EMD Serono Position Statement on Responsible Clinical Trial Data Sharing For External Use

Transcription

1 EMD Sern Psitin Statement n Respnsible Clinical Trial Data Sharing Fr External Use Why it Matters EMD Sern believes that as a bipharmaceutical cmpany, the sharing f infrmatin related t cmpany spnsred Clinical Trials is central t ur missin. The sharing f clinical trial infrmatin enables the medical and scientific cmmunity t further develp the medical and scientific knwledge base and permits the public t make infrmed healthcare decisins. It is als ne f the best ways t infrm prescribers and patients abut scientific findings relating t ur medicines. We are cmmitted t enhancing public health thrugh respnsible sharing f clinical trial data in a manner that is cnsistent with: (a) safeguarding the privacy f patients (b) respecting the integrity f natinal regulatry systems and (c) maintaining incentives fr investment in bimedical research. EMD Sern Psitin Statement EMD Sern spnsred trials are thse studies in which ur cmpany is ultimately respnsible fr all aspects f the study, even if sme r all f these activities are transferred t anther party (such as a cntract research rganizatin). Clinical Trial Registratin What: EMD Sern registers the designs f all f its clinical trials in patients 1. Where: EMD Sern registers this infrmatin n the publicly accessible website maintained by the U.S. Natinal Institute f Health (NIH), and ther applicable websites as required. This allws healthcare prviders, patients and their families t easily lcate clinical trials. Hw: EMD Sern cmmits t assign each trial a unique identifier (e.g., a cmpany-assigned study ID) t ensure transparency and allw users t track the trial thrugh multiple databases, including clinical trial results databases. When: These clinical trials are registered befre the trial starts. Clinical Trial Results Disclsure What: EMD Sern disclses the results f all clinical trials in patients nce a prduct has been apprved fr marketing and is cmmercially available in at least ne cuntry, regardless f utcme. Investigatinal prducts whse develpment prgrams are discntinued, will als be disclsed, regardless f utcme. 1 The mst imprtant clinical trials are thse that test a medicine n subjects wh actually require medical care: patients. The results f trials such as these are integral t drug develpment, because they prvide medical evidence regarding the safety and effectiveness f medicines in the ppulatin intended t use the medicine. These are the clinical trials fr which EMD Sern cmmits t prviding registry and results infrmatin. EMD Sern is an affiliate f Merck KGaA Darmstadt, Germany.

2 Where: EMD Sern makes this infrmatin available n the publicly accessible website maintained by the U.S. Natinal Institute f Health (NIH), and EudraCT, maintained by the Eurpean Medicines Agency and ther applicable websites as required. Hw: In all cases, disclsures will be undertaken in a manner cnsistent with applicable natinal laws and rules gverning prtectin f patient privacy and intellectual prperty. When: EMD Sern disclses the results t clinicaltrials.gv within 30 days after apprval f the drug, r within 12 mnths after a trial ends, whichever ccurs first. Fr trials with marketed prducts that are terminated prir t cmpletin, infrmatin will be prvided n the date f cessatin and reasns leading t the decisin t discntinue the trial. Clinical Study Reprts - Fllwing apprval f a prduct and indicatin in bth the Eurpean Unin and the United States, EMD Sern will make publicly available Clinical Study Reprt synpses that were filed with regulatrs n r after January 1, Clinical Trial Participant Cmmunicatin - EMD Sern will supprt investigatrs in prviding their research participants with a summary f the trial results after cnclusin f the trial. In additin, EMD Sern will wrk with regulatrs t adpt mechanisms fr prviding a factual summary f clinical trial results and make the summaries available t study participants. Investigatr Access t Data and Review f Results - EMD Sern values the significant cntributin f each clinical investigatr withut whm advancements in medical science wuld nt be pssible. We seek t prvide ur investigatrs access t clinical data frm the studies in which they participate. Individual investigatrs in multi-site clinical trials will have their wn research participants data, and will be prvided the randmizatin cde after cnclusin f the trial. We will prvide a summary f the study results t the investigatrs. Any investigatr wh participated in the cnduct f a multi-site clinical trial will be able t review data fr the entire study at the spnsr s facilities, r ther mutually agreeable lcatin in respnse t a reasnable scientific inquiry. Investigatrs wh are authrs f study-related manuscripts will be given all study data needed t supprt the publicatin. Infrmatin Shared with Researchers Fllwing apprval f a new prduct r a new indicatin fr an apprved prduct in bth the Eurpean Unin and the United States after 1 January 2014, EMD Sern will share study prtcls, annymized patient level, and study level data and redacted clinical study reprts frm clinical trials in patients with qualified EMD Sern is an affiliate f Merck KGaA Darmstadt, Germany.

