Market Access                                          Newsletter               also applies to regulatory decision making...
Managed Market Entry for Drugs inItaly                                                                      Expert Viewpoi...
Performance linked reimbursement agreement in Italy on cancer drugsMolecule       Brand        Indication      Date       ...
Are you looking for an insightful          course in Market Access?                                                       ...
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Market Access Newsletter Emaud June2012


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Market Access Newsletter Emaud June2012

  1. 1. Market Access Newsletter also applies to regulatory decision making. Regulators have Editorial instituted in Europe : conditional approval or risk management plans which could also be seen as a kind of regulatory conditional approval process; the qualification of a lack of by Prof. Mondher Toumi efficacy as adverse events, which altogether contribute to shift EMAUD Chairman the marketing authorization approval from a punctual decision making process to a lengthy procedure. This shift from decision point to decision window becomes an increasing a reality for pharmaceutical companies. An understanding of the Dear Colleagues & Friends, needs to close or minimize the uncertainty gap before approval and market access negotiations has become a critical At the time of approval for new pharmaceuticals/ issue for the pharmaceutical industry.technologies, payers usually have to deal with difficult Through these changes, we observe that the tacit sharedecisions on whether to cover or not these new technologies. of risk between manufacturers and payers has come to anDe facto, positive and negative decisions are difficult to make end. Indeed, manufacturers used to bear the whole pre-and a “wait and see” attitude is often taken. The lack of approval risks while payers used to bear post-approval risks.appropriate information to inform payers’ decision making has Nowadays, the trend is that manufacturers bear all the risks atoften been the main reason for delayed or negative decisions. pre- and post- approval, which leads to an increasing requestOver the years, we could see in many countries the for payers to acknowledge these factors and increase drugdevelopment of conditional coverage schemes usually called prices.Coverage with Evidence Development (CED). CED allows the Addressing these risks requires manufacturers toadoption of a careful attitude that grants patients early access develop and optimize the use of appropriate modelingwith all the associated benefits of promising new technology. techniques to support internal decision making. Such expertiseThe CED implies that some clear uncertainty has been already exists within the pharmaceutical industry, but is oftenidentified and a clear study design is able to address the too restricted to target external payers, forgetting internalaforementioned uncertainty at a reasonable cost and stakeholders.timeframe. Moreover, the decision under CED is considered as Over the next ten years, companies’ internal decisionpotentially reversible without major damages or costs for making capabilities will have to evolve and be able to managesociety. During the CED period, the intervention is covered and product’s uncertainty to feed go/no-go decisions and tofunded by the insurance. However, if it appears to payers that narrow uncertainty at the time of launch.the resulting evidence does not support the intervention’s It is my pleasure to welcome Prof. Claudio Jommi fromvalue; the expenses should be reimbursed by the Bocconi University in Milan, who will be discussing about whatmanufacturer. Some payers then have developed escrow is going on in Italy after agreements like risk-sharing, paymentagreements to complement the CED. by performance and cost-sharing have taken place, and it may Sweden has been a leader in implementing CED to be time to draw an outcome and question whether CED willsecure quick access to innovation and appropriate data come on stage soon. Alongside Prof. Jommi, I am also gratefulcollection to address the new intervention’s uncertainty. We for having the contribution of Mr. Love Linnér, from TLV incould see such agreements growing over the next few years, Sweden, who highlights how Sweden has signed up with CED.and are already being widely adopted in countries like theNetherlands, and more recently France. As a matter of consequence, the access of medicinesthat used to happen at a distinct point in time, has become along term process that now takes place over a window periodof time. This process is not specific to market access but it ©EMAUD. Newsletter #5, June 2012
  2. 2. Managed Market Entry for Drugs inItaly Expert Viewpoint Prof. Claudio Jommi References Associate Professor of Management, Fattore G, Jommi C, The last decade of Italian pharmaceutical policy: Università del Piemonte Orientale and instability or consolidation? Pharmacoeconomics. 2008;26(1):5-15. Director of Pharmaceutical Observatory, (last CERGAS Bocconi – Italy access, June, 22nd, 2012). Russo P, Mennini FS, Siviero PD, Rasi G. Time to market and patient access to new oncology products in Italy: a multistep pathway from European context to regional health care providers, Ann Oncol. 2010 The most important drivers of pharmaceutical policy Oct;21(10):2081-7. Garattini L, Casadei G, Risk sharing agreements: what lessons from Italy?in Italy are the drug budget, a tough price negotiation, Int J Technol Assess Health Care. 2011 Apr;27(2):169-72.managed market entry contracts, and regionalization. (last access: June, 22nd, 2012).A national drug budget became law in 2001 and changedmany times. At present, retail and hospital drugs are the industry takes usually the form of credit note forrespectively subject to a 13.3% and a 2.4% budget hospitals, that can be converted into a cash transfer, ifrespectively (of the total health fund). If the retail and agreed with the relevant company. 