Policy For Engaging With Industry

Produced by the Pyruvate Kinase Deficiency Foundation

July 1, 2021

Article l - Purpose

The Pyruvate Kinase Deficiency Foundation is a national nonprofit organization whose mission is to enhance the quality of life for patients and their families by providing awareness, expanding education and promoting advocacy.

To accomplish this mission, the Pyruvate Kinase Deficiency Foundation has created, and seeks to enhance, a landscape that encourages investment in research into pyruvate kinase deficiency by all stakeholders. This includes actively engaging and collaborating with pharmaceutical companies.

The Pyruvate Kinase Deficiency Foundation has developed this policy to ensure the highest level of ethical conduct is followed in the Pyruvate Kinase Deficiency Foundation’s collaborations with pharmaceutical companies. The Pyruvate Kinase Deficiency Foundation’s goal in engaging pharmaceutical companies is to enable development of therapies to meet patient needs while maintaining independence and neutrality as a patient organization.

The Pyruvate Kinase Deficiency Foundation’s priority is the best needs of patients, and no collaborations with pharmaceutical companies will influence the Pyruvate Kinase Deficiency Foundation’s efforts to advocate for accessible, affordable treatments for patients or cause the Pyruvate Kinase Deficiency Foundation to provide biased information to constituents.

The principles outlined in this document are intended to guide the Pyruvate Kinase Deficiency Foundation including board members, staff, committees and all related parties in engagement with the pharmaceutical industry.

Article II - Company Engagement

The Pyruvate Kinase Deficiency Foundation seeks mutually beneficial dialogue and information exchange with pharmaceutical companies developing potential therapies for pyruvate kinase deficiency according to the following:

  1. The Pyruvate Kinase Deficiency Foundation will actively engage with companies that show interest or activity in drug discovery, preclinical research, or clinical research in pyruvate kinase deficiency.

  2. The Pyruvate Kinase Deficiency Foundation will collaborate with companies, at Pyruvate Kinase Deficiency Foundation’s discretion and in consultation with our medical and scientific advisors, which are conducting ethical, high-quality research in a responsible manner, according to industry and international regulatory standards.

  3. The Pyruvate Kinase Deficiency Foundation will seek insight into the company’s objectives, plans, and the potential drug being evaluated and will provide companies with community-wide insight and perspective as needed and appropriate.

  4. The Pyruvate Kinase Deficiency Foundation will advocate on behalf of the patient community to make new therapies accessible. Medications can only change lives if patients have access to them.

  5. The Pyruvate Kinase Deficiency Foundation will actively seek guidance and utilize the expertise of the Pyruvate Kinase Deficiency Foundation Medical Advisory Board throughout the process of working with each company.

  6. The Pyruvate Kinase Deficiency Foundation Executive Board and Medical Advisory Board members will disclose any and all relationships with companies with whom the Pyruvate Kinase Deficiency Foundation engages and will annually sign a statement agreeing to the Pyruvate Kinase Deficiency Foundation Conflict of Interest Policy.

  7. The Pyruvate Kinase Deficiency Foundation Executive Board and/or Medical Advisory Board must approve industry presentations of education, information or services that bear its name.

  8. Industry or Corporate sponsors must give the Pyruvate Kinase Deficiency Foundation complete editorial control and approval of all materials that bear its name.

Article III - Patient Engagement

The Pyruvate Kinase Deficiency Foundation encourages and enables direct dialogue between patients and pharmaceutical company representatives for the purposes of promoting disease awareness and sharing patient perspective, according to the following principles:

  1. Direct interactions between patients and pharmaceutical companies are best arranged with the involvement of the Pyruvate Kinase Deficiency Foundation because including a patient organization in these interactions can:

a. ensure fairness and transparency within the patient community;

b. ensure that the patient community is well and adequately represented to the pharmaceutical company;

c. allow for access to experts and professional advisers who can inform the dialogue;

d. help avoid misunderstanding in the conversation;

e. ensure the protection of patient privacy in any data collection activities;

f. allow for the formation of an advisory group, when appropriate;

g. allow the Pyruvate Kinase Deficiency Foundation to better understand the needs of both the patient and the pharmaceutical company.

2. Any information learned from these interactions should be shared in an open manner. 

3. Personal health data are not recorded by the company without proper informed consent, and patient identifiers are not recorded at all. 

4. For the purpose of raising awareness and providing education about pyruvate kinase deficiency, the Pyruvate Kinase Deficiency Foundation Executive Board and Medical Advisory Board members, staff or individuals representing the pyruvate kinase deficiency community may be invited by pharmaceutical companies to speak about pyruvate kinase deficiency at internal company meetings, public events hosted by the company, or in meetings with regulatory agencies.

5. To avoid any potential appearance of conflict of interest, board members, who have a fiduciary responsibility to the Pyruvate Kinase Deficiency Foundation and direct the acceptance and use of funds provided by pharmaceutical companies, should not testify at regulatory hearings. Patients and members of the pyruvate kinase deficiency community with a connection to the pharmaceutical company, such as a relative of an employee or ownership of stock in the company, should also not testify at regulatory hearings.

