Medicines- from Trial to Market View from Pharma

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					       PROCUREMENT &
    DISTRIBUTION INTEREST
           GROUP

    Autumn Symposium 2007

http://www.pdig.org.uk/
                    Medicines- from
                    Trial to Market
                      View from
                        Pharma


      Dr David Gillen
     Medical Director
         Pfizer UK
Coventry November 8th 2007
                                      2
The second Gap in Translation –   Cooksey 2006




                                                 3
          The Future Challenge: Demographics!
As the population ages healthcare costs grow rapidly
                                                                      Growing burden of age
                              12
                                        US
Healthcare cost relative to




                              10        Canada
                                        UK
   50 - 64 age group




                                        Australia
                               8
                                        Japan
                                        Germany
                               6        Sweden
                                        Spain
                               4

                               2

                               0
                                    50-64                   65-69                          70-74                          75-79                            80+
                                                                                    Age Group
                              Older people consume more healthcare than younger people do
                                                                                                                                                  Eye-4-Pharma,
                                        Sources: Laurence Kotlikoff and Christian Hagist, “Who’s Going Broke?” National Bureau of Economic Research, Working      March 2007
PricewaterhouseCoopers LLP              Paper No. 11833, December 2005, p.25; World Factbook, 2006; OECD Health Data 2006; IMS Sales Data 2005.]                      Slide 6

                                                                                                                                                                                4
Medicines Extend Quantity and Quality of Lives
Drop In Death Rates For Diseases 1965-1996




        Disease                                          Treatment


      Atherosclerosis    Statins, ACE inhibitors, beta blockers, nitrates                 74%


     Ulcer of Stomach
                         H2 blockers, proton pump inhibitors                             72%
       and Duodenum


            Ischemic
        Heart Disease
                         ACE inhibitors, beta blockers, nitrates                   62%


          Emphysema      Anti-Inflammatories, bronchodilators                57%


                         Anti-Hypertensives,
       Hypertension      diuretics                 21%


                        0%                   20%                     40%     60%           80%

                                                                DEATH RATE
   Source: Lasker/Funding First

                                                                                                 5
       Yet…New Product Development Is a
       Risky and Expensive Proposition

                                     Years
                                                                                  Compound
                                                                                  CompoundSuccess
                                                                                              Success
                                                                                    Rates by Stage
                                                                                    Rates by Stage
                                      0
                                      0
                                                                                        5,000–10,000
                                      2
                                      2                                                 5,000–10,000
                                                                                           Screened
                 Discovery                   Preclinical Testing                          Screened
               (2–10 Years)                  Laboratory and
                                      4
                                             Animal Testing                                   250
                                                                                               250
                                      6
                                      6                                                     Enter
                         Phase I                                                     Enter Preclinical
                                                                                      Preclinical Testing
  20–80 Healthy Volunteers Used to           Phase II
     Determine Safety and Dosage      8
                                      8      100–300 Patient Volunteers
                                                                                            Testing
                                             Used to Look for Efficacy                          5
                       Phase III      10
                                      10     and Side Effects                                 Enter
    1,000–5,000 Patient Volunteers                                                       Clinical Testing
          Used to Monitor Adverse
       Reactions to Long-Term Use
                                      12
                                      12
                                      14
                                      14     FDA Review Approval
            Additional Post-                                                                    1
           Marketing Testing
                                      16
                                      16                                             Approved by the FDA

                                                                               Net Cost: £400 Million
                                                                              Invested Over 15 Years
Sources: 1) Increased Length and Complexity of the Research and Development Process. Chapter 1 in: PhRMA Pharmaceutical
Industry Profile 2003. 2) DiMasi, JA, Hansen, RW, Grabowski, HG. The Price of Innovation: new estimates of drug development
costs. Journal of Health Economics. 2003; 22:151-185.                                                                         6
2.a, 2.b
 So much for so little…

                          50,000       Global R&D Spending ($ in MN)   NME and New Biologics Approved     60
                          45,000




                                                                                                               No. of NME, BLA Approvals
                                                                                                          50
 R&D Spending ($ in MN)




                          40,000
                          35,000
                                                                                                          40
                          30,000
                          25,000                                                                          30
                          20,000
                                                                                                          20
                          15,000
                          10,000
                                                                                                          10
                           5,000
                              0                                                                           0
                                   1995 1996 1997 1998 1999 2000 2001 2002 2003 2004* 2005* 2006*

 Source: FDA/CDER Data, PhRMA data, Pw C analysis                                           * includes Biologics


                          Even allowing for inflation, the industry is investing twice as
                          much in R&D as it was a decade ago to produce two-fifths of
                          the new medicines it then produced.
                                                                                                                                           7
So what is the reality of this translation
and how can we improve it?