15 PLRAs have signed forhospital budget are overrun, the industry/distributors and 14 different products so far (Table below). Many otherregions are required to cover the deficit respectively. The cancer drugs are subject to price-discounts (generally 50% ofrules are changing: (i) hospital budget should increase, but prices) for first therapeutic cycles.the industry will be asked to cover 30% of the deficit, and (ii) PLRAs have had the great advantage to guaranteeretail budget should be cut. market access at a nominal price consistent with priceReimbursement and prices are simultaneously negotiated by corridor desired by the industry and to link coverage withthe National Drugs Agency (Aifa) and the industry, response to treatment. The literature has also shown thatconsidering therapeutic added value (and comparator drugs subject to PLRAs have a faster market penetration thanprices), prices in other countries, the impact on the drug other cancer drugs. In addition, reimbursement is appliedbudget, and, in principle, cost-efficacy for the most immediately after treatment failure, whereas coverage withinnovative drugs. The negotiation takes a long time and evidence development through observational studies wouldcompanies rarely consider Italy first for market launch. require that studies are concluded.Hence, Italy shows an average of 306 days in the delay The main critical issue of PLRAs is that they rely on thebetween marketing authorisation and reimbursement. collaboration of professionals into hospitals. Actually, despite Finally, national approval does not mean immediate hospitals are obliged to enrol patients into registry to getregional and local access. Regions have been putting many public coverage for drugs, some regions are pushing theirefforts to contain costs because of their responsibility for hospitals to implement PLRAs. This indirectly demonstrateshealth care budget. Regional policies include regional binding that the system is not deep-rooted in the whole country.formularies, public procurement based on the lowest offer Finally the evidence on the impact of PLRAs is very limited soprice, budget constraints at the hospital level, prescription far. In 2007, the Aifa had published a National Report ontargets for GPs and specialists. Cancer Drugs subject to the National Registry including Despite of this tough environment and regardless the information on non responders, but the Report has neveradded value, a product for severe diseases is likely to get the been updated afterwards.reimbursement status, thanks to managed market entry Should Italy rely on PLRAs also in the future, it wouldcontracts. Aifa has initially mostly relied on financial be desirable to have (i) their application when the responsecontracts (price-volume agreements, general price-discounts, rate to drugs is easy, not costly to detect, and notprice-discounts on first therapeutic cycles and yearly controversial (ii) a re-evaluation of the agreement based onspending cap per patient). More recently, performance- actual response rate, (iii) and more information on impact oflinked reimbursement agreements (PLRAs) have been signed PLRAs. In addition, coverage with evidence developmentfor expensive cancer medicines. These contracts provide, for could integrate PLRAs when an uncertainty on the risk-non-responder patients, an obligation for the industry to benefit profile and costs is very high and can be measuredreimburse 50% or 100% of the price to the hospital. PLRAs only in the long-term.may be suggested by the companies or Aifa (supported bythe Oncology Sub-Committee) during the negotiationprocess. The contracts are confidential and include the nameof the drug, the indication, response criteria, and paybackprovisions. PLRAs have relied, at least in principle, on theNational Registry for cancer drugs. A national IT Company(Cineca) is in charge with managing the Registry. Cliniciansand hospital pharmacists populate the Registry (patientsenrolment, drug request, patient assessment andreimbursement request for non-responders). The payback by
  3. 3. Performance linked reimbursement agreement in Italy on cancer drugsMolecule Brand Indication Date Payback Timing Non responders after 3 Expert ViewpointEverolimus Afinitor mRCC Jun. 2010 100% treatment months mRCC (EGRF+, Non responders after 2Cetuximab Erbitux KRAS wild-type) Jun. 2009 50% treatment months three or four years, describing the real world outcomeCetuximab Erbitux Advanced squamous HNC Jun. 2010 100% Non responders after 2 treatment months related to one or more of the assumptions made in theTrastuzumab Herceptin EGJ AC Dec. 2010 50% Non responders after 2 original cost-effectiveness analysis. cycles NSCLC EGFR Non responders after 3 At the time of a conditional reimbursement decisionGefitinib Iressa May 2010 100% mut treatment months there is no clearly stated accepted level of the variable that is Non responders after 2Vinflunine Javlor UCC Dec. 2010 100% cycles to be studied by the company, but the findings from theMozobil Plenixafor Stem cell Dec. 2011 100% Refund Treatment (if study are to be compared to the assumptions made in the mobilization CD34+ > 2 x106/Kg) Non responders after 2 application. If the real world evidence deviates from theSorafenib Nexavar HCC Jun. 2008 100% treatment months original assumptions, TLV may choose to re-evaluate the Non responders after 1Dasatinib Sprycel CML / ALL May 2007 100% treatment month reimbursement status of the product in question. CML Non responders after 1Nilotinib Tasigna Philadelphia Aug. 2008 