Pharmaceutical Executive Europe - May 2008 - (Page 21) Pharmaceutical Executive Europe May 2008 Market Access 21 ● In other indications, PRO is not critical to approval, but does improve market access (e.g. reimbursement). ● Finally there are situations, where PRO allows the label to be broadened beyond the launch indication. Figure 3: Need to link claims with endpoints. Desired claim ‘Painbuster’ relieves pain quicker without upsetting your stomach Concepts of measurements Pain relief Time to onset Stomach upset Gl-symptom diary Measurement tools Pain diary Time to pain relief Use of PRO data for market access Figure 2 shows how market access issues are influenced by data from many different sources. The gold ovals are the data required to support labelling and advertising. Patient satisfaction and, generally, caregiver impact are not used in FDA-approved labelling but can be used to support advertising. The right panel shows the diversity of PRO collected outside clinical trials that helps other decision makers, including patients, understand the effects of a treatment. The FDA has produced ‘guidance’ on PRO to fit the science of PRO with regulatory needs.1 Although not a regulation, it is FDA’s current thinking. In Europe, the EMEA hasn’t produced a guidance but instead has published a ‘reflection paper.’2 Unlike FDA, the EMEA currently has no special group to aid in PRO review. Although there are some differences in approach, the bottom line is that companies must develop PRO early so they can be adapted, translated and validated. Development of PRO at the end of Phase II is too late. Outcomes people often working at the margins of clinical teams must be involved at Phase I or earlier. Even at pre-IND stage, a range of potential claims should be explored. PRO instruments developed without rigor will not stand up to scrutiny of regulators. Figure 3 illustrates how claims need to be linked to endpoints. Each element of the desired claim needs to be linked to concepts of measurement which are supported be measurement tools or assessments derived from them.3 Conclusion PRO is important for maximum market access. Although the regulatory hurdles are high, they are manageable. PRO success requires early planning and full integration into a clinical programme so that PRO measure focused concepts that match claims are delivered. PRO should be developed embracing the regulatory and access needs through discussion with organisations responsible for assessment and approval. They have to be validated for the populations who will be asked to complete PRO assessments and be carefully documented for regulatory review. Finally, communication and education strategies need to build on understanding information needs across all stakeholders. References 1. FDA 2006. Draft guidance for industry, “Patient reported outcome measures: use in medical product development to support labelling claims.” http://www.fda.gov/CDER/GUIDANCE/5460dft.pdf 2. EMEA 2006. Reflection paper on regulatory guidance for the use of healthrelated quality of life in the evaluation of medicinal products. www.emea.eu.int/pdfs/human/ewp/13939104en.pdf 3. Scott J., Drug Information Association. Annual Meeting, 2004 Use of PRO for advertising claims In the US, direct-to-consumer (DTC) advertising in magazines and on TV lends itself to the endorsement of ‘patient speak.’ As a result, the FDA is concerned about the images and words used in DTC ads, because once they are printed, they are there for ever. Words used in labelling can show up in DTC ads. This is a worry to the FDA because patients may misinterpret the meaning, for example, how many patients understand the true clinical meaning of ‘depression’? The FDA issues warning letters to cover a range of scenarios: ● exaggerated images of patients. One consequence of this is illustrated in a cartoon where the patient is saying to the physician “I think the dosage needs adjusting. I’m not nearly as happy as the people in the ads" (Smaller, Physician’s Weekly, 31 December, 2001). ● claims based on patient satisfaction surveys rather than ‘substantial evidence,’ such as adequate and well-controlled trials using validated instruments. ● lack of fair balance in presenting risks and side effects. About the Author Stuart Cooper (stuart.cooper@adelphigroup.com) is the CEO of Adelphi Group, which provides services in strategic marketing, business intelligence, health and economic outcomes research, market access, health communications and medical education. Stuart's professional focus in the last 20 years has been on global pharmaceutical developments — its success factors and the needs and demands for successful global brand development to optimise healthcare interventions throughout the life cycle. http://appliedclinicaltrialsonline.findpharma.com/appliedclinicaltrials/Regulatory+Articles/Grasping-the-FDAs-PRO-Guidance/ArticleStandard/Article/detail/365045 http://www.emea.europa.eu/pdfs/human/ewp/13939104en.pdf http://www.fda.gov/CDER/GUIDANCE/5460dft.pdf http://www.emea.eu.int/pdfs/human/ewp/13939104en.pdf
Table of Contents Feed for the Digital Edition of Pharmaceutical Executive Europe - May 2008 Pharmaceutical Executive Europe - May 2008 Contents From the Editor News and Analysis Calendar Rising in the East Use Your Strategic Discretion A Blueprint for Success Market Access and the Patient Activating Effective Product Differentiation What Doctors Want IC Success in Four Steps Pharmaceutical Executive Europe - May 2008 Pharmaceutical Executive Europe - May 2008 - (Page Bellyband1) Pharmaceutical Executive Europe - May 2008 - (Page Bellyband2) Pharmaceutical Executive Europe - May 2008 - Pharmaceutical Executive Europe - May 2008 (Page Cover1) Pharmaceutical Executive Europe - May 2008 - Pharmaceutical Executive Europe - May 2008 (Page Cover2) Pharmaceutical Executive Europe - May 2008 - Contents (Page 3) Pharmaceutical Executive Europe - May 2008 - From the Editor (Page 4) Pharmaceutical Executive Europe - May 2008 - From the Editor (Page 5) Pharmaceutical Executive Europe - May 2008 - News and Analysis (Page 6) Pharmaceutical Executive Europe - May 2008 - News and Analysis (Page 7) Pharmaceutical Executive Europe - May 2008 - Calendar (Page 8) Pharmaceutical Executive Europe - May 2008 - Calendar (Page 9) Pharmaceutical Executive Europe - May 2008 - Calendar (Page 10) Pharmaceutical Executive Europe - May 2008 - Calendar (Page 11) Pharmaceutical Executive Europe - May 2008 - Rising in the East (Page 12) Pharmaceutical Executive Europe - May 2008 - Rising in the East (Page 13) Pharmaceutical Executive Europe - May 2008 - Use Your Strategic Discretion (Page 14) Pharmaceutical Executive Europe - May 2008 - Use Your Strategic Discretion (Page 15) Pharmaceutical Executive Europe - May 2008 - A Blueprint for Success (Page 16) Pharmaceutical Executive Europe - May 2008 - A Blueprint for Success (Page 17) Pharmaceutical Executive Europe - May 2008 - Market Access and the Patient (Page 18) Pharmaceutical Executive Europe - May 2008 - Market Access and the Patient (Page 19) Pharmaceutical Executive Europe - May 2008 - Market Access and the Patient (Page 20) Pharmaceutical Executive Europe - May 2008 - Market Access and the Patient (Page 21) Pharmaceutical Executive Europe - May 2008 - Activating Effective Product Differentiation (Page 22) Pharmaceutical Executive Europe - May 2008 - Activating Effective Product Differentiation (Page 23) Pharmaceutical Executive Europe - May 2008 - Activating Effective Product Differentiation (Page 24) Pharmaceutical Executive Europe - May 2008 - What Doctors Want (Page 25) Pharmaceutical Executive Europe - May 2008 - What Doctors Want (Page 26) Pharmaceutical Executive Europe - May 2008 - What Doctors Want (Page 27) Pharmaceutical Executive Europe - May 2008 - IC Success in Four Steps (Page 28) Pharmaceutical Executive Europe - May 2008 - IC Success in Four Steps (Page 29) Pharmaceutical Executive Europe - May 2008 - IC Success in Four Steps (Page 30)
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