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Single Patient Use of Investigational Anticancer Agents:

Single Patient Use of Investigational Anticancer Agents:. An Industry Perspective Gerard T. Kennealey, MD Vice President, Clinical Research, Oncology AstraZeneca Pharmaceuticals December 14, 2000. Rising Public Expectations Make Earlier Access Necessary.

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Single Patient Use of Investigational Anticancer Agents:

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  1. Single Patient Use of Investigational Anticancer Agents: An Industry Perspective Gerard T. Kennealey, MD Vice President, Clinical Research, Oncology AstraZeneca Pharmaceuticals December 14, 2000

  2. Rising Public Expectations Make Earlier Access Necessary Patients are better informed than ever before: • Worldwide web • Patient advocacy groups • Media coverage (health and medicine, including cancer, is the single most covered issue in the general media.)

  3. Single Patient INDs Provide Needed Access to Promising Experimental Anticancer Compounds: • Usually initiated near NDA submission • Extremely low prevalence diseases where there may no other therapeutic option • Unresponsive tumors • Responsive tumors that have exhausted all standard therapies

  4. Initiating the Single Patient IND • Substantial activity • Safety data sufficient for filing • Standard therapy exhausted • Positive risk benefit ratio Single patient INDs are a useful tool in meeting patients’ needs:

  5. Additional Treatment Approaches Are Needed • Patients with more common tumors are exhausting currently available approved and experimental treatment options • The sheer number of patients who meet this description requires a more efficient means of meeting their legitimate medical needs

  6. AstraZeneca ExperienceZD1839 (Iressa™) • EGFR tyrosine kinase inhibitor • Orally bioavailable, once daily dosing • Phase I clinical trials begun May 1998 • AACR presentations May 2000, including plenary session showed activity • AACR and institutional media focus

  7. Targeted therapies offer best hope for beating cancer The drug unexpectedly shrank tumors and prolonged survival in a first stage study, where efficacy is rarely shown.

  8. Initial Plan: Initiate Phase III Trials • Since AACR, AstraZeneca’s information center and various patient and professional groups have received more than 7500 inquires about the compound • Calls from elected representatives, celebrities, and other high profile individuals expecting “instant access” were received every week

  9. Expanded Access Program (EAP)Previous Precedents • Treatment IND, parallel track, and compassionate use programs for HIV/AIDS drugs, e.g., ddI • The first such Expanded Access Programs in oncology, including Herceptin and STI 571

  10. Principles: Iressa Expanded Access Program (EAP) • Driven by clinical data in ~300 patients (NSCLC) • Patient safety is primary concern • Equal access throughout the world • No special cases – backing of senior management • Drug supply a potential issue • No interference with registration program

  11. Registration Is The Best Route To Expanded Access • Patients • Physicians • FDA • Pharmaceutical company

  12. The Iressa (EAP): Initial Steps • Create dedicated EAP Team • Involve FDA Oncologic Drugs Division and Cancer Liaison Office • Inform and collaborate with patient advocacy groups eg. ALCASE • Identify experienced CRO • Collaborate with National Organization for Rare Disorders (NORD)

  13. Iressa EAP Protocol • Restricted to patients with NSCLC • Disease that failed to respond to or relapsed on standard therapy • No concomitant therapy for tumor • Not a candidate for registration trials • Adequate general health • Informed written consent

  14. EAP Challenges • Create effective communication pathways • Create one informed consent document acceptable to all • Determine what data must be collected, recognizing that safety data allows for only minimal safety signalsEACH IS A BARRIER TO CORPORATE PARTICIPATION

  15. EAP Concerns • Potential for impact on registration trials • Potential impact on submission • Adequacy of drug supply in view of rapidly expanding clinical trial programs • Demand created by the media • Insuring equity • Other tumor types

  16. Focus corporate talent, energy, and resources on a promising compound beyond registration trials Develop productive ties with partner groups, e.g., FDA, patient and professionals Potential for additional safety data at time of approval and launch Reinforce commitment to timely, well controlled clinical trials EAP Opportunities

  17. Implications for the Future • Cancer drug development now operates in the public eye • An Expanded Access Protocol should be considered to meet patient needs, particularly those with advanced disease • Successful programs require commitment from all parties involved

  18. FDA Guidance Needed To ensure appropriate understanding, FDA may wish to offer guidance on these unresolved issues: • The role of these data in NDA filings • Potential opportunity for early registration

  19. Thank You • 150+ patients enrolled in this program • AstraZeneca EAP Team • National Organization for Rare Disorders • Patient Advocacy Groups • Patty Delaney & the Cancer Liaison Office • Oncologic Drugs Division • Mark Krueger & Associates, Inc

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