100% chromosome+ treatment month During the past year, TLV has evaluated follow-upTemsirolimus Torisel mRCC Oct. 2008 100% Non responders after 2 treatment months reports for a number of products with conditionalLapatinib Tyverb mBC May 2009 100% Non responders after 3 reimbursement and established a process for how the cycles (6 months) mRCC (EGRF+, Non responders after 2 outcome of the evaluation guides further decisions for thePanitumumab Vectibix Jan. 2009 50% KRAS wild-type) treatment months product: Non responders afterVotrient Pazopanib RCC May 2011 100% 24 treatment weeks Non responders after 2 • TLVs’ evaluations of the reports for Risperdal ConstaTrabectedina Yondelis ASTS Jan. 2009 100% cycles (risperidone i.m.), Lucentis (ranibizumab) and ChampixAcronyms (varenicline) resulted in no further actions by the agencyALL Acute Lymphoblastic Leukemia since the assumptions made in the original applicationsASTS Advanced Soft Tissue Sarcoma for reimbursement, and that was studied, appeared to beCML Chronic Mieloid Leukemia valid.EGFR mut Epidermal Growth Factor Receptor - activating mutationsEGJ AC Early esophago-Gastric Junction AdenoCarcinoma • Cymbalta (duloxetine) is a product among many withHCC HepatoCellular Carcinoma limited reimbursement in Sweden, i.e. only a subset ofHNC Head and Neck Carcinoma the patients included in the full indication for the productmBC metastatic Breast Cancer should have it prescribed with reimbursement. ThemRCC metastatic Renal Cell CarcinomaNSCLC Non-Small-Cell Lung Cancer follow-up report for Cymbalta indicated that prescribersUCC Urothelial Carcinoma (transitional cell carcinoma) in Sweden in some instances prescribe the product with reimbursement to patients not included in the limitation set by TLV. The report was based on data from theRecent development for CED in Swedish Prescribed Drug Register which was analyzed in aSweden joint effort by TLV and the company. The price of the product was later changed by the company to compensate for the limitations in quality of prescribing. The report for Replens (water-based gel), also a product Mr. Love Linnér, PhD with limited reimbursement, was later analyzed via a Coordinator observational studies The Dental and Pharmaceutical similar methodology and the price was also corrected Benefits Agency, Sweden based on data from the Prescribed Drug Register. • TLVs’ evaluations of the reports for Firazyr (icatibant) and Berinert (human C1 esterase inhibitor) resulted in a The Dental and Pharmaceutical Benefits Agency, TLV, recommendation to re-evaluate the pricing of productsis a central government agency whose remit is to determine used for the treatment of hereditary angioedema.whether a pharmaceutical product shall be subsidized by thestate. The reimbursement of a product may in someinstances be coupled to product specific restrictions, e.g.second line usage only, or other special conditions such asrequirements for the company to report outcomes fromfollow-up studies.Conditional reimbursement based on further evidencedevelopment (CED) has been included in a limited share ofthe reimbursement decisions made by the TLV since 2003. Acompany whose product is subject to a conditionalreimbursement is normally to report a follow up study within
  4. 4. Are you looking for an insightful course in Market Access? EMAUD News --------------Save the Date----------- The programme that follows each step of the life cycle of a drug, integrating market access theory and practice for results to be applied in the real world. Students will acquire the necessary skills to develop and implement a market access plan. Candidates, registration open until 30 September! Market Access Day IV 11 December 2012, CIU ParisStudents Corner Contributions and debates will take place around the theme: “From point decision to window decision” In more and more countries, pricing and reimbursement decisions are no longer taken at a fixed time point, but over a period of time during which pharmaceutical companies are expected to provide additional information to reduce any uncertainty around the value of a new product. Recently, regulators have been dealing with three main facts: conditional approvals; the lack of efficacy now considered as an adverse event, and as such should be reported to by prescribers; risk management plans that have been instituted to track the lack of efficacy as one of the potential adverse Prof. Toumi & the Students of Class 2011-2012 events. The first two being recent in the EU compared to the May 2012, Module 5 USA. Therefore, there has been a move from a decisionIn 2011-2012, 60 students attended the session: point for regulatory approval, pricing and market access • 36 students from the industry, decisions, towards a timeframe that has turned into a • 13 students from public institutions, decision window. This allows regulators and payers to • 11 students from consulting and other, minimize the risk associated with the introduction of a new •18 countries were represented, mostly European product. This is also contributing to the increased dialogue countries, and also Brazil, Canada, China, Russia and the between regulators and payers. USA. In 2012, the Annual Market Access Day will review the impact of the change of paradigm on patient access, payers new practice, and will review the evolution ofStudents: Connect on Linkedin! companies to fit a new changing environment.The EMAUD Alumni group is intended as a debating andnetworking platform for the students and contributors of our Join us for living and inspiring discussions!educational programme. Be aware of latest news, articles,regulations etc. via our discussions and connections. EMAUD Contact Prof. M. Toumi, Chair Mrs. O. Barthez, Programme Coordinator & Communications Manager Mrs. F. Guetatlia, Administrative support