3 scientific and medical researchers, upn researcher request, as necessary fr cnducting legitimate research. Data will nt be shared fr prducts and indicatins apprved prir t the effective date f this Plicy. In additin, data will nt be shared with EMD Sern cmpetitrs. Publicatins All EMD Sern clinical trials in patients will be cnsidered fr publicatin in the scientific literature, regardless f utcme. In additin, this cmmitment als pertains t investigatinal medicines whse develpment prgrams have been discntinued. Frequently Asked Questins: 1. Questin: What Platfrm is EMD Sern using t share its clinical trial data? Answer: EMD Sern is registering the design f all clinical trials in patients as well as the summary results fr these trials n the website maintained by the U.S. Natinal Institute f Health (NIH), and ther applicable websites as required. Trial results fr an investigatinal prduct that has failed in develpment will als be psted t this website. In additin, EMD Sern has agreed t share annymized patient level clinical study data, study level clinical data and prtcls, t qualified researchers fr the purpse f advancing science and mst imprtantly, benefiting patients. Currently, we are sharing this data via ur webprtal. 2. Questin: Why hasn't EMD Sern jined the platfrm Clinicalstudydatarequest.cm like Bayer, Behringer Ingelheim, GSK, Nvartis, Rche, Sanfi and ViiV Healthcare?" Answer: There are several industry/academic/cnsrtia led platfrms being develped fr the purpse f Data Sharing. Like many pharma cmpanies, EMD Sern is evaluating these ptins. 3. Questin: Can yu prvide mre details abut the type f infrmatin EMD Sern is prepared t share with qualified medical and scientific researchers in respnse t a legitimate request? Answer: EMD Sern is cmmitted t sharing with qualified medical and scientific Researchers, patient-level data, study level data, and clinical study designs and prtcls. Patient-level data refer t infrmatin n individual patients cllected during a clinical study and recrded n case reprt frms (CRFs) and inputted int electrnic databases r captured directly in electrnic frmat, where it can be readily rganized int patient-level listings and datasets (e.g., demgraphy and baseline characteristics, expsure, medical histry, cncmitant medicatins, clinical EMD Sern is an affiliate f Merck KGaA Darmstadt, Germany.

4 efficacy, safety events and safety lab, bimarker and pharmacgenetic). Data gathered depends n the study prtcl (clinical study design infrmatin and prtcl). This infrmatin is handled thrugh what the Institute f Medicine (IOM) has described as a prcess by which data in a clinical study riginate with CRFs, either handwritten r electrnic, then g thrugh several stages f auditing, queries, and refinement by riginal investigatrs and study staff t reslve ambiguities, and then ultimately yield individual participant data. 2.. This is typically available by sharing the annymized raw and analysis datasets, tgether with the related metadata and the Statistical Analysis Plan. Study-level data cnsist f patient-level data that have been amalgamated, cmpiled and tabulated, transfrmed, stratified, r therwise rganized int study-level data sets, t be used in interpreting the utcme f a clinical study. Study-level data present clinical trial data in an bjective manner, withut subjective analysis r interpretatin, usually in tabular, graphic, r statistical frm shwing, fr example, averaged, stratified, r patterned presentatins f study data gathered. Examples wuld include a table that presents crss-patient data n baseline patient characteristics (demgraphic and disease-related), patient dispsitin (i.e., numbers/percentages f patients wh cmpleted r discntinued the trial), endpints (primary, secndary, and ther), study drug expsure, adverse events, vital signs, and labratry and ther safety measures prvided fr the verall study ppulatin, and by subgrups. This is typically available thrugh the final versin f the study reprt. Clinical study design infrmatin and prtcls direct investigatrs hw t run a particular study. Prtcls give instructins t the investigatrs n, fr example, what drug t give and when, what study measurements t take and when and hw t recrd them, and hw t treat and recrd adverse events. 4. Questin: What is the ratinale fr prviding the synpsis f Clinical Study Reprts (CSR)? Why nt prvide the full CSR as prvided t the regulatr(s)? Answer: Given the vlume f data cntained in regulatry submissins ften cntaining millins f pages EMD Sern cmmits t publishing a synpsis after marketing apprval in the US, EU, r member states. The synpsis will prvide patients and their physicians with enhanced infrmatin abut the results f clinical trials and the evidence used t apprve a new medicine. The synpsis is a part f the CSR and is reviewed by the FDA and EMA as part f their apprval. In rder t accelerate research and advance scientific understanding, EMD Sern will als evaluate requests fr full CSRs, including patient-level and study-level data, and share them under the cmmitment utlined in Infrmatin Shared with Researchers. 2 Institute f Medicine, Sharing Clinical Research Data: A Wrkshp Summary 10 (2013). EMD Sern is an affiliate f Merck KGaA Darmstadt, Germany.

5 5. Questin: Why may it be necessary t limit the availability f patient-level data fr clinical trials cnducted invlving patients whse data are likely t be re-identified? Answer: Prtecting the privacy f patients wh participate in clinical trials is a critical cnsideratin fr EMD Sern s. It may be pssible even fr annymized patientlevel data t be re-identified using mdern data mining techniques3. Fr this reasn, EMD Sern generally withhlds patient level infrmatin frm disclsure when there is a reasnable pssibility that patient privacy culd be jepardized. The risk f reidentificatin is significantly higher when the number f patients is small, such as is typically the case fr trials invlving patients with rare diseases, which may include as few as 25 r fewer patients. 6. Questin: Hw will EMD Sern determine wh can receive patient level data r ther prprietary infrmatin? Answer: Research requests must be submitted in writing t the EMD Sern prtal. Fllwing receipt, researchers requests will be evaluated initially by an internal cmmittee at EMD Sern, which may decide t apprve the request. If the EMD Sern cmmittee denies the request, the request will be escalated t the EMD Sern Scientific Review Bard fr a secnd review (de nv). The Bard shall include scientists and/r healthcare prfessinals wh are nt emplyees f EMD Sern. The Bard shall be respnsible fr reviewing the request t determine whether the request meets the criteria fr researcher qualificatins and legitimacy f the research purpse, ntwithstanding the denial by the EMD Sern cmmittee. 2 Institute f Medicine, Sharing Clinical Research Data: A Wrkshp Summary 10 (2013). EMD Sern is an affiliate f Merck KGaA Darmstadt, Germany.