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Clinical Research

Early Access Programs​

These programs go under various names, including early access, special access, or compassionate use, compassionate access, named-patient access, temporary authorization for use, cohort access, and pre-approval access, etc. They are often considered as too challenging or too risky to implement but if strategically planned, they are efficient and cost-effective options. ​

Patient benefits are the primary goals but they can be valuable for other stakeholders. ​

For what purpose?

  • Patient Benefit  ​
  • Create patient awareness of the product​
  • Bridging the gap​
  • No satisfying alternative ​
  • Market access strategy ​
  • Revenue generation  

For whom?​

Our experience:​

​

What we offer:​

  • Assistance with named-patient and cohort Early Access Programs:​
  1. Strategic advice including program feasibility and country selection​
  2. Submission to regulatory authorities​
  3. Operationalize Early Access including safety reporting​
  4. Early funding opportunities, pricing strategy​
  • Dedicated highly experienced in-house experts’ support​
  1. Protocols authoring with a transversal approach including patient engagement and market access​
  2. Early dialogue with HTA body ​
  3. Assistance with operational activities

 

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F.A.Q.

Under what circumstances is Early Access Program typically considered for patients?

The Early Access Program (EAP) is typically considered for patients under specific circumstances where there is an unmet medical need and there is therapeutic available or deemed satisfactory. Patients facing life-threatening conditions for which there are no approved treatments in their countries may be eligible for early access to potentially benefit from promising therapies.

The patient’s medical condition must align with the targeted use of the investigational product, and the potential benefits should outweigh the risks. Participation in an EAP requires informed consent from the patient or their legal representative, ensuring they are aware of the experimental nature of the treatment and the associated uncertainties.

EAPs offer ethical, compliant, and controlled mechanisms of access to investigational drugs outside of clinical trials and before the commercial launch of the drug, to patients with life-threatening diseases. Nevertheless, it’s important to note that the decision to implement an EAP is multifaceted and involves careful consideration by healthcare professionals, regulatory authorities, and the pharmaceutical or biotech company developing the intervention. Striking a balance between providing access to potential life-saving treatments and safeguarding patient safety is paramount in the evaluation process.

The implementation of Early Access Programs (EAPs) is regulated to ensure patient safety, ethical considerations, and the collection of relevant data. The specific regulations governing EAPs vary by country, and competent authorities play a crucial role in overseeing and approving these programs. Companies seeking to initiate an EAP typically need to submit data to demonstrate the safety and potential efficacy of the investigational product. There are no consolidated regulations or guidelines regarding EAP and each country often specify the content and format of documents to be submitted.

Some regulatory authorities require transparency and information on EAP are publicly available on their websites. This may include providing details on the investigational product, the number of patients involved, and key outcomes.
Regulatory landscape for Early Access is rapidly evolving, and specific requirements may differ between countries. Companies and healthcare professionals involved in EAPs must adhere to the regulations set forth by the relevant regulatory authorities in the jurisdiction where the program is being conducted.

The Early Access Program (EAP) protocol and a Clinical Trial protocol differ primarily in their objectives, study design, and the stage of drug development. The primary objective of an EAP is to provide patients with serious or life-threatening conditions early access to investigational drugs or treatments that have shown promise in earlier stages of development. The focus is on addressing an unmet medical need and providing potential therapeutic options. Clinical trials are research studies designed to assess the safety and efficacy of a drug or treatment in a controlled and systematic manner. The goal is to gather scientific evidence to support regulatory approval for broader use.

While both EAPs and Clinical Trials involve the testing of investigational treatments, their objectives, patient populations, study designs, and regulatory requirements differ significantly.

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