                                             8
For patients, standards are still held back by the slow
uptake of medicines
Share of products grouped by year of first sales in total non-generic pharmaceutical
sales (%, 2006)

 Year of first sales

                   100%
                                                            20                18                 17   15    16
 Post-2002                                23

                                                                                                      28    24
                                                                              33                 32
                                                            33
 1998-2001                                36



                                                                                                      56    60
                                                           47                 49                51
 Pre-1998                                41


                                     Spain                 US          Germany France                 UK   Italy


         NOTE: IMS data includes parallel trade for the UK, but excludes it for all other countries
         See appendix for additional analyses on uptake
 Source: IMS Health, team analysis                                                                                 9
Relative survival in All Malignancies in
Europe- Lancet Oncology 2007




                        …UK relative survival below the
                        Mean Eurocare Region 47 % men
                        55 % women….

                        Lancet Oncology 2007 8 784-796




                                                          10
Sunitinib- An Oral Multitargeted Tyrosine
Kinase Inhibitor




                                            11
Sunitinib- An Example of the challenge in
the UK




            Professor Bob Hawkins from Christie hospital said:
          "We would like [Sutent] to be available for every patient.
             "We would still encourage patients to go into the
                          trials of new treatments
             because that's the only way we're going to make
                           progress on drugs like
                                    Sutent.



                                                                       12
Compassionate Use



   Must Adhere to
   Regulatory
   Requirements




                    13
Use of unlicensed medications


    An unlicensed relevant medicinal product may
     only be supplied to third parties if it is:
      supplied in response to a bona fide unsolicited
       written request;
      formulated in accordance with the requirement
       of a doctor or dentist registered in the UK;
      for use by their individual patients on their
       direct personal responsibility;
      supplied under specific conditions

                              MHRA Guidance Note 14 2006



                                                           14
  Phase III b study for Sunitinib
  Renal Cancer UK Globally highest recruiter


To be enrolled                              n = 5,000


Enrolled as of 1 May ‘07                    n = 4,470
                                                                  ITT population
                                                                  Safety
Data available                               n = 2,341            Treatment duration
                                                                  Response rate

With potential FU >6 mo                                          PFS
                                            n = 2,125
(incl. PD / )                                                   OS



                           No prior cytokines:           Prior cytokines:
                                 n = 285                     n = 1,840


                                                          Subgroups
                                                                                       15
Sunitinib- NICE review due 2009




                                  16
Oncology: A Particular Challenge for HTA


1.   HTA is limited in its application to end stage oncology in large
     part because of the nature of these conditions.
        QALY assessments do not capture the true value of therapy and
         fail to weight survival gains relative to life expectancy.
        Conditions are rare so share many of the limitations presented by
         the application of HTA to orphan products, data scarcity and
         uncertainty, necessarily high costs plus associated high budget
         impact.
2.   Cancer treatment evolves incrementally. This has produced
     significant improvements in survival beyond what is seen in
     the Phase III trials of existing products which can not
     efficiently be achieved through formal trial programs. Denial
     of access obviously stifles this evolution
3.   The UK now has one of the lowest uptakes of new oncology
     products in Europe.


                                                                             17
Moving forwards- Is there a different way?



      Drug Development and the opportunity of In-
       line Real World testing


      Risk Sharing

      HTA reform




                                                     18
The future - integrated healthcare?


                        Mainly biologically based
                        Based on clinically validated
                         target(s)
                        Targeted to specific disease
                         populations
                        Disease modifying
                        Compliance and persistence
                         monitored and ensured
                        Prophylactic treatment of
                         susceptible
                        Supported by a network of services




                                                              19
Drug Development and the opportunity of
In- line Real World testing




                                          20
Risk Sharing : Velcade (Bortezomib)



    Britain's NICE (on Wednesday 24th Oct) published final
     guidance to the National Health Service (NHS) on the funding
     of Janssen-Cilag's Velcade (bortezomib) in multiple
     myeloma, including the introduction of a novel risk-sharing
     scheme.

