Orphan Drugs

EAP

Early Access Program or Expanded Access Program (EAP) / Compassionate Use / Named Patient Sales (NPS)

 

Early Access Programs / Expanded Access / Compassionate Use / Named Patients Sales Programs are country-specific regulatory tools that allow patients with unmet medical needs to have access to drug candidates in clinical development before their official launches, providing that they fulfill specific criteria.

 

NPS should not be confused with Compassionate Use, where a drug is supplied by the company for patients as a follow-up after a clinical trial or for pure humanitarian reasons. In this case, treatment is free of charge for the patient until a local authorization is obtained.

 

Recent success stories have raised the profiles of EAP, yet many companies remain unaware of the significant advantages and possibilities offered by European EAP programs.

EAP

Early Access Program or Expanded Access Program (EAP) / Compassionate Use / Named Patient Sales (NPS)

 

Early Access Programs / Expanded Access / Compassionate Use / Named Patients Sales Programs are country-specific regulatory tools that allow patients with unmet medical needs to have access to drug candidates in clinical development before their official launches, providing that they fulfill specific criteria.

 

NPS should not be confused with Compassionate Use, where a drug is supplied by the company for patients as a follow-up after a clinical trial or for pure humanitarian reasons. In this case, treatment is free of charge for the patient until a local authorization is obtained.

 

Recent success stories have raised the profiles of EAP, yet many companies remain unaware of the significant advantages and possibilities offered by European EAP programs.

Orphan Drugs

In addition, EAPs are wrongly assumed to be too risky, too complicated or too costly by many companies to consider, as:

 

♦ Pharmacovigilance teams sometimes fear potential safety issues that could be reported during an EAP, especially if the drug is used other than indicated (whereby there is no protocol).

 

♦ From a regulatory point of view, potential adverse safety results could be considered to endanger the marketing approval of the drug. In addition, regulatory teams in companies sometimes might have minimal knowledge and/or experience in EAPs and the country-specific nature of the programs.

 

♦ EAPs are also assumed to require a significant administrative workload in terms of paperwork, etc. Marketing and commercial teams often do not see how they could benefit from an EAP due to the perception that this is purely a regulatory tool. In addition, the fact that promotion of EAPs is prohibited makes these teams reluctant to go through this path.

In addition, EAPs are wrongly assumed to be too risky, too complicated or too costly by many companies to consider, as:

 

♦ Pharmacovigilance teams sometimes fear potential safety issues that could be reported during an EAP, especially if the drug is used other than indicated (whereby there is no protocol).

 

♦ From a regulatory point of view, potential adverse safety results could be considered to endanger the marketing approval of the drug. In addition, regulatory teams in companies sometimes might have minimal knowledge and/or experience in EAPs and the country-specific nature of the programs.

 

♦ EAPs are also assumed to require a significant administrative workload in terms of paperwork, etc. Marketing and commercial teams often do not see how they could benefit from an EAP due to the perception that this is purely a regulatory tool. In addition, the fact that promotion of EAPs is prohibited makes these teams reluctant to go through this path.

Orphan Drugs

♦ Unlike standard clinical trials, the number of patients and sites involved in EAPs are unpredictable (particularly in named-patient EAPs), which makes assessment of adequate drug production and supply difficult.

 

♦ Companies may not have a real presence in Europe and may not have developed any distribution channels yet. EAPs must be managed on a country-by-country basis, which might be challenging with limited knowledge of the European markets.

 

In this context, is an EAP worth considering? The answer is definitely YES.

 

CarthaGenetics® will provide all the stakeholders within your company with clear and detailed answers to these legitimate concerns.

 

For more information, please contact us at : eap@carthagenetics.com

♦ Unlike standard clinical trials, the number of patients and sites involved in EAPs are unpredictable (particularly in named-patient EAPs), which makes assessment of adequate drug production and supply difficult.

 

♦ Companies may not have a real presence in Europe and may not have developed any distribution channels yet. EAPs must be managed on a country-by-country basis, which might be challenging with limited knowledge of the European markets.

 

In this context, is an EAP worth considering? The answer is definitely YES.

 

CarthaGenetics® will provide all the stakeholders within your company with clear and detailed answers to these legitimate concerns.

 

For more information, please contact us at: eap@carthagenetics.com

Carthagenetics, your Orphan Drug Companion!

Carthagenetics, your Orphan Drug Companion !