Leveraging Experience from KOLS: Early Access Routes in Latin America

November 27, 2019

Different types of medicine

by Gabriela Honda, Carolina Quesada-Rodriguez, and Mariana Torgal

Early access programs are a great opportunity to improve KOL/physician support prior to formal reimbursement. We discuss the programs available in Brazil, Mexico, Argentina, and Colombia.

It is common for pharmaceutical companies to explore early access programs, such as ATU in France or Law 648 in Italy. Given the typical delay in regulatory and reimbursement decisions in Latin America, early access programs are a great opportunity to improve key opinion leaders’ (KOL) and payers’ product value perception in these markets as well. However, there is not a lot of transparency on which drugs are approved through early access programs and why. Here we highlight a few available programs in selected Latin America markets:

Two programs available in Brazil

In Brazil, there are two programs available for severe and life-threatening diseases when no other satisfactory therapeutic alternative is registered. The “sponsor”, typically a private or public healthcare facility, submits the request to ANVISA, the Brazilian regulatory agency, and must provide a guarantee of coverage of full treatment free of charge for the patient. This means that the patient cannot incur any out-of-pocket expenses for the treatment.

  1. The compassionate use program. Available for individual patients, this program is applicable for drugs without ANVISA registry in any phase of clinical development and with scientific evidence demonstrating efficacy
  2. The expanded access program. Available for patient groups, for drugs without ANVISA registry, and with phase III trial under development or concluded.

These programs are different from legal injunctions where the patient gets access granted in court due to their universal constitutional right to healthcare, and in which the manufacturer cannot be involved.

Special treatments imported in Mexico

In Mexico, the importation of special treatments is available for low incidence diseases with social repercussions that represent a risk to vulnerable groups. This applies for drugs without local regulatory approval. Requests can be made by any person, manufacturer, or health service provider (public or private) and the authorization is valid for 180 days. However, there is limited information available regarding the funding and other criteria that may apply.

Two programs available in Argentina

In Argentina, there are two programs very similar to the ones available in Brazil. Both are for severe and life-threatening diseases for which there is no other satisfactory therapeutic alternative approved by ANMAT, the Argentinian regulatory agency.

  1. The exceptional access program. This must be requested by the physician and is only applicable for drugs commercialized or in clinical trial in countries with regulatory similarities. The maximum treatment authorization is for 60 days, or 180 days for chronic treatments (new requests can be made thereafter)
  2. The expanded access program. This must be requested by the manufacturer, but requires the manufacturer to commit to market authorization submission within the next 12 months (if not already submitted). The maximum treatment duration is 12 months. It must be provided for free for the patient and the healthcare system (prepagas, private payer, or social security)

By law, there is no explicit statement regarding who funds the treatment for the exceptional and expanded access program.

National program in Colombia

Vitales no Disponibles is a national program for products treating life-threatening or debilitating medical conditions that are not available in the market. These are either not approved, or are approved but not commercialized. Vitales no Disponibles is applicable for individual patients or groups. Funding is granted through ADRES, the health resources administrator, following a request by any “natural or juridical person, public or private”. Authorization is valid for the duration prescribed by the physician. If additional treatment is needed, a new request must be made. There is a public list available, which makes Colombia more transparent than the other markets previously mentioned.

An opportunity to enhance reimbursement prospects

Overall, these programs, despite a lack of transparency and/or wide reach, could represent an opportunity to enhance reimbursement prospects by:

  • Increasing KOL/physician support early in these markets (which is of high importance for reimbursement decisions)
  • Enabling the collection of real world evidence locally, which is often helpful in gaining access in these markets.

Manufacturers should target a few reference centers in the market, usually led by university hospitals, to roll out these programs. This will secure physician uptake, awareness, and much needed local advocacy and support when local public funding negotiations come to play.

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