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Pv2011_templatePublished in May 2010
NAMED PATIENT Programs
Named Patient Programs
Provide Pre-Launch Access to Drugs
The dire outlook facing AML patients motivated a company
to provide its drug in advance of the commercial launch.
Named patient programs (NPPs) enable physi- cians and patients in Europe to access medications JACK V. TALLEY
PRESIDENT AND CEO, EPICEPT
Medicines Agency (EMEA), but not yet launchedcommercially in European Union (EU) member EpiCept is a specialty pharmaceutical company
countries. Regulations also allow patients to access that focuses on fulfilling unmet medical needs drugs that are approved outside of the EU, but not in cancer treatment and pain management. yet in their home countries. In both scenarios,requests for access are made by physicians on behalf product and a deep and balanced pipeline of Ceplene is available on a named patient basis in three major clinical product candidates. For Europe and in dozens of other key markets, including more information, visit epicept.com or e-mail Latin America, Asia/Pacific, Australia, Israel, and Canada.
Similar programs that enable pre-approval or pre- launch access exist in many countries around theworld; respective governments have set well-definedrules for access criteria, collection of safety data, and The grim prospects for long-term survival in acute
control of drug distribution. In the United States, such myeloid leukemia (AML) strongly motivated EpiCept
government-sanctioned initiatives are referred to as to provide patients a route of access in advance of
expanded access programs and may be implement- the commercial introduction of its drug, Ceplene, the
ed for individuals, intermediate-sized groups (10 to first approved immunotherapy for remission mainte-
100 patients), and large groups of patients. nance and prevention of relapse in adult AML
patients in first remission.
THE ROUTE TO ACCESS
At the heart of the decision to establish a pre- Time Frame for Planning a Named Patient Program
launch or pre-approval access program is the patientwith an unmet medical need. EpiCept sought to offer a route to access that was legal and ethical. Fuelinginterest in providing access were numerous requests being received by the company from physicians in Europe and other parts of the world.
The successful development and implementa- tion of NPPs require the involvement and coordina- tion of many disciplines within the sponsoring com-pany, including representatives from medical affairs,pharmaceutical development, regulatory, supply chain, business development, and finance. The cross- functional team is responsible for ensuring that theclinical criteria for patient participation are estab-lished, physician educational materials are defined,the supply of drug is adequate to support the pro-gram, and enrollment in any ongoing clinical trials 6 MONTHS 12 MONTHS
The NPP has allowed EpiCept to make Ceplene Note: The optimal time frame for implementing the program is about 12 months available to patients in the following situations: before the drug is expected to receive approval.
• In the EU following centralized approval but prior to commercial launch in member countries.
NAMED PATIENT Programs
Named Patient Program Requirements in Select EU Member States
• In countries where applications for approval were As with all NPPs, the sponsoring company has a cal need who do not meet a clinical trial’s inclusion responsibility to report serious adverse reactions to • In countries where approval won’t be sought.
• Bridging the gap between the end of Phase III and While the drug approval process in Europe is Working with Idis, EpiCept established a full phar- centralized via the EMEA, the drug reimbursement macovigilance system to capture and report such • Bridging the gap between approval and commer- system is decentralized; decisions regarding reim- information. The company also established a series of bursement take place within each respective EU standard operating procedures to guide the pro- • Throughout a staggered global launch while country. This process often results in a delay in com- gram — these include procedures related to enroll- approval and reimbursement is being sought mercial launch as some countries can take between ment of patients, release of the drug to the hospital, 12 and 18 months to establish reimbursement fol- as well as regular reporting mechanisms on usage.
For patients with life-threatening illnesses such as lowing EMEA approval of a drug. During this lag peri- Programs in the various countries will terminate AML, license approval or commercial launch of an od, AML patients in the EU can access Ceplene with the commercial launch of the drug. Those innovative new drug in their home country may patients accessing Ceplene on a named patient come too late. In some cases — those involving the While pre-launch access is permitted by EU legis- basis will simply transition to the commercial drug EU registration and approval process, for example — lation, NPPs are governed by the individual member once available and continue treatment in an unin- a drug may not clear reimbursement hurdles in an states; each of the 30 member states of the European terrupted manner. In countries where approval is not individual country until well after centralized Economic Area (EEA) has its own regulations regard- being sought, patients will continue to access Cep- approval is granted. In cases where a formal launch may not even be planned, a patient may not have To navigate the regulatory and logistical path- any other opportunity to receive the drug other than ways in more than 100 countries and remain in com- MEETING UNMET NEEDS
pliance with all authorities, EpiCept chose to partner These programs also provide access to patients with Idis, a specialist in NPP development and man- Pre-launch access to drugs can provide profound who would otherwise not be able to participate in agement, rather than rely on internal resources. benefits for patients with unmet medical needs company-sponsored clinical trials and allow spon- Once the decision was made to offer Ceplene on when all other therapeutic options have failed or no soring companies to engage with physicians and a named patient basis, establishment of the program other options are available. NPPs facilitate patient provide early, hands-on access to life-saving required about 10 weeks. Initiation of the NPP in the access in markets that a company may not be pur- medicines. In all of these scenarios, named patient EU was timed to coincide with the date the drug was suing in any other way. For example, it can be imprac- programs allow companies to provide access to ready from a manufacturing standpoint and had tical for a small company to credibly pursue approval innovative drugs in an ethical, legal manner while complete and final EMEA approval and sign off. in more than 100 countries, such as those that the maintaining strict control over where the drugs go Along with each request for Ceplene the pre- scribing physician provides the patient’s age and Using NPPs, access can be expanded to markets remission status. EpiCept provides complete pre- that would otherwise not be served, where gaps scribing information as contained within the Sum- exist between approval and launch or in situations article. E-mail us at [email protected]
mary of Product Characteristics (SmPC) to those where a patient cannot enter a clinical trial.
physicians requesting the drug. A copy of a peer- Named patient programs can provide access at a To download a FREE Podcast from Idis on Named Patient reviewed article on Ceplene’s Phase III clinical trial is number of stages throughout a product’s life cycle: Programs, visit www.pharmavoice.com/podcasts • During Phase III, for patients with an unmet medi- PharmaVOICE
Golden Boy Pies, Inc. Pie, GBP, Fruit, Apple Nutrition Amount/serving Amount/serving Total Fat 23g Total Carb. 44g Calories 380 Cholest. 5mg Protein 2g Sodium 320mg *Percent Daily Values (DV) arebased on a 2,000 calorie diet. • Vitamin C 150 % • Calcium 0 % • Iron 8% INGREDIENTS: Apples (Granny Smith Apples, Ascorbic Acid, Citric Acid, Salt), Water,