   In a statement, the cost-effectiveness watchdog said:
 "The guidance confirms the response-rebate scheme which will
   allow patients at first relapse who show a full or partial
   response to bortezomib to carry on with the treatment, fully
   funded by the NHS, and patients who show no or minimal
   response to be taken off the drug and the drug costs
   refunded by the drug's manufacturer."




                                                                    21
Pfizer principles of HTA (1)


   1. The views of physicians and patients are an
    essential component in assessing the value of
    medicines and healthcare decision-making
       HTA should formally incorporate physician and
        patient views
       Guidance should recognise that patient preferences
        and responses to medicines vary and thus permit
        physicians to prescribe contrary to guidance without
        undue burden to justify their decision

                            Alzheimers –
                          limit treatment to
                              moderate
                               patients

                                                               22
Pfizer Principles of HTA (2)

 HTA should reflect the full value of medicines to UK society
    HTA should focus on cost and benefits of new
      medicines to society rather than the NHS alone
    HTA needs to recognise that the value of medicines
      takes many forms and incorporates all these
      dimensions rather than focus solely on clinical
      outcomes
    The development of new healthcare technology
      requires that HTA appraisals recognise and reward
      incremental innovation
                                    Alzheimers – Residential
                                       care costs borne by
                                        patients and their
                                     families were excluded
                                        in the Alzheimer’s
                                             appraisal


                                                                23
Pfizer Principles of HTA (3)

 HTA, especially close to launch, requires a pragmatic
   approach to assessing evidence and developing
   guidance

      The real value of a medicine will only be confirmed
       after launch following sufficient and appropriate use
       in a real world setting
      Uncertainty about treatment effect and economics
       will always exist. HTA needs to ensure that risk
       averse decision making does not stifle innovation
       and deny patients access to medicines
                                               Single Technology
                                             Appraisals – The need
                                             for pragmatism is not
                                               explicitly stated in
                                             guidance to evidence
                                                 review groups

                                                                      24
Pfizer Principles of HTA (4)


      4. The reliability of economic analyses need to be
       explicitly considered within the decision making
       process
       Measurement and valuation methods are evolving but
          remain imprecise
       Models are only as good as the underlying structure
          and assumptions
       Models often contain a high degree of uncertainty
       Models can be relatively unstable – a small change in
          one or more parameters can lead to very different
          results and conclusions

           The Alzheimer’s Disease economic model - assumptions and
        parameter estimates that do not reflect current practice, resource use
                                     and costs.


                                                                                 25
Pfizer Principles of HTA (5)

   .    HTA must include an open, transparent decision making
        process that involve all stakeholders, covers all health
        technologies, makes full balanced use of all evidence,
        contains a fair appeal process, and makes decisions in
        a timely manner.

       Manufacturers are not represented at the appraisal committee hearing.




        The appeal process focuses on whether process has been followed




         NICE do not engage with manufacturers during drug development
                      process to discuss data requirements.



                                                                               26
Pfizer Position on HTA

   The UK government should ensure that it receives
    value for money from the medicines it procures
    through the NHS. Current methods for assessing
    value for money through Health Technology
    Assessment (HTA) are limited.

       HTA increasingly undermines patients’ access to
        innovative new treatments
       HTA leads to uncertainty in the drug development
        process
       HTA risks undermining the future development and
        availability of new medicines.


                                                           27
Key issues during the transition from Trials to
licensed products



  The pharmaceutical industry needs
  to be a partner with not an
  adversary of the NHS and we must
  work with all stakeholders to allow
  the next generation of Innovative
  Medicines to be available …..




                                                  28
“ We try never to forget that medicine is for the
  people. It is not for the profits. The profits
  follow, and if we remembered that, they have
  never failed to appear. The better we
  remembered that, the larger they have been…”
              George Merck- Founder of MSD




                                                    29

				
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