Article IV - Financial Contributions

All collaboration and communication efforts outlined in this document will be conducted with pharmaceutical companies regardless of their financial support of the Pyruvate Kinase Deficiency Foundation, or lack thereof. The Pyruvate Kinase Deficiency Foundation will not operate in any manner as a paid service provider to a pharmaceutical company. However, financial resources are critical for the Pyruvate Kinase Deficiency Foundation to achieve its mission and vision to improve the lives of all patients affected by pyruvate kinase deficiency and therefore, the Pyruvate Kinase Deficiency Foundation will gratefully accept donations from pharmaceutical companies according to the following principles

  1. Financial contributions from a pharmaceutical company should be initiated by a written request from the Pyruvate Kinase Deficiency Foundation stating the organization’s mission, activities, programs, and reason for the request. 

  2. A single, unrestricted donation from any given pharmaceutical company in a year is preferable, rather than multiple smaller donations, whenever possible.

  3. Donations from pharmaceutical companies must be given in a named manner; the Pyruvate Kinase Deficiency Foundation does not accept anonymous donations from pharmaceutical companies.

  4. The Pyruvate Kinase Deficiency Foundation may accept money or in-kind payment of property or services as long as there is no arrangement or expectation that the sponsor will receive a substantial return benefit, as defined by IRS regulations.

  5. Financial contributions should be made either as (1) an unrestricted grant or (2) sponsorship of a specific activity or program initiated by the Pyruvate Kinase Deficiency Foundation to support its stated mission.

  6. The Pyruvate Kinase Deficiency Foundation will not use brand names of drugs or other products to acknowledge corporate sponsors.

  7. The Pyruvate Kinase Deficiency Foundation Executive Board and Medical Advisory Board members, staff and volunteers may not receive honoraria, gifts or other goods from corporate sponsors. They may accept travel costs and meals, however, for charity-related business.

  8. The Pyruvate Kinase Deficiency Foundation Executive Board must review gifts of more than $15,000 in a calendar year from drug companies for possible conflicts of interest. 

Article V - Clinical Trial and Approved Therapy Communication

The Pyruvate Kinase Deficiency Foundation will provide information about clinical trials, according to the following principles: 

  1. The Pyruvate Kinase Deficiency Foundation shares information about clinical trials with the community to ensure that patients and families are aware of clinical trials and have the opportunity to make informed decisions about participating. The choice to participate in any given trial is an individual one; the Pyruvate Kinase Deficiency Foundation does not seek to influence that choice, but rather to ensure an informed decision can be made. Patients and families with questions about various trials will be provided with factual information regarding all options and directed to their provider.

  2. The Pyruvate Kinase Deficiency Foundation disseminates accurate, fair and balanced information about clinical trials provided by the pharmaceutical company without additional commentary or opinion that may influence an individual’s decision to participate in a clinical trial or that may change the meaning of the information.

  3. The Pyruvate Kinase Deficiency Foundation recognizes that the choice to share information regarding participation in a clinical trial is personal and does not seek or encourage the sharing of individual clinical trial experiences in social media, email or other means. However, when unsolicited information is shared on the Pyruvate Kinase Deficiency Foundation’s social media platforms, the Pyruvate Kinase Deficiency Foundation provides pharmaceutical companies with community-wide observations and insight regarding needs and barriers to participation to encourage optimal design and implementation of the trial.

  4. The Pyruvate Kinase Deficiency Foundation Board, consultants, volunteers and staff have the responsibility to represent the Pyruvate Kinase Deficiency Foundation in their conduct. Information about clinical trials that is accessible to the community via social media, including in personal blogs or other forms of communication, must adhere to the principles outlined in this document.

  5. The Pyruvate Kinase Deficiency Foundation only provides information about clinical trials that have been approved by appropriate regulatory bodies.

  6. The Pyruvate Kinase Deficiency Foundation does not communicate information in a manner that could be interpreted as advertising or promoting a drug or treatment that has not been approved.

  7. When a therapy has received appropriate regulatory approval, the Pyruvate Kinase Deficiency Foundation disseminates information for educational purposes only and does not promote or encourage the use of any treatment over another. Patients and families with questions about various treatments will be provided with factual information regarding all options and directed to their provider.

Article VI - Patient Privacy

The Pyruvate Kinase Deficiency Foundation places high value on the privacy of patients. Thus, the Pyruvate Kinase Deficiency Foundation does not disclose any personal information or information that can identify a patient. The collection of personal health information by a pharmaceutical company for research purposes should be guided by a protocol and reviewed by an ethics committee. 

The approach outlined in this policy was influenced by the consultation with other similarly sized health organization, including the international FOP Association, the European Federation of Pharmaceutical Industries and Associations (EPPIA) “Code on Interactions between Pharmaceutical Companies and Patient Organizations” and the Pharmaceutical Manufacturers of America (PhRMA) “principles on Interactions with Patient Organizations.”