The Critical role of Real-World Data in Orphan Drug Development and the Role of Early Access Programs

We asked Heather Manna, VP, Global Regulatory Affairs, Inceptua Early Access, to answer 8 key questions about Real World Data, the important role Early Access is playing and the multiple-stakeholder benefits.

  • The possibilities with Real World Data (RWD) are receiving more and more attention in relation to EAPs. What role is RWD playing in the EAP setting in 2022 and beyond?

RWD has been a topic of discussion for many years on the conference circuit, within the pharmaceutical and biotechnology industry and eventually health authorities around the world. 

The conversations began with asking about the possibility of RWD collection in an EAP, evolving to the potential use of RWD from an EAP to where we are today which includes identification of multiple instances where RWD from an EAP was used to satisfy both regulatory and commercial objectives. 

Health authorities (HA), such as the FDA (US), EMA (UK) and ANSM (Fr) have openly expressed support for collection of data from EAPs and even revised country-specific early access regulations requiring collection of some data.  Most recently the EMA endorsed a joint statement calling for international collaboration to enable the generation and use of real-world evidence into regulatory decision making.

 

  • When it comes to innovation of novel therapies – especially in the rare diseases space – what potential do you see in Real World Data and EAPs?

Typically, patient populations in the rare disease space are limited.  It is often challenging for the small number of patients with a specific rare disease to meet specific clinical trial (CT) inclusion/exclusion criteria for recruitment and participation in a CT.

Considering the small number of patients afflicted with a rare disease and therefore a limited amount of CT data that can be collected as a result, the EAP with RWD (treatment-related) collection can help address the treatment needs of these patients while providing valuable data for potential use in regulatory decision making.  Health authorities will have to adjust to assessing and measuring data from these types of EAPs, especially in rare disease, whereby the data is often subjective.

The EAP does not replace the CT, but collection of RWD in the less confined (real-world) EA patient population has value and could potentially be used to support regulatory decision making if a HA agrees. 

 

  • How should Real World Data be used in rare disease Early Access Programs?

The primary purpose of an EAP is for treatment of eligible patients who have unmet medical needs.  As stated in the answer to question 2 above, the EA does not replace the CT, however, real-world data  from the EA patient population does indeed have value and could inform the development of a novel therapy and could potentially be used to support regulatory decision making by pharmaceutical and biotechnology companies and health authorities.

 

  • What differs from conventional clinical research studies?

Conventional CTs are designed to answer a specific question(s), research or study a drug/therapy in a specific indication in a confined patient population.  A conventional CT may inform the development of a drug/therapy or provide pivotal or supportive data for use in a regulatory filing used in health authority decision making.  The patient populations in CTs are often much narrower than in an EAP. The EAP allows collection of data beyond that narrow range, perhaps more reflective of the clinical/commercial setting.

 

  • What are the key things to consider before starting up and implementing an EAP, with an aim to also take advantage of the potential in Real World Data?

Inceptua has and continues to design, implement, and execute EAPs which vary from the basic program (i.e., ‘all comers’, no data collection) to more restricted programs, limiting patient access to specific subsets or the collection of outcomes data in addition to the basic demographic data often collected.  To ensure that data collection within an EAP is properly designed, it is important that pharmaceutical and biotechnology companies address some fundamental questions, for example: 

  • What are the intended objectives of the data collection?
  • Is there a question(s) to be answered through treatment use?
  • What data points would be valuable to collect?
  • How will data be used?
  • How many patients will be required in order to meet the intended objectives?

 

  • What regulatory requirements should you be aware of, relating to data collection in the EAP setting?

Regulatory requirements are stipulated in EA regulations (i.e., safety, outcomes or other).  Typically, basic demographic data is collected in an EAP, the degree of which may be limited by country-specific privacy laws (i.e., any data that may lead to patient identification).  Health authorities, such as the EMA (UK) and ANSM (Fr) have revised early access regulations requiring collection of some data in an EAP.  Collection and reporting of safety data is always mandatory and in some instances regulation (i.e., FDA) requires treatment outcomes data.  Global EA regulations continue to evolve and it should be anticipated that EAP RWD collection and use will evolve as well.

 

  • Which stakeholder groups can benefit and how – what are the benefits?

All stakeholders benefit in some way:

  • Pharmaceutical and Biotechnology Companies

Valuable treatment-related data collected from EAPs could potentially inform drug development, identification of safety signals, new indications or formulations, potential use in regulatory/commercial decision making or potentially used in regulatory filings.

  • Health Authorities

Health authorities may agree to the use of RWD from an EAP to provide supportive data (especially in rare disease) to aid in the review of regulatory filings. (i.e., approvals, label expansion, etc.)

  • Health Care Practitioners

In addition to supporting patient early access to novel drugs/therapies in development, the HCP plays an important role in learning more and potentially publishing articles about the use of drug/therapy in development within a non-confined real world patient population.

  • Scientific and Medical Communities (i.e., Journals, Published Papers, etc.)

In addition to direct HCP to HCP and patient to patient communication, published content (i.e., journals, papers, posters, social media posts) often provides summarization of treatment use, data, and information about an EAP and/or EAP RWD.

  • Patients

With immediate access to information, patients are more educated than ever before. Patients participating in an EAP play an active part in the drug development process by agreeing to the collection and use of their treatment-related data.  Eligible patients can review published EAP data to assist in making the decision as to whether to participate in an EAP for treatment with an investigational drug which has not yet been approved by a health authority or approved for the treatment of their disease or condition in their country or any country. 

  • Public

Caregivers, authorized representatives, family members and anyone in the general public should be able to access registries and published data from EAPs, as much as is required for clinical trials.

 

  • The future for RWD collection in EAPs

The primary intent and focus of the EAP continues to be for the treatment of patients who have unmet medical needs.  Over the last decade the discussion regarding the collection and potential use of RWD from EAPs has grown within the pharmaceutical and biotechnology industry. In my opinion, the collection and use of treatment-related and treatment outcomes data generated from EAPs will become a standard component of an EAP, and will likely become normalized, and even perhaps formalized in EAP regulations. Recent steps by the MHRA (UK) and ANSM (Fr) to encourage the collection of treatment-related data (RWD) is likely to be followed by other health authorities.

There is growing concern amongst some health authorities about national systems paying for high-value products in the EAP setting that have not yet been approved, and therefore not been confirmed to have clinical value.  Expecting companies to collect data to support and prove a product’s value is an inevitable step.  I suspect that the recent approval of Vijoice® (alpelisib) for patients with PIK3CA-related overgrowth spectrum (PROS) based off data from a compassionate use/early access program in France will make more companies consider the potential value of RWD in the EAP setting.

As health Authorities express their views and provide guidance about the value of the collection and use of RWD from an EAP, the pharmaceutical and biotechnology industry will likely feel reassured about collecting such data. The key question is, how do we strike that balance of providing easy access to treatment, but ensuring data collected is robust enough to be used for meaningful decision-making?

 

References

FDA approves Novartis Vijoice® (alpelisib) as first and only treatment for select patients with PIK3CA-Related Overgrowth Spectrum (PROS). April 6, 2022, Novartis website.

Heather Manna

Heather is a leading EAP Subject Matter Expert in the development, design and execution of expanded access programs across multiple therapy areas including rare diseases and oncology.

Connect with Heather to learn more and stay in touch

Heather Manna is on LinkedIn

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