the Sydney Morning Herald on 10th November?  Well, ironically, I was first made aware of these articles by three antipodean, but internationally stranded sea turtles!

The article titles themselves paint a view that is shared by many ‘international’ Australians who, having left for foreign shores at various stages of their careers, are faced with the desire of returning home; it hits a particularly raw nerve with those of us closely involved in the development and commercialisation of medicines.

The strengths of Australian science, discovery and optimisation of potential new drugs, are appreciated here at home, and also abroad. We celebrate our fair share of Nobel laureates and the discovery triumphs of brilliant scientists: Ian Frazer and his Queensland team’s cervical cancer vaccine discovery, the discovery of flu-fighting Relenza at CSIRO and Monash University; and world class research productivity from institutes like the Walter and Eliza Hall Institute of Medical Research that showcases Australia’s outstanding research focused medical fraternity.

So why is it that Australia sits 107th on the 2012 Global Innovation Index, behind Georgia, Malawi and Colombia?  As Peter Beattie highlighted, “Our basic research is world-class, our commercialisation is not”. Australia is viewed as a rich hunting ground for “early stage opportunities” for international medicine developers.  Real value capture in the life sciences space for Australia is lost when we sell our drugs too early in the development process. Rather, we should be managing risk and intelligently moving some of these early opportunities further along the development path. This requires “development and commercialisation” skills.

This process of “development and commercialisation” of a medicine, like rocket science, is extremely complex, expensive (>$1Bn), risky (<10% of drugs that eventually make it into humans reach the market), and it is definitely not an “individual” sport. It requires “teams” of dedicated, top minds tackling a problem from multiple perspectives in parallel. A typical core drug development team may have 10 or more highly qualified representatives (most with doctoral degrees, and many with multiple qualifications across disciplines)   from diverse functional areas, each supported by many others. Roles include medical development, clinical pharmacology, clinical safety, toxicology, pharmaceutical science, project management, operations, commercial, intellectual property, modelling and simulation, epidemiology, regulatory affairs, health economics as well as very smart project leadership. For a molecule to become a medicine, each of these elements must come together and be integrated successfully.  A seasoned “medicine developer” is someone who has not only deep technical knowledge, but importantly, an ability to integrate knowledge (and to ask for expert help as required) gained from substantial experience working within cross-functional development teams  solving development problems. The process of applying science in this way is not a discipline which is taught in Universities, but rather is a skill and art which is only learned by experience.

In contrast to US and Europe, this profile of a development professional is under-recognized, under-valued and under-resourced in Australia. A major reason is Australia’s lack of critical mass in this area, a fact which unfortunately drives abroad Australians seeking to pursue leading edge experiences in drug development and commercialisation. A seldom celebrated fact is many Australian’s do incredibly well in these environments. Some have established themselves internationally as leaders in their respective fields, making significant contributions to human health. In fact, the next time you open a medicine’s patient information leaflet, you should reflect that it is quite likely an Australian abroad had a hand in creating the product. Unfortunately, when after years away, such talented Australians ask themselves the question, “What opportunity could I return home too?”, the answer is generally silence.

Can we fix this? What really strikes me, is the chicken and egg problem that we have in Australia.

On one hand we bemoan the fact that we are not good at commercialising the discoveries from our brilliant scientists. I would argue, that an important driver is lack of critical mass in “development and commercialisation” experience in Australia. On the other hand, we actually have a bale (thanks Wikipedia) of sea turtles with critically relevant experience wanting to come home. China has recognised the value of its diaspora, encouraging the return home of internationally experienced scientsist: Australia should do the same.

Now is the time for Australia to be investing in our success beyond the mining boom.  I believe an important challenge for Australia, is determining how we can best access such “development and commercialisation” talent to support the existing strong discovery and innovation base our life sciences industry is reputed for.  However, retaining the talent will also require creating the right soft infrastructure for the sector. Bring our turtles home but make sure they have any environment in which they may thrive!

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Dr. Craig Rayner BPharm BPharmSc(Hons) PharmD MBA MAICD
Adjunct Associate Professor, Monash Institute of Pharmaceutical Sciences, Monash University
Director of Clinical Pharmacology, and previously Global Due Diligence Director at Roche

Personal Note: After many years abroad, I was extremely fortunate to be able to continue a global role in an International Pharmaceutical company working from my home in Melbourne with teams based in Europe, US and China. What amazes me, is that incredibly talented Australian’s are peppered throughout the international industry.  If we could gather them all up and bring them home, I am sure we could assemble a faculty that could rival any major Pharmaceutical company.