Why It Is Important to Develop a Good Expanded Access Policy

As access to investigational drugs has captivated the attention of a wide range of stakeholders, pharmaceutical companies must have a well-structured expanded access policy in place and be prepared to handle potentially sensitive and urgent requests.

The 21st Century Cures Act in the United States, which was enacted in December 2016, required drug companies developing investigational drugs to make their policy for evaluating and responding to requests for expanded access for individual patients publicly available. Despite increased adoption, concerns remain that pharmaceutical companies make it unnecessarily hard for patients and physicians to navigate companies’ expanded access/compassionate use request process.

All companies with products in phase 2 and phase 3 of development must have an expanded access policy on their website. This implies that companies must decide whether they will or will not provide expanded access and must then provide a written explanation of their policy. This aims to make it easier for patients and physicians to find information about treatment options and to improve access when possible.

Some drug companies may still be unaware of the expanded access transparency requirement under the 21st Century Cures Act, and thus fail to understand the negative PR and lost stakeholder engagement opportunities that are tied to a poorly developed and implemented expanded access policy. Recent stories within the ALS community about patients trying to obtain access have underscored the need for companies to determine whether they comply with the expanded access policy disclosure requirement and whether they can implement a response that explains the intent of their policy.

Expanded Access policy elements

Although company policies differ, seven elements may be included in an expanded access policy.

1) Purpose and philosophy behind the policy

• A statement that often references a commitment to bringing new, innovative therapies to patients by conducting rigorous clinical trials and obtaining marketing approval by the FDA and other regulatory authorities while explaining the company’s position on expanded access.

2) Approach to requests for access to investigational drugs

• Participation in clinical trials is the first and most preferable route.
• If participation in clinical trials is not an option, physicians may consider other options, including expanded access programs, managed by the company, or single-patient expanded access.
• Requests are considered fairly and equitably, on a case-by-case basis.

3) General criteria used to evaluate such requests for individual patients

• The patient has a serious or life-threatening condition with no satisfactory alternative.
• Assessment that benefits for the patient outweigh the risks
• Assessment that the company has an adequate supply
• A determination that expanded access will not interfere with the company’s ability to complete clinical trials in a timely fashion or which might otherwise delay marketing approval and ultimately availability to all patients.

4) Statement that a medically qualified physician should be the first point of contact with the company either by phone or email

• The company may request additional information, including patient history, to fully evaluate the request.
• If the physician will be the sponsor for the IND, he or she is required to obtain necessary ethical and regulatory clearances, including informed consent, as well as provide reporting on treatment outcomes.

5) Commitment to acknowledging the request by a certain period, e.g. five working days

6) Contact information

• ​​Such information may include a general email address, individual’s name, web form, and/or telephone number.

7) Name of drug and indications

• It is also helpful to indicate the name of the investigational drugs and the corresponding conditions for which they may be made available.

Expanded access policy transparency implications and opportunities

Although section 561A of the Food, Drug, and Cosmetic Act does not specify the penalty for noncompliance, recent penalties from the FDA on companies that have failed to post clinical trial results may suggest a new attitude towards non-compliance with transparency requirements. IMCRA and WHO also recently released a joint statement on this issue, which may lead to increased attention to the transparent publication of EA policies.

These events notwithstanding, having a good EA policy may provide several benefits for companies:

• It enhances trust with patients and providers and reduces review costs for unqualified inbound patient/physician requests.
• For patients and providers, it reduces chaos and time spent searching for options.
• It facilitates efficiency in requesting potential treatment and appropriately acknowledges patient requests.
• For physicians, it may lower the barrier to actually requesting the drug.

Thus, having a compliant, ethical, and fair policy in place to manage requests for access to a company’s investigational drugs should undoubtedly yield multiple benefits. Consulting with patients and patient advocates well in advance to establish an EA policy can bring additional benefits such as trust and further engagement.

Additional expanded access awareness considerations

Companies may decide that posting their expanded access policy (according to section 3032 of the Cures Act) on their company website suffices. However, as terminology remains complex, companies may want to develop a video or brochure explaining their policies in a straightforward manner accessible to patients at various educational levels.

One avenue often used to create awareness is to list the availability of an EAP on trial registry sites such as clinicaltrials.gov, EudraCT, or the Reagan-Udall Expanded Access Navigator company directory. These government-sponsored websites create awareness of the program’s availability. In addition to these government websites, companies also have the opportunity to create awareness and easier access to their clinical trials and EAPs through the more advanced yet user-friendly platform myTomorrows.

An additional approach is creating specialized landing/web pages that may function as a centralized source to educate patients and their physicians about what an EA program entails and how to request a drug, as well as provide answers to commonly asked questions.

Conclusion

myTomorrows has helped drug companies contextualize the EAP disclosure requirement and understand the regulatory considerations around developing and implementing their EA policies. In addition, myTomorrows has a deep understanding of the various online and search-engine strategies that can create a compliant but highly effective strategy for program awareness. Understanding the journeys of both patients and physicians remains pivotal to making access an equitable process.

Crafting a good expanded access policy will help patients and physicians seeking expanded access (compassionate use) to make more informed choices about which investigational drugs are potentially offered and help them understand the criteria drug companies use to evaluate expanded access requests. Download a sample template here.

Once alignment is achieved on the company’s strategic approach to offering expanded access, companies can quickly develop their internal expanded access policies and publicly make available their compliant EAPs. This will create a collaborative mindset that can help more patients while generating potentially valuable insights.