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.