Monthly Archives: October 2008

Leukaemia – 60 years of progress – its what we work for.

I have just returned from most engaging presentation being held at the Nowgen centre in Manchester, the meeting entitled Leukaemia 60 years of Progress was a look a the progress we have made in treating Leukaemia in the past 60 years and I found it a massive boost to my moral and why I was attracted to working in this industry in the first place.

The Presentation was made by Dr Emm Barnes and Professor Tony Whetton, as part of the Our Kid Medical Manchester celebrations.

The story starts with a staggering fact, in 1948 the survival time from diagnosis to death was 3 days. But in 1948 a Dr Sydney Farber reported the first ever remissions from drug therapy in in the New England Journal Of Medicine, this is considered the birth of modern anti-cancer drug therapy.

The Medical Research Council were quick to support British Researchers such as Henry Basil Marsden from Manchester, in taking up and researching these new technologies, and Edith Paterson so set up the worlds first cancer registry a lead that remains in place today.

Childhood Leukaemia has always being a disease that has attracted alot of attention, the reason for this is clear, following on from the nuclear bombs benign dropped on Nagasaki and Hiroshima, there saw a massive increase in the incidence of Leukaemia in the local child population that survived the initial attacks. There was general fear that the increased use of Nuclear power and weapons discharging radiation into the atmosphere that there would be a huge upsurge in Childhood Leukaemia globally.  Also at this time there was an upsurge in diagnosis of the condition, following improvements in Antibiotic therapies meaning that children were surviving infections and the underlying condition was then being diagnosed.

In the 1960′s and 1970′s the first specialist services for children with cancer were rolled out in the NHS, after a decade of research demonstrated that children who were in specialist centers survived 2.5 times longer.

In the 1970′s the survival in childhood leukaemia stopped being reported in time and started being recorded in percentages, this was a significant milestone in the treatment of this disease, as 50%  has a chance of survival. Issues of long term care started to become important, such as social stigma, employment and insurance problems.

1980′s there was still no standard treatment, (there is still no standard today) but all patients were treated in what is basicaly an ongoing clinical trial, with enrolment being at 80 to 90%, we are currentley at stages 14 of this ongoing programme.

1990′s there were leaps forward in survival and masses of progress in reduced side effects. Gleevec was discovered and developed.

2000′s it has now being discovered that there are 100 different genetic mutations that cause leukaemia, so it is 1 disease of 100 diseases with common symptoms, these are the questions that are facing researchers now and the progress of personalised medicine is starting to define how research will move forward in the future. Gleevec was also launched in 2001, this drug revolutionised therapy, however it is not a cure but a treatment and as with antibiotic therapy we are now starting to see patients develop gleevec resistant leukaemia.

In summary in 60 years of medical research we have gone from a survival time of three days to stage where patients are held in remission almost indefinitely, in this most complex genetic disease.

Damien Bové works as a drug development consultant (pharmaceutical or biotechnology), we work with our clients to define a development target, define a development strategy, define a regulatory strategy or define a commercial strategy. Our clients are generally raising funds or looking to license out their technology and we help them achieve it. If you want to know more don’t hesitate to get in touch using the contact form or email Damien at damien.bove@idaconsultants.com

Adaptive Clinical Trial Designs

The days of simple clinical trials are numbered, recent history has shown a steady increase in the duration, complexity and overall cost, this has been exemplified by a continued decrease in the number of New Molecular Entities Being approved by the FDA over the past few years.

Clinical trials have increase in duration by approximately 10% in the last 5 years, the number of patients and the number of interventions per patient are steadily increasing, this has lead to a steady increase in per patient costs and overall trial costs increasing. In addition to this phase III clinical trial failure rates have increase by 10% over the past 5 years.  The overall result of these trends has been the gradual reduction in the return on R&D investment. The industry has been looking for a new way of doing things and adaptive clinical trial design has been providing some of the answers.

Adaptive Clinical Trials offer the following benefits.

Reduced attrition

  • Careful selection of drug candidates and target indications
  • Disposal of unsuccessful products earlier
  • Working closely with regulatory agencies.

Optimized clinical trial design

  • Minimizing, duration, complexity, and size,
  • without compromising quality of data output

All done via

  • Dose optimisation
  • Patient selection and sub-grouping
  • Placebo/comparator utilization

The regulators are keen for companies to pursue development with these techniques:

Janet Woodcock, Director of FDA’s CDER, in 2006, “improved utilization of adaptive and Bayesian methods” could help resolve low success rate of and expense of Phase 3 clinical trials.

There are a number of different types of Adaptive Clinical Trials: the thing that adaptive trials have in common is that you are developing your trial as you progress, altering the dose of patietns, the mix of patient types, changing or cancelling treatment arms but all done by design. Adapting your design as you progress means you can optimise in response to the data generated.

Types of Adaptive Trial

  • Stopping early (or late) for efficacy or futility
  • Dose finding (& dose dropping)
  • Seamless Phases
  • Indication Finder
  • Adaptive randomisation
  • Adapting treatment to population
  • Modifying accrual rate.

You can’t just jump in with adaptive trial designs there is allot more preparation that is required, this preparation time should be rewarded in the long run many times over.

Methods

Lots of modelling is required to ensure your designs are robust, you can get a feel for how your adaptations will impact the outcomes and which outcomes are going to drive your decisions and adaptations, and that you have all the logistics in place that you need to support this chaning trail.

The data needs to be gathered quickly and reviewed, often prior to cleansing (reconsilation required later), ongoing statistical analysis needs to be taken care of and a data monitoring board needs to be onbaord and capable of dealing with this fast flow of data.

Also logistics needs to be managed flexibly, doses can change, recruitment rates can change, patient mixes can adjust and different clinical centres need to be responsive.

If you would like to know more how these programmes can help you speed up your development, reduce your costs and manage your risks just get in touch.

Damien Bové works as a drug development consultant (pharmaceutical or biotechnology), we work with our clients to define a development target, define a development strategy, define a regulatory strategy or define a commercial strategy. Our clients are generally raising funds or looking to license out their technology and we help them achieve it. If you want to know more don’t hesitate to get in touch using the contact form or email Damien at damien.bove@idaconsultants.com

Drug R&D Licensing Economy was US$340bn in 2007

Most drug discovery and development companies rely on licensing to fund their technology development programmes, either directly or as an exit for investors. We have complied a report that represents an attempt to put a figure on the amount of money that is circulating in the drug discovery and development economy. (full report available see bottom of article)

Our research led us to believe that:

The appetite for deals appears to have grown over the past 10 years but stabilised in recent years and this trend is followed by most of the individual indication groups.

The amount of money circulating in the drug discover and development economy in 2007 was roughly US$340bn, the level is pretty much the same as 2006, but an increase from 2005.

chart1 Drug R&D Licensing Economy was US$340bn in 2007

Get the full report!

This report makes interesting reading and should you wish to view the report in full, just drop a message in the contact form below with your e-mail and I will send you a password so you access the pdf from the reports section of the site (see top link). The report contains comparisons by indication group on deal volume and deal values with a brief outline of how we came to the global figure.

Damien Bové works as a drug development consultant (pharmaceutical or biotechnology), we work with our clients to define a development target, define a development strategy, define a regulatory strategy or define a commercial strategy. Our clients are generally raising funds or looking to license out their technology and we help them achieve it. If you want to know more don’t hesitate to get in touch using the contact form or email Damien at damien.bove@idaconsultants.com

Saving the World at Work – Book Review

saving the world at work Saving the World at Work   Book ReviewA new book by Tim Saunders is always a source of excitement for those who know of Tim and his work, and this one is no exception.

Tim has produced what can best be described as a prescription for corporate harmony with mankind and the planet. The books lays out what individuals within companies and corporations can do to save the planet and drive their organisations profits through the roof.

The book presents evidence that a business revolution is un derway and that revolution is a social responsibility revolution. Like the quality revolution that preceded it, those companies that embrace it now are going to prosper and those that don’t are going to shrivel and shrink.

Social responsibility is going to save the planet but also generate huge profits. The book is well argued and well written, it does not preach it just presents a well argued and well researched case and then presents a whole raft of actions that can be implemented.

Read this book it will change the way you think about your business, and what it can do for the planet.

For more information visit Tim’s Website and blog, but whatever you do read this book, and whilst your at it read Tim’s other books, Love is the Killer Application, and The Likeability Factor.

Five stars *****

However as I always say Business Books are only worth reading if you read plenty of them.

Damien Bové works as a drug development consultant (pharmaceutical or biotechnology), we work with our clients to define a development target, define a development strategy, define a regulatory strategy or define a commercial strategy. Our clients are generally raising funds or looking to license out their technology and we help them achieve it. If you want to know more don’t hesitate to get in touch using the contact form or email Damien at damien.bove@idaconsultants.com

Paediatric Investigation Plans – New Regulations

New regulations have come into force relating to the development of drugs for Children, and these regulations will impact on every single drug development programme currently ongoing. It is critical that you know about these regulations and how to adapt to them.

Every product must have agreed a Paediatric Investigational Plan (PIP) with the EMEA (via the PDCO – Paediatric Committee) before they register their product. In the future this will change to every product must be implementing a PIP before they can register their product.

The PIP describes the investigation that you propose to do in order to establish safety and efficacy in a paediatric population. You must put this plan together with the expectation of carrying it out. Every product must have one, it will not be acceptable to just say “children don’t get this disease” you will have to prove it.

The committee will not give a waver for any technology that can not prove that their therapy could benefit children. If there are enough Paediatric Patients to power a comparison study it will have to be undertaken.

If you would like to know more about these rules have a look at the this website.

We will be publishing more information on this in the near future.

Damien Bové works as a drug development consultant (pharmaceutical or biotechnology), we work with our clients to define a development target, define a development strategy, define a regulatory strategy or define a commercial strategy. Our clients are generally raising funds or looking to license out their technology and we help them achieve it. If you want to know more don’t hesitate to get in touch using the contact form or email Damien at damien.bove@idaconsultants.com